Three patients who received SPK-8011 a gene therapy developed by Spark Therapeutics for hemophilia A show increased production of clotting factor VIII without any safety issues reported, says the company.

The patients are the first to receive the treatment as part of a Phase 1/2 clinical trial, with early data showing they have not yet experienced spontaneous bleeding episodes.

The encouraging start of our SPK-8011 clinical trial reinforces the strength of our gene therapy platform, delivers human proof-of-concept in a second liver-mediated disease a significant achievement in the gene therapy field and positions us well to potentially transform the current treatment approach for this life-altering disease with a one-time intervention, Katherine A. High, president and CEO of Spark Therapeutics, said inreleasingsecond-quarter 2017 financial results.

The study (NCT03003533) examines escalating doses of the treatment a one time infusion of a non-infectious virus that delivers a functional copy of the gene encoding clotting factor VIII.

The first two patients received the lowest dose and have now been followed for 23 weeks and 12 weeks. Measurements show their levels of the clotting factor steadily rising, reaching a stable level of 11 and 14 percent of normal values. Withthose results in hand, researchers decided to go for a higher dose in the third patient. So far, the increase of factor VIII in that third patient is higher than that observed in the other two, corresponding to the increased dose.

Researchers have not detected immune reactions to the therapy in any of the patients. This is important, as antibodies to SPK-8011 ould potentially render it ineffective.No serious adverse events have been reported and none of the patients required treatment with corticosteroids.

Spark, based in Philadelphia, has also reported on its second gene therapy, SPK-9001, now being developed to treat hemophilia B.

Researchers presented data from the Phase 1/2 study (NCT02484092) at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress, showing that the 10 participants had lowered their annual bleeding rate by 96 percent. They also lowered their annualized infusion rate by 99 percent.

In addition, their levels of clotting factor IX, 12 weeks after the treatment, had stabilized at 33 percent. Four of the patients have been followed for more than a year after the treatment, and one for as long as 18 months.

None have developed immune reactions to the treatment and no bleeding episodes have been recorded. One patient with severe joint disease had precautionary factor infusions for persistent knee pain.

We are excited about the progress we are making to achieve our goals of our investigational hemophilia A and B programs: to safely achieve predictable, consistent and sustained activity levels that prevent spontaneous bleeding, said High.

Read the rest here:

Spark's Gene Therapy SPK-8011 to Treat Hemophilia A and B Advance in Clinical Trials - Hemophilia News Today

Uniqure, the company best known for having the first approved gene therapy put on the market (for familial chylomicronemia syndrome), is expanding its pipeline to include a gene therapy for Huntingtons disease. While still in early development, the company stated they plan to file an Investigational New Drug (IND) application next year with the US Food and Drug Administration (FDA) in order to begin clinical studies in humans. Huntington's disease is a genetic neurodegenerative disorder that leads to loss of muscle coordination, behavioral abnormalities and cognitive decline when a person enters their 3rd or 4th decade. The disease is an autosomal dominant mutation, meaning that if a person has the condition, there is a 50% chance their offspring will have it as well, and is due to a mutation on the huntingin gene. Despite good understanding of the condition, current treatments can only alleviate the symptoms of it, but they do not delay the onset or slow the progression of the disease. It is hopeful that gene therapy can do that. In Huntingtons disease animal models, the gene therapy called AMT-130, an AAV5 vector carrying a DNA cassette encoding artificial micro-RNA, is effective. In April, data was presented at the 12th Annual CHDI HD Therapeutics Conference in Malta showing the drug was able to silence the human mutant huntingtin gene in pig model. In the animal study, AMT-130 was administered into the striatum and thalamus of minipigs that had the mutant Huntingtin gene. Three months after treatment, the vector was observed throughout the minipig brain and expression of mutant HTT mRNA was significantly reduced in the striatum and thalamus by 50% to 80% and reductions were also observed in the cortex (reduced up to 40% compared to controls). Lead author of that study, Prof. Jan Motlik, Director of the Institute of Animal Physiology and Genetics in the Czech Republic said, "This study demonstrated that a single administration of AAV5-miHTT resulted in significant reductions in HTT mRNA in all regions of the brain transduced by AMT-130, as well as in the cortex. Consistent with the reduction in HTT mRNA, we also observed a clear dose-dependent reduction in mutant huntingtin protein levels in the brain, with similar trends in the cerebral spinal fluid. Taking into account the similarities of CHDI's proprietary transgenic pig model to the human brain, these results provide additional data to support moving forward with clinical trials of uniQure's promising gene therapy for Huntington's disease." For more news and information about orphan drugs in development, followRare Disease ReportonFacebookandTwitter.

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Gene Therapy for Huntington's Disease in Development - Rare Disease Report

Investment Thesis

With an undervalued stock, a critically-acclaimed drug leading in its space and a barrage of financial and expertise-based backing, Sangamo Therapeutics (NASDAQ:SGMO) is the ideal vehicle for exposure to a market with high growth potential and a hedge against the antiquated methods (CRISPR/Cas9) of treating hemophilia. The same method has also been found to work to a high degree of accuracy on other genetic disorders.

Sangamo Therapeutics' accomplishments to date have helped to draw integrity to its operations and raise its profile against common competitors in the space (QURE, GSK, ONCE, BMRN, BLUE, BIVV (a pure-play hemophilia company). Principally, Sangamo has secured an exclusivity agreement (and subsequent vote of confidence) from Pfizer (NYSE:PFE) (also once a competitor) regarding the development and commercialization of gene therapy programs for hemophilia A (SB-525), which affects 85% of hemophilia sufferers (Nelson Pediatrics), bagging $70 million upfront and a potential $475 million in milestones and royalties (Reuters). (The Zinc Finger Protein Nuclease technology method adopted is targeted at diseases which are caused by genetic defects, as their removal is known to respond best to gene editing targeting, in particular, tauopathies, thalassemias, hemophilia, and HIV/AIDS).

The company's strong balance sheet was further bolstered by a raise of circa $78.1 million through public offering at a price of $7.25 per share, with a revised analyst share price target estimates from Jefferies LLC of $17 per share (jefferies.com), as a "financial investment hedge against emerging gene-editing technology of CRISPR," the CRISPR/Cas9 system, has seen less successful testing, coupled with scientific criticism for its reported high incidence of nonspecific DNA cleavage, mosaicism (failure to replicate mutant alleles in only some of the cells) and inversely, and overproduction of mutations in a given organism.

