Olaparib (Lynparza) significantly improved overall survival (OS) versus enzalutamide (Xtandi) or abiraterone acetate (Zytiga) in patients with metastatic castration-resistant prostate cancer (mCRPC) who harbor BRCA1, BRCA2, and/or ATM aberrations, according to results from the final OS analysis of the pivotal phase 3 PROfound trial (NCT02987543).1

In the trial, patients with mCRPC who progressed on previous treatment with a new hormonal agent and harbored BRCA1, BRCA2, or ATM aberrations (n = 245; cohort A) or other alterations in the homologous recombination repair (HRR) pathway (n = 142; cohort B) were randomized 2:1 to receive either olaparib or enzalutamide or abiraterone acetate.

Final OS data from cohorts A and B were presented during the 2020 ESMO Virtual Scientific Program. Results showed that the median OS in cohort A was significantly longer with olaparib than with physicians choice (HR 0.69; 95% CI 0.50-0.97; P = .0175).1In cohort B, the median OS was 14.1 months with olaparib versus 11.5 months with the control (HR, 0.96; 95% CI, 0.63-1.49).

What is exciting about this particular trial is that it showed the feasibility of personalizing care and using precision medicine strategies to preselect patients to maximize the chance of benefit for those who are candidates for these treatments, said Maha H.A. Hussain, MD, FACP, FASCO.We can also help patients avoid unnecessary exposure to ineffective treatments.

Previously published data showed that olaparib resulted in a 66% reduction in the risk of disease progression or death compared with abiraterone or enzalutamide (HR, 0.34; 95% CI, 0.25-0.47;P<.0001).2 Based on these results, the FDA approved olaparib in May 2020 for the treatment of adult patients with deleterious or suspected deleterious germline or somatic HRR genemutated mCRPC who have progressed following prior treatment with enzalutamide or abiraterone.

In an interview with OncLive, Hussain, the Genevieve E. Teuton Professor of Medicine in the Department of Medicine of the Division of Hematology Oncology and the deputy director at the Robert H. Lurie Comprehensive Cancer Center of the Northwestern University Feinberg School of Medicine, further discussed the updated findings from the PROfound trial, its clinical significance in the treatment of patients with mCRPC, and the promise of precision medicine.

Hussain: PROfound is a randomized phase 3 clinical trial. It is one of the first precision medicine clinical trials to complete; patients were preselected based on specific genomic alterations and then randomized accordingly. Patients with mutations in the HRR genes or DNA damage repair genes were assigned to 2 different cohorts. The primary cohort was [comprised of] patients who had BRCA1/2 or ATM mutations, while cohort 2 included [those who harbored] other genes that are involved in the HRR pathway. Patients were randomized 2:1 to olaparib or standard of care per physicians choice of either abiraterone and prednisone or enzalutamide. The primary end point [of the trial] was radiographic progression-free survival (rPFS), which is a meaningful clinical end point, while OS was one of the several key secondary end points [examined].

Data from the Stand Up to Cancer highlighted the fact that over 20% of patients with mCRPC have significant mutations in the DNA repair pathway or the HRR genes. That [research] underscored the fact that this is a clinically relevant pathway to go after. At the time that [the PROfound trial was being designed] we saw evidence of benefit [with this approach] in other tumors [such as] breast and ovarian cancers, and then subsequently, in pancreatic cancer.

The specific pathway relevance is that both normal cells and cancer cells need to repair themselves when there is damage; the HRR pathway is involved in that repair process. However, are alterations or mutations [are present], the cells are not able to repair themselves and they fall back into a different pathway, which is the PARP pathway. Basically, PARP agents tend to inhibit that enzyme so that the [cancer] cells cannot repair themselves.

[Earlier data from the trial were previously published] this past summer. Johann de Bono, MB, ChB, PhD, of The Institute of Cancer Research was the first author on the publication in the New England Journal of Medicine, which highlighted [data regarding] the primary end point of rPFS. In this particular presentation delivered at the 2020 ESMO Virtual Congress, [investigators] reported OS [data from] cohorts A and B.

We saw that the benefit [with olaparib is] not only in terms of rPFS; the benefit translated into a median OS benefit of over 4 months between the arms, despite crossover from the control arm to the olaparib arm at time of progression. Additionally, the risk of death was reduced by 31%, which is very clinically significant. In [the cohort of patients who harbored the] other 12 genes, other than BRCA1/2 and ATM, we saw a trend in OS improvement but it was not statistically significant. The trend was about a little bit over 2 months of a difference. When adjusting for crossover, the trend improved although it was still not statistically significant. However, several patients in cohort B experienced clinical benefits from treatment. The primary benefit [with olaparib] still seems to be driven by BRCA primarily.

No; the overall safety was very much consistent with what was known about olaparib. The most common adverse effects observed included anemia, nausea, and fatigue. Most of these were low-grade events, aside from the anemia. Many of these patients were heavily pretreated; while they might have previously received abiraterone or enzalutamide, they would have also received chemotherapy and other potential anticancer treatment and be fairly advanced in the course of their disease. The findings, overall, are really not surprising and the safety profile very much consistent with what has been observed with the agent in other tumors.

We have reached a major benchmark in the management of this disease. Ever since the original observations regarding androgen deprivation [therapy] in prostate cancer and subsequent treatments, and certainly since the time I entered the field in the early 1990s, prostate cancer management has been more of a one-size-fits-all approach. In fact, when we give chemotherapy and hormone treatment we don't preselect [patients].

We still have [a lot of work to do]. Patients with metastatic castration-resistant disease continue to die from prostate cancer; they also suffer from pain and other factors involved with this disease. This [research] highlights the feasibility of performing precision medicine trials. It also shows us that meaningful clinical benefits could be achieved in these patients. I would hope that our partners across the spectrum will invest further in conducting more clinical trials.

The observation that we've seen with olaparib also opens up the door for potential combination clinical trials, both in castration-resistant disease and potentially in earlier stages of disease, where we might get a better return on investment from a clinical perspective.

Genomic profile evaluation for patients is critical moving forward, not only for the purpose of treatment for the patient. Conducting or counseling the patient regarding germline testing and tumor genomics evaluation in preparation for future treatment is also very critical. Obviously, genetic testing is associated with genetic counseling, [which may allow patients] and potential blood relatives [to get ahead of the game].

Tissue-based genomic evaluation will open the door for the patient to explore different treatments, and [certain] genomic alterations might qualify them for different clinical trials opportunities. [This work] underscores the hope for patients that their cancer can be managed better with genomically targeted treatments, specifically, in this case, the PARP inhibitor. [Now we can build on] these observations in terms of different treatment strategies and combinations.

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PROfound Trial With Olaparib Shows Feasibility of Personalizing Care in mCRPC - OncLive

ROCKVILLE, Md., Oct. 13, 2020 /PRNewswire/ --Foundation to Fight H-ABC, a non-profit organization dedicated to increasing awareness and driving development of a cure for the degenerative children's disease, H-ABC, today announced a sponsored research agreement with the University of Massachusetts Medical School and Yale University to advance a targeted gene therapy for H-ABC.

"We have high hopes to quickly prove efficacy with this approach to move research forward and find a permanent cure for this devastating disease," said Michele Sloan, Co-Founder, Foundation to Fight H-ABC.

H-ABC (hypomyelination with atrophy of the basal ganglia and cerebellum) belongs to a group of conditions called leukodystrophies, diseases that affect the white matter of the brain. These diseases disrupt the growth or maintenance of the myelin sheath, a protective layer that insulates nerve cells and allows for the transmission of messages between cells.

Caused by a mutation in the TUBB4A gene, H-ABC is a rare genetic disorder that affects certain parts of the brainspecifically the basal ganglia and the cerebellum, which control movement. H-ABC targets these important structures, reducing both their size and function. As a result, children who suffer from H-ABC often experience motor problems, cannot walk, talk, or sit on their own. Currently, there is no known cure for this disabling and life-threatening condition.

The teams of Dr. Guangping Gao (University of Massachusetts Medical School) and Dr. Karel Liem (Yale School of Medicine) will combine extensive expertise in the fields of Adeno-associated virus (AAV), a platform for gene delivery for the treatment of a variety of human diseases and H-ABC disease models, to develop AAV vectors to silence or outcompete the mutated TUBB4A gene.

"To date, AAV-based gene delivery system is the vector of choice for in vivo gene therapy of many currently untreatable rare diseases including H-ABC," said Guangping Gao, Ph.D. "We are very excited for starting close collaborations with Dr. Liem's team at Yale and the Foundation to Fight H-ABC to develop potential gene therapeutics for this devastating disease."

"With the support from the Foundation to Fight H-ABC, we are excited to build upon our mechanistic studies of the disease and to collaborate with Dr. Gao of the University of Massachusetts to develop and test AAV approaches to H-ABC," saidKarel F Liem Jr., M.D., Ph.D.

For more information, please visit https://www.h-abc.org/donate.

CONTACT: Sawyer Lipari, [emailprotected]

SOURCE Foundation to Fight H-ABC

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Foundation to Fight H-ABC, University of Massachusetts Medical School and Yale University Initiate Gene Therapy Study Targeting Cure for Rare Disease...

The immune response to infections is a delicate balance. We need just enough action to clear away the offending bacteria or viruses, but not so much that our own bodies suffer collateral damage.

Macrophages are immune cells at the front line, detecting pathogens and kicking off an inflammatory response when needed. Understanding how macrophages determine when to go all-out and when to keep calm is key to finding new ways to strike the right balance particularly in cases where inflammation goes too far, such as in sepsis, colitis and other autoimmune disorders.

Two macrophages (blue) fighting to engulf the same pathogen (green). GIV/Girdin is shown in red.

In a study published October 14, 2020 in the Proceedings of the National Academy of Sciences, researchers at University of California San Diego School of Medicine discovered that a molecule called Girdin, or GIV, acts as a brake on macrophages.

When the team deleted the GIV gene from mouse macrophages, the immune cells rapidly overacted to even small amounts of live bacteria or a bacterial toxin. Mice with colitis and sepsis fared worse when lacking the GIV gene in their macrophages.

The researchers also created peptides that mimic GIV, allowing them to shut down mouse macrophages on command. When treated with the GIV-mimic peptide, the mices inflammatory response was tempered.

When a patient dies of sepsis, he or she does not die due to the invading bacteria themselves, but from an overreaction of their immune system to the bacteria, said senior author Pradipta Ghosh, MD, professor at UC San Diego School of Medicine and Moores Cancer Center. Its similar to what were seeing now with dangerous cytokine storms that can result from infection with the novel coronavirus SARS-CoV-2. Macrophages, and the cytokines they produce, are the bodys own immune-stimulating agents and when produced in excessive amounts, they do more harm than good.

Digging deeper into the mechanism at play, Ghosh and team discovered that the GIV protein normally cozies up to a molecule called Toll-like receptor 4 (TLR4). TLR4 is stuck right through the cell membrane, with bits poking inside and outside the cell. Outside of the cell, TLR4 is like an antenna, searching for signs of invading pathogens. Inside the cell, GIV is nestled between the receptors two feet. When in place, GIV keeps the feet apart, and nothing happens. When GIV is removed, the TLR4 feet touch and kick off a cascade of immune-stimulating signals.

Ghoshs GIV-mimicking peptides can take the place of the protein when its missing, keeping the feet apart and calming macrophages down.

We were surprised at just how fluid the immune system is when it encounters a pathogen, said Ghosh, who is also director of the Institute for Network Medicine and executive director of the HUMANOID Center of Research Excellence at UC San Diego School of Medicine. Macrophages dont need to waste time and energy producing more or less GIV protein, they can rapidly dial their response up or down simply by moving it around, and it appears that such regulation happens at the level of gene transcription.

