Category Archives: Gene Medicine

ABO-102 / UX111 is being evaluated in ongoing pivotal Transpher A trial in patients with MPS IIIA

Interim data featured in encore oral presentation at American Society of Gene & Cell Therapy (ASGCT) today

NOVATO, Calif. and NEW YORK and CLEVELAND, May 17, 2022 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) and Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced an exclusive license agreement for AAV gene therapy ABO-102 (now UX111) for the treatment of Sanfilippo syndrome type A (MPS IIIA). Under the terms of the agreement, Ultragenyx will assume responsibility for the ABO-102 program and in return Abeona is eligible to receive tiered royalties of up to 10% on net sales and commercial milestone payments following regulatory approval.

Based on promising data from Abeonas clinical program, regulatory feedback to date, and our experience developing treatments for other MPS diseases, we believe ABO-102 has the potential to be a transformative therapy for patients with MPS IIIA, said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx.Our teams expertise in MPS and gene therapy clinical development makes this program a seamless integration, and it has the potential to be our first gene therapy to market. The Sanfilippo community has been waiting too long for a first treatment and we believe we can help accelerate this program.

Data from the ongoing Transpher A trial demonstrate ABO-102 holds significant potential to improve outcomes for patients with MPS IIIA who experience relentlessly progressing neurodevelopmental and physical decline that is life-threatening at a very young age, said Vish Seshadri, Ph.D., Chief Executive Officer of Abeona. We believe that Ultragenyx, with deep expertise in rare, genetic, metabolic lysosomal storage disorders and a demonstrated commitment towards MPS diseases, is the ideal partner to eventually bring ABO-102 to patients.

Abeona has completed a successful Type B meeting with the U.S. Food and Drug Administration (FDA) regarding the pivotal Transpher A trial to support filing and approval for ABO-102 for the treatment of patients with MPS IIIA. Interim results from the Transpher A trial presented in an encore presentation at the American Society of Gene & Cell Therapy (ASGCT) today demonstrate that neurocognitive development was preserved in children treated before 2 years old or with a development quotient (DQ) > 60 (n=10) within normal range of a non-afflicted child after treatment with ABO-102 (3x1013 vg/kg). The interim results also showed continued or stabilized cognitive function and behavioral progress using standard developmental assessments. Some of these patients have reached 24-months post treatment and stabilization or increase in cortical gray matter, total cerebral, and amygdala volumes have been observed. Statistically significant reduction in liver volume was seen with ABO-102 treatment. Dose-dependent and statistically significant reductions in cerebrospinal fluid and plasma heparan sulfate, demonstrating replacement of enzyme activity consistent with levels required for disease correction in the central nervous system, have been sustained in treated patients for two years after treatment. ABO-102 has been well-tolerated with no treatment-related serious adverse events and no clinically meaningful adverse events reported.

MPS IIIA is characterized by severe neurodegeneration with debilitating symptoms for which there is currently no treatment, said Kevin Flanigan, M.D., director of the Center for Gene Therapy at Nationwide Childrens Hospital in Columbus, Ohio, and Transpher A study principal investigator. The promising results to date suggest a single intravenous dose of ABO-102 AAV-based gene therapy has the potential to help children with MPS IIIA sustain neurocognitive development when they are treated during early stages of their disease.

About ABO-102 / UX111ABO-102 (now UX111), is a novel gene therapy in Phase 1/2 development for Sanfilippo syndrome type A (MPS IIIA), a rare lysosomal storage disease with no approved treatment that primarily affects the central nervous system (CNS). ABO-102 is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the SGSH gene to cells of the CNS and peripheral organs. The therapy is designed to address the underlying SGSH enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. The ABO-102 program has received Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations in the U.S., and PRIME and Orphan medicinal product designations in the EU.