SGMO's latest 10-Q (SEC.gov):

In the context of gene therapy, Sangamo ($707.16 million market cap) (capitaliq.com) was the only firm with innovative gene therapy drugs to advance in two clinical trials (namely the SB-728-T program in the Sangamo ZFP Therapeutic Pipeline targeting HIV/AIDS), and the first to conduct genome editing studies in clinical trials, as covered in the Financial Times. The stock's performance has been volatile, and investors were disinclined to back the drug lottery in gene therapy as biotech became heavily saturated in players, regulations and setbacks, stunting the prospect of any tangible advancement.

Sangamo has jumped on the "radar" to become a buy. The FDA has authorized Fast Track designation, the scientific backing is legitimate (and has received industry-wide appraisal), the longer-term targets look plausible and Sangamo's management and consultants are capable of executing given their credentials and two decades of development.

By all measures of valuation, Sangamo is undervalued, and price-action enthusiasts will recognize the surge in trading volumes and the breaking of the 50-day moving average. Early riders would have capitalized on the short-term price depression triggered by the publicizing of the mucopolysaccharidosis Type I (MPS I) and MPS II delay, an overreaction from the market that posed a great value opportunity at the beginning of this month, whose benefits were felt just last week when the stock surged on the impressive FIH results.

Gene therapy (and emicizumab) renders recombinant factor VIII/IX proteins redundant. This a slice of the industry that is represented by $8-10 billion annually (with 90%+ profit margins) shared by Shire (NASDAQ:SHPG), Bayer (OTCPK:BAYRY), Novo Nordisk (NYSE:NVO), Pfizer and Bioverativ (NASDAQ:BIVV), an overvalued stock whose current operations consist entirely of the moribund method of treating hemophilia - a potential sell. It recently acquired True North and thus entering into cold agglutinin disease market, a result of severe hemolytic anemia.

Sangamo now has the backing and leeway to make its drugs commercially viable, magnetizing further interest - 48 institutions have increased their respective positions in SGMO, with Wasatch and BlackRock leading the pack. The smart money and medical experts are backing Sangamo.

Bold tickers indicate potential buys should this thesis play out as predicted.

This is not investment advice, you are advised to carry out your own due diligence.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Read more here:

Sangamo Therapeutics: The Breakthrough In Gene Therapy - Seeking Alpha

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GAINESVILLE, Florida Imagine only being able to see the things in front of you in soft focus, and just in black and white.

For people with the genetic eye condition achromatopsia, those are just some of the side effects.

Each time Tara Cataldo prepares to leave her house, she has to make sure her face is completely shielded from the sun.

"I need to have very dark, very tinted sunglasses to feel comfortable outside, to see really well," said Tara.

Tara has achromatopsia, a genetic condition that makes her eyes incredibly sensitive to light. She is also very nearsighted; even while wearing glasses or contacts, she can only see clearly at a very short distance.

"I cannot drive a car so I rely on public transportation and my bike to get around," she said.

"There are currently no approved or no effective treatments for achromatopsia," said Dr. Christine Kay, a surgical ophthalmologist at the University of Florida.

But she's working to change that.

She is one of a handful of experts testing a gene therapy.

"For achromatopsia the cells we have to target are cone cells responsible for decreased vision and color vision. and those are cells at the very bottom layer of the retina," explained Dr. Kay.

Using a tiny cannula, doctors deliver a normal copy of one of two mutated genes, the CNGA-3 or CNGB-3 gene, directly into the eye to restore vision.

Tara`s myopia is so severe that her risk of retinal detachment from any retinal surgery is high, which rules her out for the current trial.

In the meantime, Tara says she's learned to adapt to achromatopsia and live without limitations.

"And I hope, ya know, all young achromats, ya know, learn the same thing."

Dr Kay says if the gene could eventually be delivered to the surface of the retina, additional patients, like Tara, could be treated. AGTC, the biotech company that developed the therapies, and several U.S. universities have successfully tested this therapy in dogs and sheep.

NEW TECHNOLOGY: A new clinical trial is underway testing gene therapy for achromatopsia. The genes are responsible for releasing proteins essential for the function of all the cells. Researchers remove the virus from the host DNA so it does not have the capacity to make the patient sick, and then insert the gene of interest into the DNA. Surgically done by a vitro retinal surgeon, the gene is then directly delivered to the retinal tissue. Researchers try to avoid directly touching the retina, to avoid detaching it and keep the surgery less evasive. The CNGA3 or CNGB3 gene would help restore the patient`s vision. This can only be done on certain patients; those with extremely severe achromatopsia are not able to participate in this trial because of the risk of retinal rupturing.

If this story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Jim Mertens at jim.mertens@wqad.com or Marjorie Bekaert Thomas at mthomas@ivanhoe.com.

More here:

YOUR HEALTH Gene therapy for better eyesight - WQAD.com

MOSCOW, August 17. /TASS/. A Russian gene therapy drug for individuals infected with HIV called Dinavir is undergoing pre-clinical trials, and the drug has already proved its efficiency on cells. The pre-clinical tests on animal models, clinical trials and the registration procedure may take up to 10 years, senior research fellow at the Epidemiology Central Research Institute of Rospotrebnadzor (the Federal Service on Surveillance for Customers Rights Protection and Human Well-Being) Dina Glazkova told TASS.

"This is not about the next year, but rather in five years, at the earliest. It takes up to 10 years on the average," she said.

Glazkova reiterated that the registration is made after the clinical trials. "Again, the clinical trials are costly, and the drug production is costly as well," the scientist added.

Dinavir proved to be safe while tested on cells, in vitro. A Phase II pre-clinical trial will utilize animal models to test the efficiency and safety of treatment. A Phase I clinical trial will be carried out on humans to test safety of the therapy and will take up not less than a year.

"Phase II takes up two to three years, and it is unclear how much will be required from us. Phase I is about safety, and it takes a few patients: five, maybe ten. Phase II is when we have to prove that the drug works in these five to ten [patients] and that it had a positive effect on them. Phase III is when we enroll a lot of patients [in the trial] to show that the five were cured not by accident and that it [the gene therapy] really works," Glazkova explained.