Ghosh and team plan to investigate the factors that determine how the GIV brake remains in place when macrophages are resting or is removed to mount a response to a credible threat. To enable these studies, the Institute for Network Medicine at UC San Diego School of Medicine recently received a new $5 million grant from the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health. Ghosh shares this award with her colleagues Debashis Sahoo, PhD, assistant professor at UC San Diego School of Medicine and Jacobs School of Engineering, and Soumita Das, PhD, associate professor of pathology at UC San Diego School of Medicine.

Co-authors of the study include: Lee Swanson, Gajanan D. Katkar, Julian Tam, Rama F. Pranadinata, Yogitha Chareddy, Jane Coates, Mahitha Shree Anandachar, Vanessa Castillo, Joshua Olson, Victor Nizet, Irina Kufareva, Soumita Das, all at UC San Diego.

Funding for this research came, in part, from the National Institutes for Health (grants AI141630, AI155696, CA100768, CA160911, DK107585, UL1TR001442, DK 0070202), DiaComp and Helmsley Charitable Trust.

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Protein that Keeps Immune System from Freaking Out Could Form Basis for New Therapeutics - UC San Diego Health

Inherited retinal dystrophies (IRDs) are a group of blinding conditions caused by mutations in more than 270 different genes, including the RPE65 gene3. RPE65-mediated IRDs often disproportionally affect children and young adultsand cause progressive vision loss, leading to complete blindness in almost all patients2. Luxturna is designed to provide functioning copies of theRPE65gene to act in place of mutatedRPE65genes2.These functioning genes produce the RPE65 protein to help improve vision and prevent progression towards total blindness2.

"The effects of RPE65-mediated inherited retinal diseases can be life-changing. Previously, there was no treatment available and the progression towards complete blindness was inevitable." said Dr. Elise Hon, an Ophthalmologist in the Department of Ophthalmology and Vision Sciences and the Director of the Eye Genetics Program at The Hospital for Sick Children (SickKids) in Toronto. "This approval is a very important step forward in the treatment of genetic eye disorders."

Due to the highly specialized nature of the therapy, Novartis is collaborating with key centres and their multidisciplinary teams to deliver Luxturna to patients across Canada: SickKids in partnership with Sunnybrook Health Sciences Centre in Ontario, and Montreal Children's Hospital, McGill University Health Centre in partnership with Maisonneuve-Rosemont Hospital (HMR), Centre intgr universitaire de sant et de services sociaux (CIUSSS) de l'Est-de-l'le-de-Montral, affiliated with Universit de Montral in Quebec.

"Being part of tremendous innovations in the treatment of certain eye conditions over the past decades has been incredibly rewarding. Gene therapy heralds the start of a new era for IRDs and I'm thrilled to be part of this historic moment and equally excited to be able to give patients a chance to regain sight with Luxturna," said Dr. Peter Kertes, retina surgeon and Ophthalmologist-in-Chief, Sunnybrook Health Sciences Centre and staff ophthalmologist at SickKids in Toronto.

The current standard of care for people born with IRDs caused by RPE65 gene mutations is supportive in nature and focuses on monitoring, psychological support, mobility training and visual rehabilitation4. Until now, no pharmacological treatment option was available to treat the underlying disease mechanism or alter the natural history of inherited retinal dystrophies. While a genetic test is needed to confirm that vision loss is caused by mutations in theRPE65gene2, it can be a lengthy process to access testing and counselling. Novartis has entered into a partnership with Blueprint Genetics to help facilitate genetic testing where appropriate in order to validate the diagnosis.

"The approval of the first gene replacement therapy for Canadians is historical. We have been waiting for this moment in the vision community for decades. To be able to tell a parent that their child's impaired sight could now be restored or improved is remarkable,' said Doug Earle, President & CEO of Fighting Blindness Canada. "We welcome this medical innovation and hope that Canadians in need of this therapy have access to it without delay."

"Novartis is proudly reimagining medicine by bringing forward innovations like Luxturna. Today's approval will have a significant impact on patient care," said Andrea Marazzi, Country Head, Novartis Pharmaceuticals Canada. "We are grateful to the vision community for rallying behind Canadians who are impacted by vision impairment and vision loss and we are committed to helping them gain access to this game-changing gene therapy as quickly as possible."

AboutRPE65mutation-associated inherited retinal dystrophyMutations in both copies of theRPE65gene affect approximately 1 in 200,000 people and can lead to blindness5,6. Early in the disease patients can suffer from night blindness (nyctalopia), loss of light sensitivity, loss of peripheral vision, loss of sharpness or clarity of vision, impaired dark adaptation and repetitive uncontrolled movements of the eye (nystagmus)6. Patients with mutations in both copies of theRPE65gene may be diagnosed, for instance, with subtypes of either Leber congenital amaurosis or retinitis pigmentosa7.

About Novartis in Cell & Gene TherapyNovartis is at the forefront of cell and gene therapies designed to halt diseases in their tracks or reverse their progress rather than simply manage symptoms. The company is collaborating on the cell and gene therapy frontier to bring this major leap in personalized medicine to patients with a variety of diseases, including genetic disorders and certain deadly cancers. Cell and gene therapies are grounded in careful research that builds on decades of scientific progress. Following key approvals of cell and gene therapies by health authorities, new treatments are being tested in clinical trials around the world.

About Novartis in CanadaNovartis Pharmaceuticals Canada Inc., a leader in the healthcare field, is committed to the discovery, development and marketing of innovative products to improve the well-being of all Canadians. In 2019, the company invested $51.8 million in research and development in Canada. Located in Dorval, Quebec, Novartis Pharmaceuticals Canada Inc. employs approximately 1,500 people in Canada and is an affiliate of Novartis AG, which provides innovative healthcare solutions that address the evolving needs of patients and societies. For further information, please consult http://www.novartis.ca.

About Novartis globallyNovartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach more than 750 million people globally and we are finding innovative ways to expand access to our latest treatments. About 109,000 people of more than 145 nationalities work at Novartis around the world. Find out more at http://www.novartis.com.

Luxturna is a registered trademark of Spark Therapeutics Inc., used under license by Novartis Pharmaceuticals Canada Inc.

References

1.

Novartis Pharmaceuticals Canada Inc. Luxturna(voretigene neparvovec) Product Monograph. October 13, 2020.

2.

Russell S et al. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65- mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. The Lancet 2017; 390:849-860

3.

RetNet. Summaries of genes and loci causing retinal diseases. Available at: https://sph.uth.edu/retnet/sum-dis.htm.

4.

National Institute for Health and Care Excellence (NICE). Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations [ID1054]2018:199/799

5.

Novartis. Data on file. 2018.

6.

Astuti GD et al. Comprehensive genotyping reveals RPE65 as the most frequently mutated gene in Leber congenital amaurosis in Denmark. European Journal of Human Genetics 2016; 24: 107179.

7.

Morimura H et al. Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or Leber congenital amaurosis. Proceedings of the National Academy of Sciences of the USA. 1998; 95: 308893.

SOURCE Novartis Pharmaceuticals Canada Inc.

For further information: Novartis Media Relations: Julie Schneiderman, +1 514 633 7873, E-mail: [emailprotected]

http://www.novartis.ca

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Health Canada approves first-ever gene replacement therapy, Luxturna - Canada NewsWire

TORONTO -- For the thousands of Canadians at risk of blindness, eight-year-old Sam is a beacon of hope.

He is the first Canadian to be treated with gene replacement therapy for a rare form of blindness which had left Sam unable to see sky on a cloudy day, and unable to make out shapes in the dark.

Sometimes you have to walk in the night and I couldnt see things and you bump into things, Sam told CTV News.

He had to have lights on always, and had trouble seeing his shoes or objects on the floor. And the condition was progressive, meaning things would get worse as he grew older -- a daunting prospect when there was no treatment available.

But now he can see cloudy skies, shoes and more. The best part of his improved vision, says Sam, are the stars at night.

I never saw stars before, he said. And I also never saw airplanes flying at night.

He was diagnosed after birth with a genetic disorder called retinitis pigmentosa, a form of genetic retinal degeneration resulting from mutations in the RPE65 gene.

You lose perception of light, Dr. Elise Heon, of Sick Kids Hospital, explained to CTV News. You end up in darkness and [its] slowly progressive, it's relentless, your visual field shrinks and shrinks and shrinks and shrinks.

Retinitis pigmentosa (RP) affects between 1 in 3,500 to 1 in 4,000 Canadians, according to Fighting Blindness Canada. It actually refers to a group of disorders, as there are numerous versions of RP depending on which pair of genes are damaged. More than 64 genes have been identified by scientists as potentially having mutations that cause RP.

Now, Canada has approved the first-ever gene replacement therapy for this form of blindness. Sick Kids Hospital has 29 children in its program with this mutation. The drug can be used on children and adults with the condition, but the earlier its used, the more sight it will save, doctors believe.

It's a huge deal, because for these patients before, theres no treatments, Heon said.

She said she had recently met two patients, brothers, who were suffering the same problem as Sam, and for the first time, she was able to provide hope.

They're 10 years old, and they're losing their vision, she said. If we do nothing, they're just going, fine, they'll just end up with no light reception. So for the first time [we were] able to say, well, actually we need to have a discussion. And it was just, it was priceless.

The gene therapy, which goes by the brand name Luxturna, was developed in the U.S by the drug company Spark Therapeutics.

It works by placing a copy of the healthy gene into inactivated viruses, which are then injected into the retina. The gene then allows cells to produce the necessary protein to convert light into an electrical signal in the retina in order to provide healthy vision and prevent progression of the disease.

It is the first targeted gene therapy to be approved by Health Canada, which gave it the all-clear this week.

Back in 2019, Sam and his family travelled to the U.S to get the new gene therapy because it wasnt available in Canada yet.

His mother, Sarah Banon, noticed changes quickly.

About a week later, I noticed he could get dressed by [himself], she said. He could get his shoes on by himself, independently.

His improvements have continued in the year since he first received the gene therapy.

He is so much more confident, his mother told CTV News. Like getting dressed by himself, matching clothes, doesnt have to have things enlarged. Being able to [see], even when its dark outside, no lights on and it is a cloudy day. He would have to, at school, keep the lights on.

Now he is able to function as a normal child.

With the approval of this gene therapy in Canada, doctors are hoping to be able to use it on more patients who qualify -- and the earlier the better.

Dr. Peter Kertes, a vitreo-retinal surgeon and Ophthalmologist-in-Chief at Sunnybrook Health Sciences Centre, told CTV News that the approval of the therapy is fantastic.

This is a huge breakthrough, he said. Most of the advances that we have in medicine are incremental. Every once in a while, once in a generation, something revolutionary like this comes along that really changes the course of therapy.

Luxturna specifically treats individuals with biallelic mutations of the RPE65 gene -- meaning they have mutations in that gene stemming from both parents -- which manifests as either RP or Leber congenital amaurosis (LCA). Its a very small patient group compared to the entirety of Canadians with inherited retinal diseases.

This may be just one gene therapy for one condition, but it will open to the door to this strategy being used in other scenarios, Kertes pointed out.

This is the tip of the iceberg. I think this is a vector that will prove to be very effective and holds great promise, he said. I think many people who are living with blindness or facing blindness, have much to look forward to. I think we're on the cusp of a revolution in this group of diseases.

The company licensing the therapy, Novartis Pharmaceuticals Canada Inc., isnt detailing the cost, but based on the price in the U.S it could top $1.1 million in Canada, making it among the most expensive drugs in the country.

The therapy is currently under review by both the Canadian Agency for Drugs and Technologies in Health (CADTH) and the Institut national dexcellence en sant et en services sociaux (INESSS).

Novartis said in a statement that they look forward to receiving their recommendations following Health Canadas approval.

They said they are eager to help eligible Canadians affected by this rare disease gain access to the first-ever gene replacement therapy as quickly as possible.

The Patented Medicine Prices Review Board will be disclosing their new guidelines in terms of capping drug prices in an online media briefing this Thursday.

As this will likely be the first of many gene replacement therapies -- with similarly high price tags -- Ottawa and the provinces will have to make the decision on whether it will be covered by provincial health plans. The question is an ongoing ethical debate, with some saying that drug companies will only take advantage of it if governments show that they are willing to pay.

Should it be the responsibility for the government to pay for any drug at any price? Marc-Andr Gagnon, a researcher with Carleton University who looks into pharmaceutical policy, told CTV News. The problem is, if we say yes to this question, you can be sure that the day after, all the drugs in the market will be asking for much higher prices.

Its a very expensive drug, Heon acknowledged.

However, she pointed out that this is a rare disease, and its not a recurrent treatment. Its a one-time injection to the eyes.

You treat both eyes and then thats it, she said.

To be able to change someone's life is quite a privilege. And to be able to prevent someone from going blind is a real privilege.

For Sam and his mother, the gift of independence has been priceless.

This is a story of hope, his mother said. A child told it is what it is.

And now, when he looks up at night, he can see stars.

View original post here:
'I never saw stars before': Gene therapy brings back 8-year-old Canadian boy's sight - CTV News

Oct. 7, 2020 10:45 UTC

NEW YORK & BRISBANE, Calif.--(BUSINESS WIRE)-- Pfizer Inc. (NYSE: PFE) and Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today announced that the first participant has been dosed in the Phase 3 AFFINE study of giroctocogene fitelparvovec (SB-525), an investigational gene therapy for hemophilia A patients.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201007005049/en/

AFFINE is a global Phase 3, open-label, multicenter, single arm study that will evaluate the efficacy and safety of giroctocogene fitelparvovec in patients with moderately severe to severe hemophilia A. The primary endpoint is impact on annual bleed rate (ABR) through 12 months following treatment with giroctocogene fitelparvovec, compared to ABR on Factor VIII (FVIII) replacement therapy collected in the Phase 3 lead-in study period. Participants will be analyzed throughout the 5-year study period following the single infusion to further assess the durability and efficacy.

The initiation of the pivotal Phase 3 dosing study of giroctocogene fitelparvovec is a significant achievement for Pfizer as we continue our longstanding commitment to improving care for the hemophilia community, said Brenda Cooperstone, Chief Development Officer, Rare Disease, Pfizer Global Product Development. Enrollment in the lead-in study is progressing well and recruitment is on track for Phase 3. Given the Phase 1/2 study findings to date, we believe that giroctocogene fitelparvovec has the potential to sustain factor levels and reduce annual bleed rates, suggesting this one-time gene therapy could potentially transform the standard of care for eligible patients worldwide.

Data from the Phase 3 lead-in study will provide a baseline for patients evaluated in the Phase 3 study. Updated Phase 1/2 data announced at a Pfizer investor event on September 15, 2020 demonstrated that giroctocogene fitelparvovec was generally well tolerated. Each of the five patients in the high dose cohort sustained FVIII activity levels without bleeds or the need for prophylactic factor through up to 85 weeks. Factor VIII activity levels were sustained at a clinically meaningful level, with a geometric mean of ~71% when measured between the weeks of 9 and 52.

We are encouraged that findings from the Phase 1/2 Alta study met two critically important measures for the hemophilia A patient community, showing clinically meaningful factor levels and reduced bleeds, said Bettina M. Cockroft, M.D., M.B.A, Chief Medical Officer of Sangamo. The progress of this program, the most advanced of our gene therapy product candidates, into Phase 3 is an important milestone for Sangamo, as it represents our first asset in a registrational trial.

Per the terms of the collaboration agreement, Sangamo has now earned a $30 million milestone payment. The giroctocogene fitelparvovec collaboration was established in May 2017. Under the terms, Pfizer is now operationally and financially responsible for research, development, manufacturing and commercialization activities for giroctocogene fitelparvovec following the transfer of the Investigational New Drug (IND) from Sangamo to Pfizer in December 2019. Sangamo is eligible to receive total potential milestone payments of up to $300 million for the development and commercialization of giroctocogene fitelparvovec, and up to $175 million for additional hemophilia A gene therapy product candidates that may be developed under the collaboration. Sangamo will, additionally, receive tiered royalties starting in the low teens and up to 20% of annual net sales of giroctocogene fitelparvovec.

About the AFFINE study

The Phase 3 AFFINE (efficAcy and saFety Factor vIii geNe thErapy in hemophilia A patients; NCT04370054) study is an open-label, multicenter, single arm study to evaluate the efficacy and safety of a single infusion of giroctocogene fitelparvovec in more than 60 adult (ages 18-64 years) male participants with moderately severe to severe hemophilia A. Eligible study participants will have completed at least six months of routine FVIII prophylaxis therapy during the lead-in Phase 3 study (NCT03587116) in order to collect pretreatment data for efficacy and selected safety parameters.

The primary endpoint is impact on ABR through 12 months following treatment with giroctocogene fitelparvovec, as compared to ABR on prior FVIII prophylaxis replacement therapy. The secondary endpoint is FVIII activity level after the onset of steady state and through 12 months following infusion of giroctocogene fitelparvovec.

About giroctocogene fitelparvovec

Giroctocogene fitelparvovec (SB-525 or PF-07055480) comprises a recombinant adeno-associated virus serotype 6 vector (AAV6) encoding the complementary deoxyribonucleic acid for B domain deleted human FVIII. The giroctocogene fitelparvovec expression cassette was designed for optimal liver-specific expression of FVIII protein and supports production of high yields of the vector. The giroctocogene fitelparvovec transcriptional cassette incorporates multi-factorial modifications to the liver-specific promoter module, FVIII transgene, synthetic polyadenylation signal and vector backbone sequence.

The U.S. Food and Drug Administration has granted Orphan Drug, Fast Track, and regenerative medicine advanced therapy (RMAT) designations to giroctocogene fitelparvovec, which also received Orphan Medicinal Product designation from the European Medicines Agency. Giroctocogene fitelparvovec is being developed as part of a global collaboration between Sangamo and Pfizer.

About Hemophilia A

Hemophilia is a genetic hematological rare disease that results in a deficiency of a protein that is required for normal blood clottingclotting factor VIII in hemophilia A. The severity of hemophilia that a person has is determined by the amount of factor in the blood. The lower the amount of the factor, the more likely it is that bleeding will occur which can lead to serious health problems.

Hemophilia A occurs in approximately one in every 5,000-10,000 male births worldwide. For people who live with hemophilia A, there is an increased risk of spontaneous bleeding as well as bleeding following injuries or surgery. It is a lifelong disease that requires constant monitoring and therapy.

About Sangamo Therapeutics

Sangamo Therapeutics is committed to translating ground-breaking science into genomic medicines with the potential to transform patients lives using gene therapy, ex vivo gene-edited cell therapy, and in vivo genome editing and gene regulation. For more information about Sangamo, visit http://www.sangamo.com.

About Pfizer Rare Disease

Rare disease includes some of the most serious of all illnesses and impacts millions of patients worldwide, representing an opportunity to apply our knowledge and expertise to help make a significant impact on addressing unmet medical needs. The Pfizer focus on rare disease builds on more than two decades of experience, a dedicated research unit focusing on rare disease, and a global portfolio of multiple medicines within a number of disease areas of focus, including rare hematologic, neurologic, cardiac and inherited metabolic disorders.

Pfizer Rare Disease combines pioneering science and deep understanding of how diseases work with insights from innovative strategic collaborations with academic researchers, patients, and other companies to deliver transformative treatments and solutions. We innovate every day leveraging our global footprint to accelerate the development and delivery of groundbreaking medicines and the hope of cures.

Click here to learn more about our Rare Disease portfolio and how we empower patients, engage communities in our clinical development programs, and support programs that heighten disease awareness.

Pfizer Inc.: Breakthroughs that change patients lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at http://www.pfizer.com. In addition, to learn more, please visit us on http://www.pfizer.com and follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

SANGAMO DISCLOSURE NOTICE:

This press release contains forward-looking statements regarding Sangamo's current expectations. These forward-looking statements include, without limitation, statements relating to the potential to develop, obtain regulatory approvals for and commercialize SB-525 as a safe and effective therapy to treat hemophilia A, the potential long-term durability of SB-525 therapy, anticipated plans and timelines for conducting phase 3 clinical trials and sharing additional clinical data, the potential for Sangamo to earn milestone payments and royalties under its collaboration with Pfizer and the timing of such payments and royalties and other statements that are not historical fact. These statements are not guarantees of future performance and are subject to risks and uncertainties that are difficult to predict. Sangamos actual results may differ materially and adversely from those expressed. There can be no assurance that Sangamo will earn any additional milestone or royalty payments under the Pfizer collaboration. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to: the evolving COVID-19 pandemic and its impact on the global business environment, healthcare systems and the business and operations of Sangamo and Pfizer; the research and development process; the uncertain timing and unpredictable results of clinical trials, including whether final clinical trial data will validate the safety and efficacy of SB-525; the unpredictable regulatory approval process for product candidates across multiple regulatory authorities; the manufacturing of products and product candidates; the commercialization of approved products; the potential for technological developments that obviate technologies used by Sangamo and Pfizer in SB-525; the potential for Pfizer to terminate the SB-525 program or to breach or terminate its collaboration agreement with Sangamo; and the potential for Sangamo for fail to realize its expected benefits of its collaboration with Pfizer. These risks and uncertainties are described more fully in Sangamo's filings with the U.S. Securities and Exchange Commission, including its most recent Quarterly Report on Form 10-Q for the quarter ended June 30, 2020 and Annual Report on Form 10-K for the year ended December 31, 2019. The information contained in this release is as of October 7, 2020, and Sangamo undertakes no duty to update forward-looking statements contained in this release except as required by applicable laws.

PFIZER DISCLOSURE NOTICE:

The information contained in this release is as of October 7, 2020. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about an investigational hemophilia A therapy, giroctocogene fitelparvovec (SB-525, or PF-07055480), including its potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; risks associated with interim data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when drug applications for any potential indications for giroctocogene fitelparvovec may be filed in any jurisdictions; whether and when regulatory authorities in any jurisdictions may approve any such applications, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether giroctocogene fitelparvovec will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of giroctocogene fitelparvovec; uncertainties regarding the impact of COVID-19 on Pfizers business, operations and financial results; and competitive developments.

A further description of risks and uncertainties can be found in Pfizer's Annual Report on Form 10-K for the fiscal year ended December 31, 2019 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned "Risk Factors" and "Forward-Looking Information and Factors That May Affect Future Results", as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at http://www.sec.gov and http://www.pfizer.com.

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Pfizer and Sangamo Dose First Participant in Phase 3 Study Evaluating Hemophilia A Gene Therapy Treatment - BioSpace

Prostate cancer is the second most commonly occurring cancer in men and the fourth most common cancer worldwide. Furthermore, prostate cancer is the fifth leading cause of cancer-related deaths worldwide, thus representing a major health issue in the global community. The ERG oncogene, a member of the ETS family of transcription factor encoding genes, is a key regulator of cell proliferation, differentiation, angiogenesis, inflammation and apoptosis.1 ERG is not expressed in normal prostate tissue but is a genetic driver of prostate cancer where it is activated through a fusion with the androgen-responsive TMPRSS2 promoter. Importantly, this fusion is present in approximately 50% of cases, highlighting its importance as a potential therapeutic target. We have recently shown that ERG expression is increased in patients with advanced prostate cancer and that higher levels of ERG are associated with seminal vesicle invasion (stage T3b) and biochemical recurrence2 (prostate-specific antigen (PSA)only recurrence). However, targeting of these ETS transcription factors with small molecules has been challenging due to their lack of druggable active sites. As such, there is significant interest in developing novel therapeutic approaches and agents that could target ERG.