The gene therapy for HIV treatment is being developed by a group of researches at the Epidemiology Central Research Institute of Rospotrebnadzor.

In other media

Read more from the original source:

Scientists foresee Russian gene therapy for HIV cure may be registered in 5-10 years - TASS

Imagine only being able to see the things in front of you in soft focus, and just in black and white. For people with the genetic eye condition achromatopsia those are just some of the side effects. But, researchers are testing a new treatment designed to cure the condition by fixing the gene responsible.

Each time Tara Cataldo prepares to leave her house, she has to make sure her face is completely shielded from the sun.

I need to have very dark, very tinted sunglasses to feel comfortable outside and to see really well. Cataldo said.

Cataldo has achromatopsia, a genetic condition that makes her eyes incredibly sensitive to light. She is also very nearsighted; even while wearing glasses or contacts, she can only see clearly at a very short distance.

I cannot drive a car so I rely on public transportation and my bike to get around. Cataldo explained.

"There are currently no approved and no effective treatments for achromatopsia, said University of Florida surgical ophthalmologist Christine Kay.

Kay is working to change that. She is one of a handful of experts testing a gene therapy.

For achromatopsia the cells we have to target are cone cells responsible for decreased vision and color vision and those are cells at the very bottom layer of the retina, Kay explained.

Using a tiny cannula, surgeons deliver a normal copy of one of two mutated genes; the CNGA3 or CNGB3 gene, directly into the eye - restoring vision.

Cataldos myopia is so severe that her risk of retinal detachment from any retinal surgery is high, which rules her out for the current trial. In the meantime, Cataldo says shes learned to adapt to achromatopsia and live without limitations.

And I hope all young achromats learn the same thing, Cataldo said.

Kay says if the gene could eventually be delivered to the surface of the retina; additional patients, like Cataldo, could be treated. Applied Genetic Technologies Corporation, the biotech company that developed the therapies, and several U.S. universities have successfully tested this therapy in dogs and sheep.

Published 2 hours ago

Excerpt from:

Achromatopsia: Gene Therapy Cures Eye Disease? - NBC 5 Dallas-Fort Worth

LONDON--(BUSINESS WIRE)--According to the latest market study released by Technavio, the global cancer gene therapy market is expected to grow at a CAGR of almost 21% during the forecast period.

This research report titled Global Cancer Gene Therapy Market 2017-2021 provides an in-depth analysis of the market in terms of revenue and emerging market trends. This market research report also includes up to date analysis and forecasts for various market segments and all geographical regions.

The rising prevalence rate of cancer has been a huge challenge for the global economies as the disease leads to high rate of mortality and economic losses. The current treatment options available come with many drawbacks such as severe side effects and relapse of cancer. These factors have led to high investment in the R&D for development of various novel therapies with cancer gene therapy being one of the major ones of them. The therapy mainly uses three types of treatment options namely oncolytic virotherapy, gene transfer therapy, and gene-induced immunotherapy.

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Technavios healthcare and life sciences research analysts categorize the global cancer gene therapy market into the following segments by therapy. They are:

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Technavios sample reports are free of charge and contain multiple sections of the report including the market size and forecast, drivers, challenges, trends, and more.

Oncolytic virotherapy

Oncolytic virotherapy is one of the fastest growing treatment modality. In this therapy, the anti-cancer cells specifically destroy the cancer cells without causing harm to the normal cells. Each virus has a specific cellular tropism that determines which tissue will be preferentially infected by the virus and thus will further lead to the disease.

According to Sapna Jha, a lead oncology research analyst from Technavio, The oncolytic virotherapy has shown encouraging results in the pre-clinical studies. The novel treatment option holds great opportunity to make a significant effect on quality and length of the life of the individual. Adenovirus is the most commonly used virus in oncolytic virotherapy.

Gene transfer

Gene transfer or gene insertion is one of the most exciting and emerging cancer treatment methods. The therapy is expected to be the fastest growing type of therapy in the cancer gene therapy market. This is a radical new treatment method that involves the introduction of a new gene into the cancer cell or the surrounding tissues.

Genes with different functions have been proposed for this therapy; some of them include antiangiogenesis genes, cellular stasis genes, and suicide genes. Many different viral vectors are used to deliver these genes, Adenovirus being most common of them. Other than viral vectors, certain non-viral methods are also studied in the various clinical trial, which includes oligodendromer DNA coatings and naked DNA transfer, adds Sapna.

Gene-induced immunotherapy

Immunotherapy works on the concept of boosting the immune system of the individual to target and destroy cancer cells. However, traditional immunotherapy has shown limited success rate in the field. Various gene therapy techniques are being used to overcome this limitation.

The next-generation gene-induced immunotherapy vaccines are already in clinical trial. Gene-induced immunotherapy is a type of gene therapy where genetically engineered genes are used to generate an immune response against cancer. Growing knowledge and understanding of mechanisms regulating the initiation and maintenance of cytotoxic immune response has led to the designing of several genetic immunization strategies.

The top vendors highlighted by Technavios research analysts in this report are:

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Technavio is a leading global technology research and advisory company. Their research and analysis focuses on emerging market trends and provides actionable insights to help businesses identify market opportunities and develop effective strategies to optimize their market positions.

With over 500 specialized analysts, Technavios report library consists of more than 10,000 reports and counting, covering 800 technologies, spanning across 50 countries. Their client base consists of enterprises of all sizes, including more than 100 Fortune 500 companies. This growing client base relies on Technavios comprehensive coverage, extensive research, and actionable market insights to identify opportunities in existing and potential markets and assess their competitive positions within changing market scenarios.

If you are interested in more information, please contact our media team at media@technavio.com.

Link:

Cancer Gene Therapy Market - Forecasts and Opportunity Assessment by Technavio - Business Wire (press release)

August 16, 2017 09:25 ET | Source: Abeona Therapeutics Inc

NEW YORK andCLEVELAND, Aug. 16, 2017 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc.(Nasdaq:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, today announced the pivotal expansion of its gene therapy clinical trials for patients with MPS IIIA in the USA, Europe and Australia.