Here,3 we have taken a novel approach to target ERG at the gene level rather than at the protein level and to our knowledge, this is the first example demonstrating the promise of using splice-switching oligonucleotides (SSOs) to target ERG in prostate cancer. SSOs are an antisense technology that work by interfering with pre-mRNA splicing and can be used to cause skipping of specific disease-associated exons from the pre-mRNA. The fusion between ERG and TMPRSS2 most often occurs between TMPRSS2 exons 1 or 2 and exon 4 of ERG. We decided to cause skipping of ERG exon 4 (218bp in size) with SSOs resulting in a frame-shift and premature stop codon, leading to nonsense-mediated decay (NMD) of the transcript and thus reduced ERG protein levels (Fig.1).

Figure 1. Overview of the study

We designed a panel of SSOs and tested these in two ERG-positive cancer cell lines VCaPs (prostate cancer) and MG63s (osteosarcoma). In both lines we found that our SSOs caused skipping of exon 4 leading to reduced ERG protein levels (Fig.2, left panel). This in turn resulted in decreased cell proliferation, cell migration and significantly increased apoptosis in both VCaPs and MG63s (Fig.2, left panel). Furthermore, since ERG has been shown to drive Wnt/-catenin signalling in the context of prostate cancer, we analysed the levels of several Wnt pathway components as well as pathway activity with TopFlash assays and found that pathway activity was significantly reduced (Fig.2, left panel).

We next investigated the potential of our SSOs to affect tumour growth using in vivo xenograft models of prostate cancer. Here we found that our most potent SSO based on our in vitro work could reduce tumour growth when delivered systemically (Fig.2, middle panel) and when we analysed excised tumours, we found significant exon 4 skipping and reduced ERG protein levels (Fig.2, middle panel). Importantly our SSO had no apparent toxic effects and did not affect endogenous ERG levels in the mice, demonstrating strong specificity to human ERG. Finally, we tested our most-promising SSO in an ex vivo system using patient-derived tumour explants (Fig.2, right panel). In these assays we demonstrated that SSO treatment reduced ERG protein levels in cultured tumour explants resulting in increased expression of PTEN protein (Fig.2, right panel), which we have previously shown to be repressed by ERG in prostate cancer cells.4

Figure 2. Main findings of the study

Our promising study3 paves the way for larger future studies aimed at testing similarly designed SSOs with improved delivery chemistry in larger cohorts of mouse models of prostate cancer as well as additional ex vivo patient tumour-derived samples to demonstrate the translational potential of this therapeutic approach.

Written by:Sean Porazinski, Faculty of Medicine, St Vincent's Clinical School, University of NSW;The Kinghorn Cancer Centre, 384 Victoria St.Darlinghurst, Sydney, NSW, 2010, Australia and Michael Ladomery,Faculty of Health and Applied Sciences, University of the West of England, Bristol, UK.

References:

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Targeting the ERG Oncogene with Splice-Switching Oligonucleotides as a Novel Therapeutic Strategy in Prosta... - UroToday

Gosselies, Belgium, 5 October 2020, 7am CEST BONE THERAPEUTICS(Euronext Brussels and Paris: BOTHE), the cell therapy company addressing unmet medical needs in orthopedics and other diseases, Link Health Pharma Co., Ltd (Link Health) and Shenzhen Pregene Biopharma Company, Ltd (Pregene) today announce the signing of an exclusive license agreement for the manufacturing, clinical development and commercialization of Bone Therapeutics allogeneic, off-the-shelf, bone cell therapy platform ALLOB in China (including Hong Kong and Macau), Taiwan, Singapore, South Korea, and Thailand.

Under the agreement, Bone Therapeutics is eligible to receive up to 55 million in development, regulatory and commercial milestone payments including 10 million in upfront and milestone payments anticipated in the next 24 months. Bone Therapeutics is also entitled to receive tiered double-digit royalties on annual net sales of ALLOB. Bone Therapeutics retains development and commercialization rights to ALLOB in all other geographies outside of those covered by this agreement. As a result, Bone Therapeutics will continue to concentrate on its development and commercialization plans for ALLOB in the US and Europe and novel innovative cell-based products globally.

This collaboration between Bone Therapeutics, Link Health and Pregene expands our geographic reach and demonstrates the global commercial potential of ALLOB,said Miguel Forte, MD, PhD, Chief Executive Officer of Bone Therapeutics. We already have operational experience in Asia with the Phase III clinical trial of our lead product JTA-004 in Hong Kong. We selected Link Health and Pregene to partner with us in Asia as a result of their expertise in advanced therapeutics and cell therapies, their proven track record of development and commercial implementation in Chinese and Asian markets, and Pregenes well established cell therapy manufacturing capacity. Bone Therapeutics will continue to develop the ALLOB cell therapy platform for other markets while exploring additional partnership opportunities in the U.S. and Europe.

The agreement grants Link Health and Pregene exclusive rights to clinically develop and commercialize ALLOB for the treatment of human bone disorders in Greater China, Taiwan, Singapore, South Korea, and Thailand. All rights for China will be transferred to Pregene and Link Health will gain rights for the remaining countries Bone Therapeutics will share its patented proprietary manufacturing expertise for the expansion and differentiation of bone-forming cells and has the option to sell clinical supplies to Link Health and Pregene in preparation for their clinical development of ALLOB.

This collaboration and license agreement for Bone Therapeutics ALLOB provides a strong addition to our pipeline. ALLOB has demonstrated the potential to reduce the recovery time and stimulate bone growth for a variety of bone conditions, and to have a considerable impact on patients lives,said Yan Song, PhD, Chief Executive Officer of Link Health. It is important for Link Health to collaborate with companies that have strong therapeutic product portfolios and entrepreneurial management. This partnership with Bone Therapeutics is a direct result of our shared commitment to appreciate the enormous potential of cell therapy and regenerative medicine.

Pregene now has a flourishing portfolio of CAR-T cell therapy-based cancer treatments. Bone Therapeutics ALLOB provides anallogeneic, off-the-shelf cell therapy that expands our portfolio of cell therapies to include the sizable commercial potential of orthopedics,said Hongjian Li, Co-founder and Chief Executive Officer of Pregene. We expect to be able to leverage our extensive international cell and gene therapy experience to develop Bone Therapeutics ALLOB platform and subsequently launch products in China and Southeast Asian markets.

ALLOB, an allogeneic and off-the-shelf cell therapy product manufactured through a proprietary, scalable production process, consists of human bone-forming cells derived from cultured bone marrow mesenchymal stem cells of healthy adult donors. In preclinical studies ALLOB has shown to reduce healing time in a delayed-union fracture model by half, and has demonstrated good tolerability and signs of efficacy in two Phase IIa studies for two separate indications. The Companys randomized, placebo-controlled, double-blind Phase IIb clinical trial in patients with difficult tibial fractures has received approval from regulatory authorities in six of the seven planned European countries to date, and is expected to enroll the first patient later this year.

About Link Health Pharma Co., Ltd

Link Health is a leading Chinese pharmaceutical company based in Guangzhou, Southern China, focusing on the development of innovative drugs for unmet medical needs.

Link Health has created a highly professional team with diverse expertise in drug development, medical affairs and regulatory affairs. Leveraging deep understanding of China market, regulatory environment and strong network with global biopharmaceutical companies, Link Health is well positioned to bring innovative drugs to the market efficiently. The company has a drug development pipeline of 5 clinical stage assets and 1 under NDA reviewing in China.

The company has also established a fully owned subsidiary in Amsterdam, the Netherlands. The Dutch office builds and further strengthen collaborations with global pharma/biotech partners and research institutes.

About Pregene Biopharma Co., Ltd

Shenzhen Pregene Biopharma Co. Ltd is a leading enterprise in the cell and gene therapy field with the core technology for industrialization. The companys core team comes from well-known institutions and companies including the Academy of Military Medical Sciences, the University of Toronto, and the US FDA.

Pregene has established the gene editing platform, viral vector and cell production platform, nanobody selection platform and other small to pilot trial manufacturing system, with total investment over 100 million CNY. It has the laboratories and GMP plants for cell and gene therapy of over 10,000 square meter.

The company focuses on the research and development of cell and gene therapy drugs, and participated in the drafting the national standard Considerations for CAR-T Cell Quality Study and Non-clinical Evaluation issued by the National Institutes for Food and Drug Control in June 2018. The CAR-T cell therapy for the treatment of multiple myeloma have obtained NMPA IND clearance as the Class I new drug, which is the first in China and fastest in the world using the humanized single domain antibody in CAR construct, and phase I clinical trials are now in progress. Other pipelines such as CAR-T, TCR-T and mRNA drugs for tumors, autoimmune diseases and other indications are in the development at different stages. The company has broad development prospects with the abundant backup technologies.

Looking forward to the future, the company will build the core capacity in one-stop solution for cell and gene therapy drugs, and fulfill the Express of innovative medicine development from drug discovery to clinical products.

About Bone Therapeutics

Bone Therapeutics is a leading biotech company focused on the development of innovative products to address high unmet needs in orthopedics and other diseases. The Company has a, diversified portfolio of cell and biologic therapies at different stages ranging from pre-clinical programs in immunomodulation to mid-to-late stage clinical development for orthopedic conditions, targeting markets with large unmet medical needs and limited innovation.

Bone Therapeutics is developing an off-the-shelf next-generation improved viscosupplement, JTA-004, which is currently in phase III development for the treatment of pain in knee osteoarthritis. Consisting of a unique combination of plasma proteins, hyaluronic acid a natural component of knee synovial fluid, and a fast-acting analgesic, JTA-004 intends to provide added lubrication and protection to the cartilage of the arthritic joint and to alleviate osteoarthritic pain and inflammation. Positive phase IIb efficacy results in patients with knee osteoarthritis showed a statistically significant improvement in pain relief compared to a leading viscosupplement.

Bone Therapeutics core technology is based on its cutting-edge allogeneic cell therapy platform with differentiated bone marrow sourced Mesenchymal Stromal Cells (MSCs) which can be stored at the point of use in the hospital. Currently in pre-clinical development, BT-20, the most recent product candidate from this technology, targets inflammatory conditions, while the leading investigational medicinal product, ALLOB, represents a unique, proprietary approach to bone regeneration, which turns undifferentiated stromal cells from healthy donors into bone-forming cells. These cells are produced via the Bone Therapeutics scalable manufacturing process. Following the CTA approval by regulatory authorities in Europe, the Company is ready to start the phase IIb clinical trial with ALLOB in patients with difficult tibial fractures, using its optimized production process. ALLOB continues to be evaluated for other orthopedic indications including spinal fusion, osteotomy, maxillofacial and dental.

Bone Therapeutics cell therapy products are manufactured to the highest GMP standards and are protected by a broad IP (Intellectual Property) portfolio covering ten patent families as well as knowhow. The Company is based in the BioPark in Gosselies, Belgium. Further information is available atwww.bonetherapeutics.com.

For further information, please contact:

Bone Therapeutics SAMiguel Forte, MD, PhD, Chief Executive OfficerJean-Luc Vandebroek, Chief Financial OfficerTel: +32 (0)71 12 10 00investorrelations@bonetherapeutics.com

For Belgian Media and Investor Enquiries:BepublicCatherine HaquenneTel: +32 (0)497 75 63 56catherine@bepublic.be

International Media Enquiries:Image Box CommunicationsNeil Hunter / Michelle BoxallTel: +44 (0)20 8943 4685neil.hunter@ibcomms.agency / michelle@ibcomms.agency

For French Media and Investor Enquiries:NewCap Investor Relations & Financial CommunicationsPierre Laurent, Louis-Victor Delouvrier and Arthur RouillTel: +33 (0)1 44 71 94 94bone@newcap.eu

For US Media and Investor Enquiries:LHA Investor RelationsYvonne BriggsTel: +1 310 691 7100ybriggs@lhai.com

Certain statements, beliefs and opinions in this press release are forward-looking, which reflect the Company or, as appropriate, the Company directors current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its subsidiary undertakings or any such persons officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.