We have completed the necessary regulatory and ethical committee approvals and site initiations in Europe and Australia in order to accelerate enrollment, stated Juan Ruiz, M.D., Ph.D., Abeonas Chief Medical Officer. We remain very encouraged by the improvements observed in clinically relevant biomarkers post-dosing of ABO-102, including durable reductions in heparan sulfate measured in the CNS, reduction of organ disease pathology, and signals of CNS improvement or stabilization at one-year follow-up in Cohort 1 subjects, and look forward to providing a more fulsome clinical update at important clinical conferences, including ESGCT, this fall, he continued.

The planned expansion will enroll an additional eight to ten MPS IIIA subjects, with total enrollment of fourteen to sixteen subjects to be completed by 1Q2018. Per the design of the clinical trial, subjects will receive a single, intravenous injection of ABO-102 to deliver the AAV viral vector systemically throughout the body to introduce a corrective copy of the gene that underlies the cause of the MPS IIIA disease. Subjects are evaluated at multiple time points post-injection for safety assessments and initial signals of biopotency and clinical activity, which suggest that ABO-102 successfully reached target tissues throughout the body, including the central nervous system.

Sanfilippo syndromes (or mucopolysaccharidosis (MPS) type III): a group of four inherited genetic diseases each caused by a single gene defect, described as type A, B, C or D, which cause enzyme deficiencies that result in the abnormal accumulation of glycosaminoglycans (GAGs, or sugars) in body tissues. MPS III is a lysosomal storage disease, a group of rare inborn errors of metabolism resulting from deficiency in normal lysosomal function. The incidence of MPS III (all four types combined) is estimated to be 1 in 70,000 births. Mucopolysaccharides are long chains of sugar molecule used in the building of connective tissues in the body. There is a continuous process in the body of replacing used materials and breaking them down for disposal. Children with MPS III are missing an enzyme which is essential in breaking down the used mucopolysaccharides called heparan sulfate. The partially broken down mucopolysaccharides remain stored in cells in the body causing progressive damage. In MPS III, the predominant symptoms occur due to accumulation within the central nervous system (CNS), including the brain and spinal cord, resulting in cognitive decline, motor dysfunction, and eventual death. Importantly, there is no cure for MPS III and treatments are largely supportive care.

About Abeona: Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene therapies for life-threatening rare genetic diseases. Abeona's lead programs include ABO-102 (AAV-SGSH), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type A (MPS IIIA) and EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB). Abeona is also developing ABO-101 (AAV-NAGLU) for Sanfilippo syndrome type B (MPS IIIB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) for treatment of infantile Batten disease (INCL), EB-201 for epidermolysis bullosa (EB), ABO-301 (AAV-FANCC) for Fanconi anemia (FA) disorder and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy for rare blood diseases. In addition, Abeona has a proprietary vector platform, AIM, for next generation product candidates. For more information, visit http://www.abeonatherapeutics.com.

Investor Contact: Christine Silverstein Vice President, Investor Relations Abeona Therapeutics Inc. +1 (212)786-6212 csilverstein@abeonatherapeutics.com

Media Contact: Andrea Lucca Vice President, Communications & Operations Abeona Therapeutics Inc. +1 (212)786-6208 alucca@abeonatherapeutics.com

This press release contains certain statements that are forward-looking within the meaning of Section 27a of the Securities Act of 1933, as amended, and that involve risks and uncertainties. These statements include, without limitation, our plans for continued development and internationalization of our clinical programs, that patients will continue to be identified, enrolled, treated and monitored in the EB-101 clinical trial, and that studies will continue to indicate that EB-101 is well-tolerated and may offer significant improvements in wound healing; the addition of two additional global clinical sites will accelerate our ability to enroll and evaluate ABO-102 as a potential treatment for patients with Sanfilippo syndrome type A, or MPS IIIA. Such statements are subject to numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the impact of competition; the ability to secure licenses for any technology that may be necessary to commercialize our products; the ability to achieve or obtain necessary regulatory approvals; the impact of changes in the financial markets and global economic conditions; our belief that initial signals of biopotency and clinical activity, which suggest that ABO-102 successfully reached target tissues throughout the body, including the central nervous system and the increased reductions in CNS GAG support our approach for intravenous delivery for subjects with Sanfilippo syndromes, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other reports filed by the Company with theSecurities and Exchange Commission. The Company undertakes no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

See original here:

Abeona Therapeutics Announces Pivotal Expansion of ABO-102 Gene Therapy Clinical Trials in Sanfilippo Syndrome ... - GlobeNewswire (press release)

Gene therapies for children with Batten disease are being evaluated in two clinical trials one in New York and the other in Ohio. The trials are testing ways to treat two types Batten disease by correcting the genetic defect that causes it.

One study is a Phase 1/2 clinical trial (NCT01414985) taking place at Weill Cornell Medical College in New York thats assessing the safety and effectiveness of using the AAVRh.10 virus to deliver a corrected CLN2 gene to children with late infantile Batten disease, also known as Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL).

This type of Batten disease, a lysosomal storage disorder, can be caused by a mutation in the CLN2 gene. The damage disrupts the ability of brain cells to recycle proteins, killing nerve cells within the brain and leading to progressive neurological and brain damage. The onset of symptoms, a mix of vision and motor problems, are usually evident in children between 2 and 4 years old.

The trial, which is recruiting eight patients ages 3 to 18 with a definite diagnosis of LINCL, will deliver a healthy CLN2 gene using the AAVRh.10 virus a primate-derived virus known to be safe in people as the gene transfer vector. Two study arms are planned: one, involving two patients, will receive a higher dose of gene copies, and the other, composed of six patients, will be given a lower dose.

According to a Weill Cornell document on file with the National Institutes of Health, studies in CLN2 knockout mice showed the virus to be an effective gene delivery system and the treatment to have the potential to slow down the progression of the disease.

Treatment effectiveness will be determined at months 1, 6, 12, and 18 using the Weill Cornell LINCL scale, a 12-point measure of central nervous system response through changes in feeding, gait, and motor and language skills. Secondary measures will be judged using MRI scans and questionnaires.

This study expects to conclude in December 2020, and will be led by Dr. Ronald Crystal of Weill Medical College. More information, including enrollment contacts, is available on its clinical trials.gov webpage.

In an earlier trial, the same Weill team used the AAV2 virus to deliver a corrected CLN2 gene, and reported that it showed both safety and small but significant benefit in treated patients. Researchers believe the AAVRh.10 virus is a more effective delivery system than AAV2, the NIH document states.

In the second trial, NCT02725580, the adeno-associated virus 9 (AAV9) will be used to carry a corrected CLN6 gene to patients with the variant late infantile Batten CLN6 disease. This open label and first-in-human trial, taking place at Nationwide Childrens Hospital in Columbus, Ohio, will enroll at least six people, ages 1 year or older, with a mutation in that gene. The treatment will be delivered once directly into the spinal cord through a lumbar puncture.

Safety and toxicity is this studys primary objective, but secondary measures of treatment efficacy will be made using MRI scans and cognitive, vision and language testing.

The Gray Foundation, established in 2015 by the parents of Charlotte and Gwenyth Gray, both of whom have this variant of Batten disease with a mutation in the CLN6 gene, is funding and collaborating in this study.

Its principal investigator is Dr. Jerry Mendell with the Center for Gene Therapy at Nationwide Childrens Hospital.

Trial enrollment and other information is available on the studys clinical trials.gov webpage.

Continued here:

Batten Disease Clinical Trials Enrolling Patients to Evaluate Gene Therapies - Batten Disease News

Friedreichs Ataxia (FA) is a genetic condition caused by mutations in theFXNgene. The mutation leads to a decrease in the production of frataxin protein causing progressive neuronal degeneration, loss of muscle control, fatigue, vision or hearing impairment, slurred speech, and heart problems. As FA is caused by mutations in a single gene, it might be treatable through agene therapyapproach.

Gene therapy consists of the intracellular delivery of genetic material to generate a therapeutic effect by correcting an existing abnormality or providing the cells with a new function.

Different types of gene delivery systems may be used in gene therapy to restore a specific gene function, such as viral and non-viral vectors.

Viral vectors are viruses that have been modified by deleting the harmful areas in their genomes, while non-viral vectors are physical and chemical systems, such as liposomes, nanoparticles, polymers, ultrasound, or laser-based or magnetic energy.

Non-viral vectors are advantageous in relation to viral vectors as delivery vehicles as they are simple to prepare and scale-up and usually have less pro-inflammatory effects than viral ones.

A gene therapy approach for the treatment of FA could be used either to correct the faulty frataxin geneor to correct any damage caused by the disease on other systems in the body such as the heart.

Voyager Therapeuticshas a pipeline of investigational molecules to target severe diseases of the central nervous system, such as FA. They are currently starting preclinical tests to assess the safety and effectiveness of VY-FXN01, a lead candidate for the treatment of theneurological symptoms of FA.

Earlier in 2014, mouse models of FA were used to demonstrate that gene therapy using a viral vector prevented and corrected cardiac damage.Researchers used an adeno-associated virus to introduce a normal gene into the heart tissue of mouse models of FA. This treatment restored heart function and reversed heart enlargement in mice that had already developed heart failure, a symptom seen in people with FA.

A project supported byGENEFA, a platform for an FA cure, is investigating ways to modify vectors to improve their delivery across the blood brain barrier(BBB). By generating modified viral vectors and virus-free synthetic nanoparticles the aim is to make the DNA delivery into the nervous system more efficient. It is hoped that these nanosystems will cross the BBB thanks to the peptides that are able to cross them (referred to as BBB-shuttles).

Note:Friedreichs Ataxia Newsis strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician orother qualified health providerwith any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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Gene Therapy for Friedreich's Ataxia - Friedreich's Ataxia News

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Report Scope:

Gene Therapy market forecastsfrom2017-2027

This reportassesses the approved gene therapy productsin the market and givesrevenue to 2027 for Neovasculgen

Providesqualitative analysis and forecastof thesubmarket by indicationfor the period 2017-2027: Cancer Cardiovascular disorders Rare diseases Ophthalmological diseases Other therapeutic uses

Profilesleading companiesthat will be important in the development of the gene therapy market. For each company, developments and outlooks are discussed and companies covered in this chapter include: UniQure Biogen Bluebird Bio Spark Therapeutics Applied Genetics Technologies Corporation Oxford Biomedica GenSight Biologics

Assesses the outlook for theleading gene treatment R&D pipelinefor 2016 and discusses technological progress and potential. Profiles appear for gene therapy drug candidates, withrevenue forecasts for six leading agents: SPK-RPE65 (Spark Therapeutics) Collategene (AMG0001, AnGes MG/Vical) Invossa (TissueGene-C, TissueGene Inc/Kolon Life Science) BC-819 (BioCancell) Lenti-D (Bluebird Bio) GSK2696273 (GlaxoSmithKline)

Provides qualitative analysis of trends that will affect the gene therapies market, from the perspective of pharmaceutical companies, during the period 2017 to 2027.SWOT analysisis provided andan overview of regulation of the gene therapy market by leading regiongiven.

Our study discussesfactors that influence the marketincluding these: Translation of research into marketable products modifying human DNA - gene transfer for therapeutic use, altering the nuclear genome Genomic editing technology and other supporting components Collaborations to develop and launch gene-based products - acquisitions and licensing deals Supporting technologies for human genetic modification, gene replacement and targeted drug delivery Gene therapies for ophthalmologic diseases - next-generation medicines Regulations in the United States, the European Union and Japan - overcoming technological and medical challenges to pass clinical trials.

Visiongain's study is intended for anyone requiring commercial analyses for the gene therapy market. You find data, trends and predictions.

Buy our report todayGene Therapy R&D and Revenue Forecasts 2017-2027: Cancer, Cardiovascular, Rare Diseases, Ophthalmologic, Other Diseases.

To request a report overview of this report please email Sara Peerun at sara.peerun@visiongain.com or call Tel: +44-(0)-20-7336-6100

Or click on https://www.visiongain.com/Report/1954/Gene-Therapy-R-D-and-Revenue-Forecasts-2017-2027

List of Companies and Organisations Mentioned in the Report:

Active Medical, Inc.

AngioDynamics, Inc.

Aspen Laboratories

AtriCure, Inc.

Barcapel Foundation

Biosense Webster, Inc.