Excerpt from:
Bone Therapeutics, Link Health and Pregene to develop and commercialize the ALLOB allogeneic bone cell therapy platform in China and Southeast Asia -...

Oral MET inhibitor capmatinib showed clinical activity in patients with MET exon 14mutated advanced nonsmall cell lung cancer (NSCLC), including those with brain metastases. The results of the phase 2 GEOMETRY mono-1 trial were recently published in the New England Journal of Medicine.

The trial (ClinicalTrials.gov Identifier: NCT02414139) included 364 patients with MET-dysregulated advanced NSCLC who were grouped into cohorts on the basis of prior lines of therapy and MET status (MET exon 14 skipping mutation or number of MET amplification gene copy numbers).

Among patients with MET exon 14mutated disease, the overall response rate was 41% (95% CI, 29%-53%) for those previously treated, which exceeded the 35% prespecified threshold for clinically relevant activity. The response rate was 68% (95% CI, 48%-84%) for those who had no prior treatment, which exceeded the 55% prespecified threshold for clinically relevant activity.

Activity was also seen in patients with brain metastases, with 7 of 13 patients with a MET exon 14 skipping mutation achieving an intracranial response 4 of which were complete responses.

Among patients with MET-amplified disease, the response rates were highest for previously treated and untreated patients with a gene copy number of at least 10 (29% and 40%, respectively), but neither were high enough to exceed the prespecified thresholds for clinically relevant activity.

The most common treatment-related adverse events were peripheral edema, nausea, vomiting, and increased blood creatinine level. One patient died from pneumonitis, which was suspected to be related to capmatinib.

The study authors concluded that capmatinib may be a new therapeutic option in patients with MET exon 14mutated advanced NSCLC.

Reference

Wolf J, Seto T, Han J, et al. Capmatinib in MET exon 14mutated or MET-amplified nonsmall-cell lung cancer. N Engl J Med. 2020;383:944-957. doi:10.1056/NEJMoa2002787

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Capmatinib Shows Clinical Activity in MET Exon 14Mutated NSCLC - Cancer Therapy Advisor

The foremost aim of this report titled Global CRISPR And CRISPR-Associated Genes Market isto provide valuable understandings into CRISPR And CRISPR-Associated Genes market to the industry participants including company accounts, industry investors, and industry members to facilitate them to adopt correct strategic decisions regarding the opportunities in the global CRISPR And CRISPR-Associated Genes market.

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To summarize this, the CRISPR And CRISPR-Associated Genes market report discloses major discoveries in the and conclusions about this industry. Similarly, the report reveals different CRISPR And CRISPR-Associated Genes information about various aspects including but not limited to, traders/distributors, suppliers, manufacturers, sales channel, and appendix. In a word, the complete CRISPR And CRISPR-Associated Genes report adds up to the insights available at the hands of industry participants in the CRISPR And CRISPR-Associated Genes market.

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Global CRISPR And CRISPR-Associated Genes Market Report Provide Insights into Growth strategic Adopted by Top Companies || Thermo Fisher Scientific,...

October 5-16, 2020. Report on Cell and Gene Therapy to be discussed.

CPhI Annual Reportlaunched ahead of the first CPhI Festival of Pharma(5-16 October, 2020), the worlds largest digital pharma Expopredicts dramatic growth of new mAb production in China, capacity shortages for cell and gene therapies in the USA, and the widespread global adoption of single-use technologies, but only limited continuous bioprocessing.

Three CPhI experts from BioPlan AssociatesVicky Qing XIA, Leo Cai Yang and Eric Langerexplore the rapidly changing global biologics markets, with special reference to the implications for contract outsourcing and Chinas continued emergence as a hub for both bio innovation and contract services.

Remarkably, China is predicted to continue its rapid bio growth rates, with more than 10 new mAbs predicted to be launched per year in the country by 2025. In fact, the total market size will quadruple by 2025, reaching 120bn RMB, and rising further to 190bn RMB by 2030.

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As most early-stage biotech in China lack manufacturing facilities, the need for contract manufacturing services is rising quickly, and has been accelerated further by the 2016 MAH reforms,commented Vicky XIA, senior project manager at BioPlan Associates.

According to the CPhI report, bioprocessing outsourcing in China is currently highly stratified with four tiers and just one domestic company intieroneWuXi Biologicsand a number of international CDMOs including BI, Lonza, and Merck. However, by 2025 it is anticipated that as many as five more domestic CDMOs may have reached tier one status, with FDA and EU facility approvals. Significantly, pending regulatory changes for contract vaccine production will likely spur further rapid growth amongst the existing tier one CDMOs.

WuXi Biologics in 2019 realized 35.3% of its total revenue of USD 0.57 billion from China, which is ~USD 0.2 billion and would translate to ~35% of the total biopharma outsourcing service market in China1. However, despite sizable growth in revenue, its overall share of the market will now fall, as the mAb market expands rapidly,says XIA.

In terms of global manufacturing, Single Use Systems (SUS) are now the leader at both pre-clinical and clinical stages, with nearly 85% now involving a substantial SUS component. Yet whilst its usage continues to grow, continuous bioprocessing is not anticipated to be in mainstream usage by 2025.

Eric Langer,president and managing partnerof BioPlan Associates says: Because preclinical and clinical pipeline products require flexible manufacturing, SUS lends itself to these scales, but many of these will fail as they progress through the pipeline.This means that while more commercial-scale biologics are going to be made in SUS platforms, or hybrid systems, over the next 2+ years, stainless platforms are, and will remain, critical to bioprocessing as well.

The report also suggested that in the United States and Europe there is likely to be a cell and gene therapy capacity crunch by 2025, with CDMOs investing in this area already expanding to try and meet the pipelines demand. However, capacity alone is not the biggest challenge as there is a shortage of bothspecialized platforms, and trained personnel to operate them. Significantly, for some facilities in these emerging areas the equipment required for expansion and up-scaling may not yet exist, nor are regulatory authorities fully aligned with issues around patient treatmentswhich could slow approvals.

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Tara Dougal, head of content at CPhI Festival of Pharma, commented:This first part of the CPhI Annual Report arrives at a very poignant moment as the CPhI Festival of Pharma launches next month. The cell and gene therapy space is hugely fast-moving and we will run a session on the potential for M&As as both manufacturers and CDMOs try to buy-in expertise rather than build. More generally, our report also points to sustained bio growth in China, almost universal adoption of single use technologies, with continuous processing growing but from a far lower base. In fact, we have invited the Jefferson Institute for Bioprocessing to expand on this theme, as they will explore both current strategies and look ahead at future approaches for bio manufacturing. Its one of the reasons we are so excited about the CPhI Festival of Pharma, as it provides avital platform to meet and exchange ideasat a time when the industry is changing quickly.

To download a copy of the first part of the CPhI Annual Report, please visitGlobal Pharma Insightsor register for early bird discount (ending September 20th) at theCPhI Festival of Pharmathe largest ever digital gathering of professionals with thousands of attendees from dozens of countries. Eric Langer will be part of the cell and gene therapy M&A session (Wednesday 7th, October), with theJefferson Institute for Bioprocessing session on Thursday 15th, October.

CPhI Annual Report expert summaries:

Eric Langer, President and Managing Partner, BioPlan Associates

Industry trends and opportunities in biopharma

Vicky XIAandLeo Yang,BioPlan Associates

Future trends and opportunities in China

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CPhI Annual Report 2020 Part 1: 2025 Will See Cell and Gene Therapy Capacity Shortages in the USA and 10+ mAb Approvals in China - Healthcare...

Silence Therapeutics Appoints Mark Rothera as President and Chief Executive Officer

Experienced biotech executive to lead the next phase of growth

14 September 2020

LONDON, Silence Therapeutics plc, AIM:SLN and Nasdaq: SLN (Silence or the Company), a leader in the discovery, development and delivery of novel short interfering ribonucleic acid (siRNA) therapeutics for the treatment of diseases with significant unmet medical need, today announces the appointment of Mark Rothera as President and Chief Executive Officer (CEO) and Board member, effective immediately. Iain Ross, who has been Executive Chairman since December 2019, has today assumed his previous position of Non-Executive Chairman.

Mr. Rothera brings more than 30 years of experience in the biopharmaceutical industry, with a strong record of commercial and operational leadership, including driving the successful build of multiple biotech companies, predominantly in the field of rare or specialty diseases. Prior to joining Silence, Mr. Rothera served as CEO of Orchard Therapeutics (Orchard), where he oversaw its transformation from a small U.K.-based, privately held company with two clinical-stage programmes into a leading gene therapy company with seven clinical-stage programmes and fully integrated capabilities. Under his leadership, Orchard completed an initial public offering of American Depositary Shares on the Nasdaq Global Market and during his tenure that company secured more than $600 million in financing and grew from a market capitalization of $250 million to more than $1.7 billion at its peak.

Prior to Orchard, Mr. Rothera served as Chief Commercial Officer of PTC Therapeutics (PTC), where he helped transition that company from a privately held R&D biotechnology company to a publicly traded, commercial-stage company with a global footprint, including the successful launch of two rare disease therapies. He also previously served as Global President of Aegerion Pharmaceuticals Inc. and Vice President and General Manager of commercial operations at Shire Human Genetic Therapies for Europe, Middle East and Africa. Mr. Rothera received an M.A. in Natural Sciences from Cambridge University and an M.B.A. from the European Institute for Business Administration (INSEAD).

Based out of Silences New York City office, Mr. Rothera will lead the continued global expansion of the Company. His appointment follows the completion of Silences Nasdaq listing on 8 September 2020 and aligns with the strategy of increasing the Companys presence in the United States.

Iain Ross, Chairman of Silence Therapeutics plc, said: "On behalf of the Silence Board and the entire Silence team, I welcome Mark to the Company. Following a thorough search, Marks appointment reflects his proven leadership skills and strong track record in growing successful biotechnology companies and building shareholder value. I believe he will now provide the leadership necessary to grow Silence into a leading international biotechnology company built upon our innovative siRNA technology platform, proprietary product pipeline and validating industry partnerships.

On a personal note, and on behalf of the Board, I would like to thank the management team and staff at Silence for their support, hard work and tremendous resilience during the current COVID-19 pandemic and over the past nine months whilst I have been Executive Chairman. The Company has made great strides during this period, and is now in a strong position, both operationally and financially, and ready for Mark to take the helm.

Mark Rothera, President and CEO of Silence Therapeutics plc, added: It is an honour to take the role of leading Silence at this time in the Companys history. I believe the Company is poised to capitalise on its important siRNA technology platform, pipeline and research capabilities built over 18 years, and position itself as a leader in the RNAi field. The Company has made great strides under Iains leadership and I look forward to working with the Board, the management team and Silence employees to build upon this momentum.

Director disclosures

The following information is being disclosed pursuantto Rule 17 and paragraph (g) of Schedule 2 of the AIM Rules for Companies.

Mark Rothera

Full name and age: Mark Andrew Rothera (aged 58)

Current Directorships or Partnerships:Genpharm

Previous Directorships or Partnerships in the last 5 years:Orchard Therapeutics plcPTC Therapeutics International LimitedAlliance for Regenerative Medicine

No further information in connection with his appointment is required to be disclosed under Schedule Two, paragraph (g) of the AIM Rules for Companies.