Boston Scientific Corporation

British Association of Aesthetic Plastic Surgeons (BAAPS)

BSD Medical Corporation

C.R. Bard

Cosman medical, Inc.

Covidien

DFINE, Inc.

Endosense SA

Ethicon

Food and Drug Administration (FDA)

Galil medical, Inc.

Johnson & Johnson

Linvatec Canada ULC

Macmillan Cancer Support

Medtronic

Microsulis Medical Ltd.

Monteris Medical

National Institute of Health Research (NIHR)

nContact, Inc.

NeuroTherm, Inc.

NeuWave Medical, Inc.

NxThera, Inc.

Olympus Corporation

Perseon Corporation

Profound Medical Corp.

Royal Brompton & Harefield NHS Foundation Trust

Royal Philips

Shandong Provincial Hospital

Smith & Nephew

SonaCare Medical

St Jude Medical

Terumo Europe

The American Heart Association

Trod Medical N.V.

University College London

To see a report overview please email Sara Peerun on sara.peerun@visiongain.com

SOURCE Visiongain Ltd

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Gene Therapy R&D and Revenue Forecasts 2017-2027 - PR Newswire (press release)

DUBLIN--(BUSINESS WIRE)--The "Global Cancer Gene Therapy Market 2017-2021" report has been added to Research and Markets' offering.

The global cancer gene therapy market to grow at a CAGR of 20.63% during the period 2017-2021.

The report, Global Cancer Gene Therapy Market 2017-2021, has been prepared based on an in-depth market analysis with inputs from industry experts. The report covers the market landscape and its growth prospects over the coming years. The report also includes a discussion of the key vendors operating in this market.

According to the report, one driver in the market is rising geriatric population. The global geriatric population is continues to grow at a faster pace due to several factors such as rapidly falling fertility rates and growing life expectancy due to better medical facilities. The US Census Bureau reported that the total population aging 65 years and above was estimated at 617 million in 2015 and is expected to rise to 1,566 million by 2050.

Asia has the largest and fastest growing aging population due to several factors such as the huge population of the region, government policies such as one child policy of China has reduced the addition of young population, and higher investment in the healthcare sector has led to better medical facilities and longer life expectancy.

Key vendors

Other prominent vendors

Key Topics Covered:

Part 01: Executive Summary

Part 02: Scope Of The Report

Part 03: Research Methodology

Part 04: Market Landscape

Part 05: Pipeline Analysis

Part 06: Market Segmentation By Therapy

Part 07: Geographical Segmentation

Part 08: Decision Framework

Part 09: Drivers And Challenges

Part 10: Market Trends

Part 11: Vendor Landscape

Part 12: Key Vendor Analysis

Part 13: Appendix

For more information about this report visit https://www.researchandmarkets.com/research/hrzr8h/global_cancer

Original post:

Global Cancer Gene Therapy Market to Grow at a CAGR of 20.6 ... - Business Wire (press release)

Dive Brief:

Fortress Biotech is hard to characterize. It is a hybrid of a traditional biopharma developing its own clinical candidate and a venture capital company securing financing and providing hands-on support for the companies it launches.

Describing itself as therapeutic area-agnostic, Fortress and its ten subsidiaries are developing drugs and devices across a wide range of fields, including dermatology, cancer, trauma, transplantation and neurology.

Most of the candidates under development by its subsidiaries are in preclinical or Phase 1 testing, but several from Avenue Therapeutics and Cyprium Therapeutics have advanced into mid- and late-stage testing. Another Fortress company, Journey Medical Corp, markets four drugs for skin conditions and wounds.

Aevitas will take aim at aHUS and PNH, two rare diseases with still unmet medical needs. While gene therapy offers an attractive approach to addressing each, other companies are already established in the space. Alexion Pharmaceuticals, Inc. has had success marketing Soliris (eculizumab) for both conditions.

Elsewhere in clinical testing, Ra Pharmaceuticals, Inc. is developing its Phase 2 agent RA101495 while Alnylam Pharmaceuticals, Inc. has ALN-CC5 in Phase 1/2.

Aevitas isn't Fortress' first step into the growing field of gene therapy. Cyprium, which was launched in March, is developing a gene therapy for a rare inherited copper metabolism disorder called Menkes disease.

If this Menkes disease is not treated before or soon after birth, it can cause developmental delay, epilepsy and death before the age of three. AAV-ATP7A, as Cyprium's gene therapy is known, is currently in preclinical development.

Under a Cooperative Research and Development Agreement with the Eunice Kennedy Shriver National Institute of Child health and Human Development, Cyprium gained access to a Phase 3 candidate for Menke's disease that it hopes to pair with its gene therapy.

As for Fortress Biotech itself, its business was boosted considerably through the September 2016 acquisition of National Holdings Corporation, which added $46.3 million to net revenues from the second quarter of this year.

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Fortress adds 10th subsidiary to biotech network - BioPharma Dive

A revolutionary gene therapy treatment for leukaemia is awaiting a thumbs-upin the US and could change the game in countering the disease.

If the Food and Drug Administration (FDA)approves the treatmentin September,CTL019will be the first gene therapy allowed for clinical use in the US and the fourth in the world, following Glybera and Strimvelis, two gene- therapy products recently approved in Europe for two genetic diseases, and Gendicine (for cancer) in China in 2004.

CTL019 is a custom-made, personalised drug developed by the University of Pennsylvania and health company Novartis. The treatment consists of a single shot of immune cells reprogrammed to kill the patients specific leukaemic cells.

Clinical trials showremarkable results. Originally tested in 30 patients affected by resistant acute B-lymphoblastic leukaemia (ALL) an aggressive blood cancer CTL019 induced complete remission (meaning that the leukaemia vanished) in 90 per cent of patients. The trial was later extended to 50 patients and resulted in a similar remission rate, which is extremely high for ALL.

ALL accounts for 25 per cent of all cancer in children. The standard treatment is based on chemotherapy followed by bone marrow transplantation. If the standard treatment fails, a bone marrow donor is unavailable or the patient relapses, there are no other therapeutic options and the chances of survival are slim. It is estimated that 15 per cent of cases do not respond to standard treatment.

CTL019 represents a major opportunity for these children. Indeed, families of children affected by ALL, whose lives were saved by CTL019, strongly advocated for its approval.