Enquiries:

About Silence TherapeuticsSilence Therapeutics is developing a new generation of medicines by harnessing the bodys natural mechanism of RNA interference, or RNAi, to inhibit the expression of specific target genes thought to play a role in the pathology of diseases with significant unmet medical need. Silences proprietary technology can be used to engineer short interfering ribonucleic acids (siRNAs) that bind specifically to and silence, through the RNAi pathway, almost any gene in the human genome to which siRNA can be delivered. Silences wholly owned product candidates include SLN360 designed to address the high and prevalent unmet medical need in reducing cardiovascular risk in people born with high levels of Lipoprotein(a) and SLN124 to address beta-thalassemia and myelodysplastic syndrome. Silence is also developing SLN500 in partnership with Mallinckrodt Pharmaceuticals to reduce the expression of the C3 protein for the treatment of complement pathway-mediated diseases. Silence maintains ongoing research and collaborations with AstraZeneca, Mallinckrodt Pharmaceuticals and Takeda. For more information, please visit: https://www.silence-therapeutics.com/

The person who arranged for the release of this announcement on behalf of the Company was Rob Quinn, Chief Financial Officer.

Forward-Looking StatementsCertain statements made in this announcement are forward-looking statements, including with respect to the Companys clinical and commercial prospects. These forward-looking statements are not historical facts but rather are based on the Company's current expectations, estimates, and projections about its industry; its beliefs; and assumptions. Words such as 'anticipates,' 'expects,' 'intends,' 'plans,' 'believes,' 'seeks,' 'estimates,' and similar expressions are intended to identify forward-looking statements. These statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties, and other factors, some of which are beyond the Company's control, are difficult to predict, and could cause actual results to differ materially from those expressed or forecasted in the forward-looking statements. The Company cautions security holders and prospective security holders not to place undue reliance on these forward-looking statements, which reflect the view of the Company only as of the date of this announcement. The forward-looking statements made in this announcement relate only to events as of the date on which the statements are made. The Company will not undertake any obligation to release publicly any revisions or updates to these forward-looking statements to reflect events, circumstances, or unanticipated events occurring after the date of this announcement except as required by law or by any appropriate regulatory authority.

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Silence Therapeutics Appoints Mark Rothera as President and Chief Executive Officer - GlobeNewswire

SAN DIEGO, Sept.15, 2020 /PRNewswire/ -- Cardiff Oncology, Inc. (Nasdaq:CRDF), a clinical-stage oncology therapeutics company developing drugs to treat cancers with the greatest medical need for new treatment options, today announced that it will host a key opinion leader (KOL) call focused on KRAS-mutated colorectal cancer and highlighting data from its onvansertib Phase 1b/2 clinical trial, on Wednesday, Sept. 23, 2020 from 11 a.m. 12:30 p.m. EDT.

On the call, Dr. Mark Erlander, Cardiff Oncology's chief executive officer, and Key Opinion Leaders Afsaneh Barzi, M.D., Ph.D. (City of Hope Comprehensive Cancer Center) and Heinz-Josef Lenz, M.D., FACP (USC Norris Comprehensive Cancer Center) will participate in a discussion featuring the latest data from Cardiff's Phase 1b/2 clinical trial evaluating onvansertib in combination with FOLFIRI and bevacizumab for the second line treatment of patients with KRAS-mutated metastatic colorectal cancer. Dr. Barzi will begin the discussion by providing an overview of the history of KRAS in clinical practice, the challenges of drug development and targeting of KRAS, and the value of KRAS as a biomarker for patient selection and predicting response to treatment. Dr. Lenz will follow with a presentation of the onvansertib clinical trial data featured at the European Society of Medical Oncology (ESMO) Virtual Conference 2020. A question and answer session will follow the formal presentations.

You may register for the call by clicking here.

About the KOLs

Afsaneh Barzi, M.D., Ph.D.is a practicing medical oncologist, associate clinical professor for gastrointestinal oncology, and clinical director of AccessHope at City of Hope Comprehensive Cancer Center. Prior to joining City of Hope, Dr. Barzi was an associate professor of clinical medicine at the Keck School of Medicine of the University of Southern California. She earned her M.D. from Tehran University of Medical Sciences, then went on to earn a Master's in Health Informatics and a Doctorate in Public Health Management and Policy Sciences from the University of Texas Health Science Center in Houston. Dr. Barzi completed a fellowship in hematology and oncology at the Cleveland Clinic's Taussig Cancer Center. Her research and practice are focused on gastrointestinal malignancies with an emphasis on colorectal cancers. Her unique perspective on patterns of care in patients with colorectal cancer arises from the combination of her expertise in real-world data and her experience with biomarker discovery and the use of biomarkers for personalized care.

Heinz-Josef Lenz, M.D., FACPis the associate director for clinical research and co-leader of the gastrointestinal (GI) cancers program at the University of Southern California Norris Comprehensive Cancer Center. Dr. Lenz is professor of medicine and preventive medicine, section head of gastrointestinal oncology in the division of medical oncology and co-director of the Colorectal Center at the Keck School of Medicine of the University of Southern California. Dr. Lenz received his medical degree from Johannes-Gutenberg Universitt in Mainz, Germany, in 1985. He completed a residency in hematology and oncology at the University Hospital Tbingen in Germany, a clerkship in oncology at George Washington University in Washington, DC, and a clerkship in hematology at Beth Israel Hospital of Harvard Medical School in Boston, Massachusetts. He served subsequent fellowships in biochemistry and molecular biology at the University of Southern California Norris Comprehensive Cancer Center. An active researcher, Dr. Lenz focuses on topics including the regulation of gene expression involved in drug resistance, patients at high risk of developing colorectal cancer, and determination of carcinogenesis, methods of early detection, and better surveillance of these cancers. He is a member of several professional societies, including the American Association for Cancer Research, the American Gastroenterology Association, and the National Society of Genetic Counselors. He also serves on the National Advisory Board of a number of professional organizations. Dr. Lenz is the author of numerous peer-reviewed publications and invited papers, reviews, and editorials. He also serves as co-chair of the GI Committee and Correlative Science Committee for SWOG. He is a member of the National Cancer Institute (NCI) Task Force for Gastroesophageal Cancer, the NCI Steering Committee, and the NCI Translational Science Committee.

About Cardiff Oncology, Inc.

Cardiff Oncology (formerly Trovagene, Inc.) is a clinical-stage biotechnology company with the singular mission of developing new treatment options for cancer patients in indications with the greatest medical need. Our goal is to overcome resistance, improve response to treatment and increase overall survival. We are developing onvansertib, a first-in-class, third-generation Polo-like Kinase 1 (PLK1) inhibitor, in combination with standard-of-care chemotherapy and targeted therapeutics. Our clinical development programs incorporate tumor genomics and biomarker technology to enable assessment of patient response to treatment.We have three ongoing clinical programs that are demonstrating the safety and efficacy of onvansertib: a Phase 1b/2 study of onvansertib in combination with FOLFIRI/Avastin in KRAS-mutated metastatic colorectal cancer (mCRC); a Phase 2 study of onvansertib in combination with Zytiga (abiraterone)/prednisone in metastatic castration-resistant prostate cancer (mCRPC); and a Phase 2 study of onvansertib in combination with decitabine in relapsed or refractory acute myeloid leukemia (AML). For more information, please visit https://www.cardiffoncology.com.

Forward-Looking Statements

Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of words such as "anticipate," "believe," "forecast," "estimated" and "intend" or other similar terms or expressions that concern Cardiff Oncology's expectations, strategy, plans or intentions. These forward-looking statements are based on Cardiff Oncology's current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, our need for additional financing; our ability to continue as a going concern; clinical trials involve a lengthy and expensive process with an uncertain outcome, and results of earlier studies and trials may not be predictive of future trial results; our clinical trials may be suspended or discontinued due to unexpected side effects or other safety risks that could preclude approval of our product candidates; risks related to business interruptions, including the outbreak of COVID-19 coronavirus, which could seriously harm our financial condition and increase our costs and expenses; uncertainties of government or third party payer reimbursement; dependence on key personnel; limited experience in marketing and sales; substantial competition; uncertainties of patent protection and litigation; dependence upon third parties; our ability to develop tests, kits and systems and the success of those products; regulatory, financial and business risks related to our international expansion and risks related to failure to obtain FDA clearances or approvals and noncompliance with FDA regulations. There are no guarantees that any of our technology or products will be utilized or prove to be commercially successful. Additionally, there are no guarantees that future clinical trials will be completed or successful or that any precision medicine therapeutics will receive regulatory approval for any indication or prove to be commercially successful. Investors should read the risk factors set forth in Cardiff Oncology's Form 10-K for the year ended December 31, 2019, and other periodic reports filed with the Securities and Exchange Commission. While the list of factors presented here is considered representative, no such list should be considered to be a complete statement of all potential risks and uncertainties. Unlisted factors may present significant additional obstacles to the realization of forward-looking statements. Forward-looking statements included herein are made as of the date hereof, and Cardiff Oncology does not undertake any obligation to update publicly such statements to reflect subsequent events or circumstances.

Cardiff Oncology Contact:

Vicki KelemenChief Operating Officer858-952-7652[emailprotected]

Investor Contact:

Joyce AllaireLifeSci Advisors212-915-2569[emailprotected]

Media Contact:Karen O'Shea, Ph.D.LifeSci Communications929-469-3860[emailprotected]

SOURCE Cardiff Oncology, Inc.

Cardiff Oncology

Continued here:
Cardiff Oncology to Host Key Opinion Leader Call Discussing KRAS-Mutated Colorectal Cancer and Highlighting Data from Onvansertib Phase 1b/2 Trial -...

Following is a summary of current science news briefs.

Chinese scientists develop gene therapy which could delay ageing

Scientists in Beijing have developed a new gene therapy which can reverse some of the effects of ageing in mice and extend their lifespans, findings which may one day contribute to similar treatment for humans. The method, detailed in a paper in the Science Translational Medicine journal earlier this month, involves inactivating a gene called kat7 which the scientists found to be a key contributor to cellular ageing.

China's retrieved lunar samples weigh less than targeted

Lunar rocks retrieved by a historic Chinese mission to the moon weighed less than initially targeted, but China is still willing to study the samples with foreign scientists, the mission's spokesman said on Monday. China became the third country ever to secure lunar samples when its unmanned Chang'e-5 probe, named after the mythical moon goddess, brought back 1.731 kg (3.8 lb) of samples last month, falling short of the 2 kg (4.4 lb) planned.

Branson's Virgin Orbit reaches space with key mid-air rocket launch

Billionaire Richard Branson's Virgin Orbit reached space for the first time on Sunday with a successful test of its air-launched rocket, delivering ten NASA satellites to orbit and achieving a key milestone after aborting the rocket's first test launch last year. The Long Beach, California-based company's LauncherOne rocket was dropped mid-air from the underside of a modified Boeing 747 nicknamed Cosmic Girl some 35,000 feet over the Pacific at 11:39 a.m. PT before lighting its NewtonThree engine to boost itself out of Earth's atmosphere, demonstrating its first successful trek to space.

(With inputs from agencies.)

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Science News Roundup: Chinese scientists develop gene therapy which could delay ageing; China's retrieved lunar samples weigh less than targeted and...

BOSTON, Jan. 19, 2021 /PRNewswire/ --The VHL Alliance (VHLA) today announced the 2021 recipients of the VHLA Competitive Research Grant Program. This year, three grantees were chosen. Scientists are increasingly aware that curing von Hippel-Lindau disease, a genetic form of cancer is a key step to curing cancer, leading to a significant increase in the quality of research grant applicants in the program year after year.

Over 20 top VHLA researchers from across the US, Canada, and Europe were involved in reviewing and selecting those projects which have the greatest potential to make advances toward Curing Cancer through VHL. For the second year in a row, responding to the increase in quality applications, the VHLA Board of Directors voted to expand the annual budget for research.

This year's grant recipients were:

Dr. Tirosh proposes to investigate the microenvironment of pancreatic neuroendocrine cancers using computational biology. Dr. Jonasch will test in the laboratory whether VHL-associated kidney cancers respond to a new class of medication that exploits DNA stress. Finally, Dr. Zagzag will test whether a change in a specific protein expressed in hemangioblastomas can determine their growth rate.

"The VHL Alliance is proud to fund basic scientific and translational research of the highest quality, poised to shed light into the mechanisms of VHL tumors and lead to discovery of new ways to prevent and treat VHL patient tumors,"said Othon Iliopoulos, MD, PHD, board member and chair of the VHLA Research Council, and Clinical Director, Von-Hippel Lindau Disease/Familial Renal Cell Cancer Program, Associate Professor of Medicine, Center for Cancer Research, Massachusetts General Hospital, Boston, MA.

"Once again, the VHL Alliance Research Council was impressed with the range and quality of grant applications received. We have seen a growing interest among researchers in solving the mystery of the VHL gene - an important key to unlocking the mechanisms of tumor growth and ultimately, cancer," said Chandra Clark, Executive Director of the VHL Alliance. "VHL disease affects 1 in 36,000 people around the world regardless of gender, race, socioeconomic, or geographic circumstances, and finding a cure will benefit thousands of cancer patients."

For more information about the VHL Alliance or the 2021 VHLA Competitive Research Grant Program, please visit vhl.org.

About VHLVHL or von Hippel-Lindau disease is a genetic form of cancer. VHL patients battle a series of tumors throughout their lives. The VHL gene controls the major feeding pipeline of every tumor. Curing VHL is one step closer to curing many other forms of cancer. There are currently 8 drugs being used to effectively treat cancer, mainly kidney cancers, which are direct results of VHL research.

About the VHL AllianceThe VHL Alliance (VHLA) is the preeminent resource and clearinghouse for those affected by von Hippel-Lindau disease, including patients, caregivers, researchers, and the medical community. VHLA is a 501(c)(3) non-profit organization founded in 1993, which is dedicated to research, education, and support to improve awareness, diagnosis, treatment, and quality of life for those affected by VHL. VHLA is the leading funder of VHL research, funding over $2.5 million in grants to support studies designed to find a cure. The VHL Alliance's vision is Curing Cancer through VHL.

About the VHLA Competitive Research Grant ProgramThe VHLA's Competitive Research Grant program awards two types of Grants: Pilot Grants ($25,000 for one year), designed to help researchers prepare the basic modelling required to then pursue larger grants; and Research Grants ($150,000 over three years),designed to obtain sufficient data to apply for government funded resources. Previous grants have contributed to our understanding of how the VHLgene contributes to the tumorigenesis including how to overcome the body signaling and response to what it perceives as a hypoxic (low oxygen) environment.

Contact: Heidi Leone, Director of AdvancementVHL Alliance617.277.5667 x 4[emailprotected]

SOURCE VHL Alliance

Excerpt from:
2021 Research Grant Recipients Announced by the VHL Alliance - PRNewswire

Bone Therapeutics, a cell therapy company addressing unmet medical needs in orthopedics and other diseases, and Rigenerand SRL, a biotech company that both develops and manufactures medicinal products for cell therapy applications, primarily for regenerative medicine and oncology, have signed an agreement for a process development partnership.Allogeneic mesenchymal stem cell (MSC) therapies are currently being developed at a fast pace and are evaluated in numerous clinical studies covering diverse therapeutic areas such as bone and cartilage conditions, liver, cardiovascular and autoimmune diseases in which MSCs could have a significant positive effect.Advances in process development to scale up these therapies could have major impacts for both their approval and commercial viability. This will be essential to bring these therapies to market to benefit patients as quickly as possible, said Miguel Forte, chief executive officer, Bone Therapeutics. While Bone Therapeutics is driving on its existing clinical development programs, we have signed a first formal agreement with Rigenerand as a fellow MSC-based organization. This will result in both companies sharing extensive expertise in the process development and manufacturing of MSCs and cell and gene therapy medicinal products. Bone Therapeutics also selected Rigenerand to partner with for their additional experience with wider process development of advanced therapy medicinal products (ATMPs), including the conditioning and editing of MSCs.The scope of collaborations between Bone Therapeutics and Rigenerand aims to focus on different aspects of product and process development for Bone Therapeutics expanding therapeutic portfolio. Rigenerand will contribute to improving the processes involved in the development and manufacture of Bone Therapeutics MSC based allogeneic differentiated cell therapy products as they advance towards patients. The first collaboration between the two organizations will initially focus on augmented professional bone-forming cellscells that are differentiated and programmed for a specific task. There is also potential for Bone Therapeutics to broaden its therapeutic targets and explore new mechanisms of action with potential gene modifications for its therapeutic portfolio.In addition to Rigenerands MSC expertise, Bone Therapeutics also selected Rigenerand as a partner for Rigenerands GMP manufacturing facility. This facility, situated in Modena, Italy, has been designed to host a number of types of development processes for ATMPs. These include somatic, tissue engineered and gene therapy processes. These multiple areas of Rigenerand capabilities enable critical development of new processes and implementation of the gene modification of existing processes. In addition, Rigenerand has built considerable experience in cGMP manufacturing of MSC-based medicinal products, including those that are genetically modified.Process development and manufacturing is a key part of the development for ATMPs internationally. Navigating these therapies through the clinical development phase and into the market requires a carefully considered process development pathway, said Massimo Dominici, scientific founder, Rigenerand, professor of medical oncology, and former president of the International Society for Cell & Gene Therapy (ISCT). This pathway needs to be flexible, as both the market and materials of these therapies continues to evolve alongside an improved clinical efficacy.Giorgio Mari, chief executive officer, Rigenerand, said, Rigenerand will offer considerable input from its experience of MSC-based therapies to enable Bone Therapeutics to keep and further accelerate the pace in development of the product processes of its MSC based allogeneic differentiated cell therapy as they advance towards patients. We will continue to use our MSC expertise in the development of Rigenerands own products, as well as in process development and manufacturing cell and gene therapies for partner organizations across the globe.

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Bone Therapeutics, Rigenerand Ink Cell Therapy Deal - Contract Pharma

In the dark days of World War II, a 12-year-old boy dodged the Japanese soldiers who occupied his neighborhood in Hong Kong, slipping around corners and into alleys to sell cans of kerosene hed converted from diesel oil to support his widowed mother.

When the occupation was over, he learned English in night school and rented a typewriter so he could make money exporting cheap toys to the West. Eventually, his import-export venture did well enough that he could afford to buy the typewriter outright.

Six decades later, his son, internationally recognized oncologist and investigator Tony S.K. Mok, MD, keeps that same typewriter in his office. Mok calls it his daily reminder to follow his fathers example of entrepreneurship and diligence.

Moks groundbreaking research into treatment options for lung cancer subtypes that are driven by genetic mutations is credited with producing a paradigm shift in fighting the worlds deadliest malignancy.

One look at his resume hints at a curiosity that compels him to seek out experiences not in the average oncologists day: Hes not just a world-renowned cancer investigator and clinician, but hes also the host of several seasons of televised food shows, and hes written a news-paper column for the past 14 years.

His face is so familiar in Hong Kong that an elderly patient, meeting him for the first time, assumed he was just an actor. Are you a real doctor? she asked skeptically, Mok, 60, recounted with laughter. How does he make time for it all?

When an opportunity comes along, 2 things come to my mind: Am I going to like it? And am I going to make a positive contribution to the community? If the answer to both questions is Yes, I find the time, he said.

He was born Shu Kam Mok. When his parents enrolled him in an English-speaking secondary school, the school insisted he take a Christian name. A tutor picked Tony and the name continued to make sense for the next chapter of his life, when his parents sent him to college in Canada at age 16. He received his fellowship training in medical oncology at Princess Margaret Cancer Centre in Toronto, Ontario, having chosen oncology because it seemed to be a field on the cusp of major new developments.

In 1995, he took a tour of the Prince of Wales Hospital, the main teaching hospital of the Chinese University of Hong Kong. At the end of the tour, the head of oncology surprised him with a job offer.

Hong Kong was starting to experience a brain drain of physicians worried about the impending transfer of the British colony to China. Moks credentials as a seasoned oncologist who spoke Chinese made him an attractive candidate, despite his lack of research experience.

Accepting the offer would mean giving up his comfortable life in Canada and taking a big financial hit. On the other hand, hed get to work with Chinese researchers and he was enchanted by the charm of uncertainty. Staying in Toronto meant his life would follow the very predictable path of a suburban physician.

He took the plunge and accepted the offer. He was 36, older than most of his peers, he didnt have a single publication to his name.

Mok noticed that few investigators in Asia were looking lung cancer. The lethality of the disease was discouraging, and its mysteries seemed impenetrable. Where others saw a brick wall, however, Mok saw opportunity.

One of the reasons to go into a field where little is known is because that is exactly the field where youll learn more and create more, he said. I thought, This is a great area where there is going to be great progress.

His early research tackled a grab bag of topics, including pancreatic, colon, and lung cancers, as he tried to catch up with his colleagues. Much of his research has focused on the role of the EGFR gene mutation in nonsmall cell lung cancer (NSCLC). Mok was the lead researcher for the pivotal phase 3 IPASS trial. Investigators randomly assigned 1217 previously untreated patients with advanced lung cancer in East Asia to gefitinib (Iressa) or carboplatin plus paclitaxel.

For patients with the EGFR gene mutation, gefitinib was clearly superior to standard chemotherapy; for patients without the mutation, chemotherapy was superior.

The data, published in the New England Journal of Medicine in September 2009, remade the world of lung cancer treatment and remains 1 of the most cited publications in the field. Based on that data, physicians would test patients with newly diagnosed lung cancer for genetic mutations before choosing a treatment. Lung cancer, so slow to relinquish its secrets, had entered the age of personalized medicine.

While Mok is accessible and friendly by natureand funny, as wellcolleagues say that beneath that convivial exterior is a serious intellect. His presentations at professional conferences are known for delivering not just the latest developments in research, but for put-ting that research into a broader context that every attendee can grasp, even those outside the immediate field of lung cancer.

Hes also a wine connoisseur and foodie who makes it a point to know the best restaurants in every major city. At the American Society of Clinical Oncology (ASCO) Annual Meeting, Mok traditionally arranges a big dinner with friends where shop talk is discouraged.

All his activitiesthe university teaching, the columns in the Hong Kong Economic Times, his work in the clinic, and his researchmake for a very full plate. He wouldnt have it any other way, explaining that being busy fits into his philosophy of life.

The way I see life, the way I define a happy life, is this: At the end of the day, how many happy moments have I collected? So if I collect enough moments, I can call this a happy life, Mok said.

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Embracing the Charm of Uncertainty in Lung Cancer Research - OncLive

Every drug, from morphine to ibuprofen, has a standard dose -- a sort of one-size-fits all recommendation. But a new study suggests that when it comes to drug doses, "one size fits all" rarely applies.

Stanford Medicine professor Russ Altman, MD, PhD, and a team of scientists found that almost everyone (99.5% of individuals) is likely to have an abnormal or "atypical" response to at least one therapeutic drug. This, at least, is the case for people in the United Kingdom, as the study's data came from the UK Biobank, a project that collects, studies and shares data.

The research found that nearly a quarter of the study's participants had been prescribed a drug for which they were predicted to have an atypical response, based on their genetic makeup. On average, participants were predicted to have an atypical response to 10 drugs.

"Ultimately, the hope is that we can show how pervasive drug response variability is and encourage more doctors to rethink the standard prescription protocols that are largely used today and use genetic testing to predict and adjust forthis variability," said Altman, who is an expert in pharmacogenetics, a field that studies the intersection of drugs and genetics.

An "atypical" drug response encompasses a lot of things; but generally speaking, it means a certain drug might not affect one person the way it does another.