How it works

The treatment is the result of more than 20 years of research in the fields of molecular biology, virology and haematology.

The administration of CTL019 is a lengthy procedure. Immune cells called T cells are taken from the patient using a procedure similar to blood collection. The cells are then grown and genetically modified in a laboratory for expression of a molecule receptor called CAR (chimeric antigen receptor) which is able to recognise CD19, a molecule widely expressed on B-leukemic cells. The genetically modified immune cells are then administered back to the patient in a procedure which resembles a transfusion.

Micro-management: CLT019 is added to T cells (Shutterstock)

It is unclear how long the CTL019 reprogrammed cells will persist in the patients circulation and whether this should be a reason to worry or celebrate. It is not fully understood whether they will remain for long periods of time and go on to harm the patient, for example, or persist and offer protection from any further leukaemic growth.

In the lab, CTL019 has been added to T cells by inserting specific genes inside the T cells using viruses as vectors for the genetic transfer. This is also the system used in the Strimvelis and Glybera treatments.

The approach exploits the natural ability of certain viruses to infect cells and introduce genes into them. The vectors used are not virulent and therefore they are not able to cause a disease, but they can add genes to the human genome.

The first trials using this type of approach didnt fully take into consideration the potential harm of inserting a gene into a random sequence of human DNA. The results had a tragic setback: in 2002, a gene therapy patient developed a leukaemia. This had a profound impact on public opinion.

Since then, enormous efforts have been made to improve the methodology and vector design in order to reduce the risks. Nevertheless, the long-term effects of such new treatments are unknown and patients will need to be under close monitoring for 15 years.

As scientists, we all hope and work hard to find a definitive cure for all cancers. We dont know yet if the patients that undertook the CTL019 trials can be considered cured or whether they will eventually relapse. But this drug is an opportunity to prolong the survival of patients that currently have no other option. In the meantime, we will celebrate every success in the battle to prolong the survival of cancer patients.

An analogous protocol called UCART19, is also under development at the Great Ormond Street Hospital in London. In 2015, a one-year-old baby affected by refractory ALL was the first patient ever treated with UCART19. The brilliant results led to the development of a phase I clinical trial.

No treatment can be considered 100 per cent safe. For every patient, there are risks and benefits to take into consideration. Nevertheless, CTL019 has produced some excellent results and the side effects have been managed well. At the moment, this treatment is a very promising option for patients with relapsed ALL. Lets hope it saves lives.

Maria Teresa Esposito is a lecturer in biomedical science (cancer biology) at the University of East London. This article was originally published on The Conversation (www.theconversation.com)

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A revolutionary leukaemia treatment could soon be approved here's what it means for patients - The Independent

LONDON, Aug 8 (Reuters) - Gene therapy, which aims to patch faulty genes with working DNA, has been a long time in development. The following are major milestones:

1972 - Researchers first suggest gene therapy as a treatment for genetic diseases but oppose its use in humans "for the foreseeable future", pending greater understanding of the technology.

1990 - A four-year-old girl with severe immunodeficiency became the first patient to undergo gene therapy in the United States.

1999 - American patient Jesse Gelsinger dies following a gene therapy experiment, setting the field back several years as U.S. regulators put some experiments on hold.

2002-03 - Cases of leukaemia are diagnosed in French children undergoing gene therapy in a further blow to the field.

2003 - The world's first gene therapy is approved in China for the treatment of head and neck cancer.

2007 - Doctors carry out the world's first operation using gene therapy to treat a serious sight disorder caused by a genetic defect.

2012 - Europe approves Glybera, the first gene therapy in a Western market, for an ultra-rare blood disorder.

2016 - Europe approves Strimvelis for a very rare type of immunodeficiency.

2017 or 2018 - The first gene therapy could be approved in United States. (Reporting by Ben Hirschler; editing by David Stamp)

Originally posted here:

TIMELINE-Gene therapy's long road to market - Reuters - Reuters

Human protein-coding genes number from 20,000 up to 25,000.

If just one of these genes gets altered or a code gets missing, it can be fatal to an individual.

In fact, approximately 30 per cent of infant mortality at birth in developed countries are caused by genetic disease. Almost 50 per cent of all miscarriages worldwide are due to chromosomally defective fetus.

Furthermore, according to the World Health Organization, over 10,000 human diseases are linked to single gene mutation alone. Among these monogenic diseases are thalassaemia, sickle cell anemia, haemophilia, Fragile-X syndrome, cystic fibrosis, and Huntingtons disease.

The other two major types of genetic disorders are chromosomal and complex disorder, where theres mutation in two or more genes.

Genetic disease is not also simply inherited, our environment is another factor that can trigger mutation. Cancer, diabetes, and heart disease are classified as multifactorial inheritance genetic disorders.

Considering all these, one would expect that the world will be welcoming the revolutionary gene therapy with wide-open arms.

Yet, UniQures Glybera has been recently withdrawn from the European market in spite of its promising one-time cure for lipoprotein lipase deficiency (LPLD).

LPLD is a rare genetic disorder characterized by the bodys lack of lipase, which is an enzyme that breaks down triglycerides from the blood. The deficiency results to recurrent abdominal pain, fat deposits in the skin (xanthomata), and repeated attacks of acute pancreatitis. LPLD is known to affect one person in a million. However, UniQures Glybera costs as much as $1 million per patient. Since the drugs introduction in 2012, only one patient has been subscribed to the treatment.

Another genetic drug that offers one-time cure for Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) is GlaxoSmithKlines Strimvelis. ADA-SCID is an inherited genetic condition characterized by a damaged immune system. People with SCID are prone to persistent and recurring infections since they absolutely have no immune protection from microbes. Symptoms begin to appear in a babys first 6 months of life, and afflicted infants hardly reach two years of age without treatment.

GlaxoSmithKlines Strimvelis can cure the genetic disease and save precious lifes. But the $700,000 drug had only a couple of sales in 2016 and another two expected this year. With this disappointing development, GSK might simply sell its rare diseases unit.

Data shows that the prices of the current gene therapy in the market are too hard if not impossible for most families to reach, especially since it has to be a one-time payment. And health care systems which only pay on monthly basis are not of much help to pharmaceuticals, which have made such enormous investments to formulate genetic cures.