For instance, someone who has an atypical metabolic response might process that drug more efficiently, strengthening its initial effects but decreasing its efficacy over time. On the flip side, it could mean that that person is unable to metabolize the drug at all, leaving them without therapeutic aid, or even with dangerous side effects.

These differences in response to a drug are partially due to our genetics. Specific proteins -- workhorse molecules in the body -- break down drugs in order for the body to benefit from the therapeutic. Those proteins are regulated by a specific group of genes. Natural variation in those genes leads to differences in how an individual's body reacts to a given drug molecule.

Altman and his team, including graduate students and first authors of the study Greg McInnes and Adam Lavertu, analyzed data from nearly 500,000 participants.

For 230,000 participants in the study, the team had primary care data going back about 30 years. That includes which drugs had been prescribed, the dose, and all of the patient's different diagnoses. The researchers also had access to detailed genetic information about each patient. They paid special attention to genetic variations in a group of genes that are known to influence the human drug response.

By comparing an individual's genetics against the variations known to exist in the group of drug-response-associated genes, the researchers could predict how any given patient might respond to a drug.

"Pharmacogenetics as a field has been around for a long time, but it hasn't really been adopted into clinical use," McInnes told me. "It's been growing in the last few years as more people realize the impact that it could have on personalized health. For a long time, it's been this overlooked aspect of genetics that I think is actually one of the most clinically actionable advances that has come out of human genetics."

What's more, he said, the wide variability in the human drug response applies to common therapeutics most everyone has encountered or is familiar with -- ibuprofen, codeine, statins and beta blockers among others.

Moving forward, Lavertu says that the goal is to expand drug-gene variant interaction analyses into more diverse populations. The data from the UK Biobank provided critical insight, but it was largely only representative of a British population, where the majority shares European ancestry. A next step for the researchers is to investigate the same genes in the Million Veteran Program, a government research program with a more diverse study population, that is examining how genes, lifestyle and military exposures affect health and illness.

"Our hope is that doing more of these studies will help us find new relationships between genetic variants and drug response, so that pharmacogenetics can benefit more people," Lavertu said.

Photo byMicha Parzuchowski

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Based on genes, nearly everyone is likely to have an atypical response to at least one drug - Scope

Cancer immune plays a critical role in cancer progression. Tumour immunology and immunotherapy are one of the exciting areas in bladder cancer research. In this study, we aimed to develop an immune-related gene signature to improve the prognostic prediction of bladder cancer. Firstly, we identified 392 differentially expressed immune-related genes (IRGs) based on TCGA and ImmPort databases. Functional enrichment analysis revealed that these genes were enriched in inflammatory and immune-related pathways, including in 'regulation of signaling receptor activity', 'cytokine-cytokine receptor interaction' and 'GPCR ligand binding'. Then, we separated all samples in TCGA data set into the training cohort and the testing cohort in a ratio of 3:1 randomly. Data set GSE13507 was set as the validation cohort. We constructed a prognostic six-IRG signature with LASSO Cox regression in the training cohort, including AHNAK, OAS1, APOBEC3H, SCG2, CTSE and KIR2DS4. Six IRGs reflected the microenvironment of bladder cancer, especially immune cell infiltration. The prognostic value of six-IRG signature was further validated in the testing cohort and the validation cohort. The results of multivariable Cox regression and subgroup analysis revealed that six-IRG signature was a clinically independent prognostic factor for bladder cancer patients. Further, we constructed a nomogram based on six-IRG signature and other clinicopathological risk factors, and it performed well in predict patients' survival. Finally, we found six-IRG signature showed significant difference in different molecular subtypes of bladder cancer. In conclusions, our research provided a novel immune-related gene signature to estimate prognosis for patients' survival with bladder cancer.

Journal of cellular and molecular medicine. 2020 Oct 13 [Epub ahead of print]

Yongwen Luo, Liang Chen, Qiang Zhou, Yaoyi Xiong, Gang Wang, Xuefeng Liu, Yu Xiao, Lingao Ju, Xinghua Wang

Department of Urology, Zhongnan Hospital of Wuhan University, Wuhan, China., Department of Biological Repositories, Zhongnan Hospital of Wuhan University, Wuhan, China., Department of Pathology, Lombardi Comprehensive Cancer Center, Georgetown University Medical School, Washington, DC, USA.

PubMed http://www.ncbi.nlm.nih.gov/pubmed/33048468

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Identification of a prognostic gene signature based on an immunogenomic landscape analysis of bladder cancer. - UroToday

PHILADELPHIA, Oct. 13, 2020 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today announced publication of data in a murine model of GM1 gangliosidosis (GM1) demonstrating that a single intracerebroventricular injection of an optimized adeno-associated virus (AAV) into the cerebral spinal fluid (CSF) resulted in significant expression of Beta-galactosidase (-gal) in the brain and peripheral tissues, and demonstrated dose-related reductions in neuronal lysosomal storage lesions, neurological impairment and improvement in survival. These data were published online ahead of print in the November issue of the peer-reviewed scientific journal Human Gene Therapy (HGT).

This study suggests that delivery of an AAV vector optimized to express b-gal directly into the CSF restored b-gal activity in the brain and, if further developed and tested in human clinical trials, may be effective in modifying and preventing the devastating effects of the genetic disease GM1, said James Wilson, M.D., Ph.D., director of the Gene Therapy Program at the University of Pennsylvania (Penn) and chief scientific advisor of Passage Bio. The AAV vector used in the study is the same as Passage Bios PBGM01 gene therapy, which is designed to deliver a functional human GLB1 gene into the brain and optimized to express -gal. These preclinical study data support the further development of PBGM01 as a potential therapy for patients suffering from GM1.

GM1 is a rare and often life-threatening monogenic lysosomal storage disease caused by mutations in the GLB1 gene, which encodes lysosomal acid -gal. Reduced -gal activity results in the accumulation of toxic levels of GM1 in neurons throughout the brain, causing rapidly progressing neurodegeneration. GM1 manifests as a continuum of disease and is most severe in the infantile form, which is characterized by onset in the first six months of life with hypotonia (reduced muscle tone), progressive CNS dysfunction, and rapid developmental regression. Life expectancy for infants with GM1 is two to four years, and infantile GM1 represents approximately 60 percent of the incidence of 0.5 to 1 in 100,000 live births. Currently, there are no approved disease-modifying therapies available.

Results of the PBGM01 preclinical study were reported in the paper titled, A single injection of an optimized AAV vector into cerebrospinal fluid corrects neurological disease in a murine model of GM1 gangliosidosis, by Christian Hinderer, M.D., Ph.D., and colleagues, including gene transfer pioneer Dr. Wilson, from the Gene therapy Program, Department of Medicine, University of Pennsylvania Perlman School of Medicine. The study in part was previously presented at the 22nd annual Meeting of the American Society for Cell and Gene Therapy (ASCGT) in 2019.

This research evaluated the impact of single intracerebroventricular administration of the human -gal containing AAV vector on -galactosidase enzyme activity in the murine brain and peripheral tissues, lysosomal storage lesions, neurological function (including neurological exams and gait analysis) and survival in mice lacking the -galactosidase gene. The mice received the single administration at age one month and were evaluated over 300 days. -gal activity was increased significantly in the cerebral spinal fluid and serum of the vector-treated mice compared to vehicle control-treated mice. Significant improvements in gait assessments as measured by stride length and hind paw print length and significant preservation of neurological function as measured by neurological exam scores were observed throughout the study period in the human -gal vector-treated mice. There were significant decreases in lysosomal storage lesions of vector-treated animals and by day 300 all animals that received the two highest doses were still alive, whereas none of the vehicle control-treated animals had survived.

Were excited about being able to soon advance PBGM01 into the clinic, and the potential promise it holds for patients with GM1, the majority of whom are infants and for whom there are no approved disease modifying treatments, said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio. Our plan is to administer PBGM01 through intra-cisterna magna delivery into the brain, which we believe may offer several benefits in terms of safety, efficiency and distribution compared to other approaches.

Passage Bio expects to initiate dosing of PBGM01 in a Phase 1/2 trial late in the fourth quarter of 2020 or early in the first quarter of 2021 and remains on track to report initial 30-day safety and biomarker data late in the first half of 2021.

This research was supported by a research, collaboration and license agreement with Passage Bio. HGT is the Official Journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies. Click here to read the full-text article on the HGT website.

About PBGM01PBGM01 is an AAV-delivery gene therapy currently being developed for the treatment of infantile GM1, in which patients have mutations in the GLB1 gene causing little or no residual -gal enzyme activity and subsequent neurodegeneration. PBGM01 utilizes a next-generation AAVhu68 capsid administered through intra-cisterna magna (ICM) to deliver a functional GLB1 gene encoding -gal to the brain and peripheral tissues. By reducing the accumulation of GM1 gangliosides, PBGM01 has the potential to halt or prevent neuronal toxicity, thereby restoring developmental potential. In preclinical models, PBGM01 has demonstrated broad brain distribution and wide uptake of the -gal enzyme in both the central nervous system (CNS) and critical peripheral organs, suggesting potential treatment for both the CNS and peripheral manifestations of GM1. The Company has received Orphan Drug and Rare Pediatric Disease designation for PBGM01 for patients with GM1 and expects to initiate dosing of its Phase 1/2 trial late in the fourth quarter of 2020 or early in the first quarter of 2021 and remains on track to report initial 30-day safety and biomarker data late in the first half of 2021.

About Passage BioPassage Bio is a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders with limited or no approved treatment options. The company is based in Philadelphia, PA and has a research, collaboration and license agreement with the University of Pennsylvania and its Gene Therapy Program (GTP). The GTP conducts discovery and IND-enabling preclinical work and Passage Bio conducts all clinical development, regulatory strategy and commercialization activities under the agreement. The company has a development portfolio of six product candidates, with the option to license eleven more, with lead programs in GM1 gangliosidosis, frontotemporal dementia and Krabbe disease.

University of Pennsylvania (Penn)Financial DisclosureDr. Wilson is a Penn faculty member and also a scientific collaborator, consultant and co-founder of Passage Bio. As such, he holds an equity stake in the company, receives sponsored research funding from Passage Bio, and as an inventor of certain Penn intellectual property that is licensed to Passage Bio, he may receive additional financial benefits under the license in the future. He is an inventor of intellectual property covering the technology described in paper published in HGT that is licensed from Penn to Passage Bio, and he may receive financial benefits under this license in the future. Penn also holds equity and licensing interests in Passage Bio.

Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about timing and execution of anticipated milestones, including our planned IND submissions, initiation of clinical trials and the availability of clinical data from such trials; our expectations about our collaborators and partners ability to execute key initiatives; our expectations about manufacturing plans and strategies; our expectations about cash runway; and the ability of our lead product candidates to treat the underlying causes of their respective target monogenic CNS disorders. These forward-looking statements may be accompanied by such words as aim, anticipate, believe, could, estimate, expect, forecast, goal, intend, may, might, plan, potential, possible, will, would, and other words and terms of similar meaning. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop and obtain regulatory approval for our product candidates; the timing and results of preclinical studies and clinical trials;; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; the risk that positive results in a preclinical study or clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; failure to protect and enforce our intellectual property, and other proprietary rights; our dependence on collaborators and other third parties for the development and manufacture of product candidates and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; and the other risks and uncertainties that are described in the Risk Factors section in documents the company files from time to time with theSecurities and Exchange Commission(SEC), and other reports as filed with theSEC. Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

For further information, please contact:

Investors:Sarah McCabe and Zofia MitaStern Investor Relations, Inc.212-362-1200sarah.mccabe@sternir.comzofia.mita@sternir.com

Media:Gwen FisherPassage Bio215.407.1548gfisher@passagebio.com

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Passage Bio Announces Publication of Preclinical Data That Show Single Injection of Optimized AAV Vector into Cerebral Spinal Fluid - BioSpace