Is there real hope?

Many drug companies still think so.

Pfizer, Sanofi, and Shire are now also making the revolutionary pursuits. And GSK has not completely given up as it strives to use its gene therapy platform in the development of cure for more common genetic illnesses.

Yes, at the moment, the whole picture may appear dim. But, by creating new business models, the leading companies in the biopharmaceutical industry if they are really serious about doing something in relation to rampant increases in drug prices, can start by creating a business model which is first based on humanism and then their respective bottom lines.

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Gene Therapy is Finally Here, But Who Will Foot the Bill - Wall Street Pit

A proof-of-concept study in mice has demonstrated how skin grafts could deliver gene therapy for obesity and diabetes.

'We think this platform has the potential to lead to safe and durable gene therapy, in mice and we hope, someday, in humans, using selected and modified cells from skin,' said senior author Dr Xiaoyang Wu of the University of Chicago, Illinois.

The technique explores the potential of glucagon-like peptide 1 (GLP1), a hormone which could help to treat conditions like diabetes and obesity. GLP1 reduces appetite and stimulates the release of insulin to lowerblood sugar, butdoes not last long in the blood and is challenging to deliver orally.

The researchers used CRISPR to edit skin stem cellstaken from newborn mice. They inserted a modified version of the GLP1 gene, designed to increase the duration of the hormone, and a genetic'switch' to turn the gene on in the presence of an antibiotic.

They grew the skin stem cells into a skin organoid, and grafted them onto mice. When the mice were fed small amounts of antibiotic, theysuccessfully produced modified GLP1, which lasted for three months, and showed higher levels of insulin and lower levels of glucose.

The researchers also tested feeding the mice a high-fat diet. Compared to controls, the mice with modified GLP1 skin grafts put on less weight.

Dr Wu said the skin graft method could be safer than using engineered viral vectorsto edit genes in patient's own tissues, as viruses 'may cause a very strong immune reaction and inflammation in vivo.' He added that lab-grown skin grafts have been used clinically for some time to treat burns, and have been proven safe.

Being able to control the gene expression using a drug would also allow doctors to calibrate how much of the enzyme enters a patients bloodstream.

'We think this can provide a long-term safe option for the treatment of many diseases,' Dr Wu said. 'It could be used to deliver therapeutic proteins, replacing missing proteins for people with a genetic defect, such as haemophilia. Or it could function as a metabolic sink, removing various toxins.'

Dr Jeffrey Millman of Washington University, St Louis, who was not involved in the study, told The Scientist that more research would be needed to ensure that neither the genome editing nor the stem cell culturing method inadvertently introduce dangerous mutations.

Excerpt from:

Gene therapy skin grafts for obesity and diabetes - BioNews

Pfizer is moving forward with plans to invest in a new clinical and commercial gene therapy manufacturing facility in Sanford, NC, but the work is still in the preliminary stages, said the company. A $100-million investment in the Sanford facilities is expected to create 40 jobs, according to a press release from the North Carolina governors office.

The facility will build upon a technology first developed at the University of North Carolina at Chapel Hill. Gene therapy focuses on highly specialized, one-time treatments that address the root cause of diseases caused by genetic mutation. The technology involves introducing genetic material into the body to deliver a correct copy of a gene to a patients cells to compensate for a defective or missing gene.

Gene therapy is an important area of focus for Pfizer. In 2016, the company acquired Bamboo Therapeutics, a privately held biotechnology company based in Chapel Hill focused on developing gene therapies for the potential treatment of patients with certain rare diseases related to neuromuscular conditions and those affecting the central nervous system. Pfizer also committed $4 million to support postdoctoral fellowships in North Carolina universities for training in gene therapy research, according to the press release.

A performance-based grant of $250,000 from the One North Carolina (NC) Fund will help facilitate Pfizers expansion. The One NC grant will formally be awarded to Wyeth Holdings, a wholly owned subsidiary of Pfizer. The One NC Fund provides financial assistance to local governments to help attract economic investment and to create jobs. Companies receive no money upfront and must meet job creation and capital investment targets to qualify for payment. All One NC grants require a matching grant from local governments and any award is contingent upon that condition being met.

Source: Pfizer, NC Governors Office

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Pfizer Plans Gene Therapy Manufacturing Investment in North ... - BioPharm International

News & Observer

Pfizer's push into gene therapy adds more jobs in Sanford News & Observer Pharmaceutical giant Pfizer is expanding its facilities in Sanford to accommodate the company's push into gene therapy. The state Department of Commerce announced earlier this week that Pfizer would invest $100 million in the site and create 40 jobs ...

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Pfizer's push into gene therapy adds more jobs in Sanford - News & Observer

A new consortium, led by Oxford BioMedica, will embark on a two-year, 2 million project focused on gene and cell therapy manufacturing.

Other partners include the Cell and Gene Therapy Catapult, Stratophase and Synthace, and the collaboration is co-funded by Innovate UK.

The aim of the consortium is to explore and apply novel advanced technologies to further evolve OXBs proprietary suspension LentiVector platform to deliver higher quality vectors for both clinical and commercial use. The project aims to deliver tangible benefits to patients by shortening the time-to-clinic and time-to-market as well as to improve the cost and access of bringing novel gene and cell therapies to patients.

Each partner in the collaboration holds proprietary technology and know-how that can be used to develop an innovative approach to viral vector manufacturing. The aims of this pioneering project are closely aligned with the current government national priorities to make the UK a global hub for manufacturing advanced therapies, which will benefit economic growth and create and retain more highly skilled employment.

John Dawson, CEO of Oxford BioMedica, commented: Cell and gene therapies offer unprecedented promise for the cure, treatment or long term management of disease and we are delighted that this consortium has been awarded funding from Innovate UK that will help to keep Oxford BioMedica, our partners and the UK at the forefront of innovation in industrial viral vector manufacturing."

Keith Thompson, CEO of Cell and Gene Therapy Catapult, added: Collaborating on developing improved process analytic technologies with our partners will help drive productivity in viral vector manufacturing, accelerating the development of these transformative advanced therapies. We have the opportunity to both transform patients lives and grow an industry in the UK that we can be proud of.

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2m UK consortium to tackle gene therapy - PharmaTimes