About the Transpher A StudyThe Transpher A Study (ABT-001) is an ongoing, two-year, open-label, dose-escalation, Phase 1/2 global clinical trial assessing ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA). The study is intended for patients from birth to 2 years of age, or patients older than 2 years with a cognitive developmental quotient of 60% or above. ABO-102 gene therapy is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

Further details can be referenced here: https://clinicaltrials.gov/ct2/show/NCT02716246

About Sanfilippo syndrome type A (MPS IIIA)Sanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease with no approved treatment that primarily affects the CNS and is characterized by rapid neurodevelopmental and physical decline, often by age three. MPS IIIA has a global incidence of one in 100,000 with a median life expectancy of 15 years.

Children with MPS IIIA present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive facial features with thick eyebrows or a unibrow, full lips and excessive body hair for ones age, and liver/spleen enlargement are also present in early childhood. MPS IIIA is caused by genetic mutations that lead to a deficiency in the SGSH enzyme responsible for breaking down glycosaminoglycans, which accumulate in cells throughout the body resulting in rapid health decline associated with the disorder.

About Ultragenyx Pharmaceutical Inc.Ultragenyx is a biopharmaceutical company committed to bringing novel therapies to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved medicines and treatment candidates aimed at addressing diseases with high unmet medical need and clear biology, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyxs strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the company's website at: http://www.ultragenyx.com.

About Abeona TherapeuticsAbeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Abeonas lead clinical program is EB-101, its investigational autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development. The Companys development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeonas novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. Abeonas fully integrated gene and cell therapy cGMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL study and is capable of clinical and potential commercial production of AAV-based gene therapies. For more information, visit http://www.abeonatherapeutics.com.

Ultragenyx Forward-Looking Statements and Use of Digital MediaExcept for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its business plans and objectives, the therapeutic potential and clinical benefits of its products and product candidates, expectations regarding the safety and tolerability of its products and product candidates, and future clinical developments or commercial success for its products or product candidates are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the ability of Ultragenyx and its third party partners to successfully develop product candidates, including ABO-102 / UX111, the effects from the COVID-19 pandemic on the companys clinical and commercial activities and business and operating results, risks related to reliance on third party partners to conduct certain activities on the companys behalf, the potential for any license or collaboration agreement, including the companys license agreement with Abeona as described in this press release, to be terminated, uncertainty and potential delays related to clinical drug development, the companys ability to achieve its projected development goals in its expected timeframes, risks and uncertainties related to the regulatory approval process, smaller than anticipated market opportunities for the companys products and product candidates, manufacturing risks, competition from other therapies or products, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the companys future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyxs products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 5, 2022, and its subsequent periodic reports filed with the SEC.

In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyxs investor relations website (https://ir.ultragenyx.com/) and LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/mycompany/).

Abeona Forward-Looking StatementsThis press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. We have attempted to identify forward-looking statements by such terminology as may, will, believe, estimate, expect, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition; continued interest in our rare disease portfolio; our ability to commercialize our EB-101 product candidate; obtaining a strategic partnership to take over development activities for ABO-102; our ability to enroll patients in clinical trials; the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies; the impact of competition; the ability to secure licenses for any technology that may be necessary to commercialize our product candidates; the ability to achieve or obtain necessary regulatory approvals; the impact of changes in the financial markets and global economic conditions; risks associated with data analysis and reporting; reducing our operating expenses and extending our cash runway; our ability to execute our operating plan and achieve important anticipated milestones; and other risks disclosed in the Companys most recent Annual Report on Form 10-K and other periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

ContactsUltragenyx Pharmaceutical Inc. Investors Joshua Higair@ultragenyx.com

Media Jeff BlakeMedia@ultragenyx.com

Abeona Therapeutics Investors and Media Greg Ginir@abeonatherapeutics.com

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Ultragenyx Acquires Global Rights to AAV Gene Therapy ABO-102 for Sanfilippo Syndrome Type A (MPS IIIA) from Abeona Therapeutics - GlobeNewswire

In Greenland in January 2021, a child just under two years old was sickvery sick. And his doctors couldnt figure out why. He was feverish, vomiting, having seizures. Meningitis was suspected to be the cause; a tuberculosis diagnosis was also being tossed around. The child was transferred to Copenhagento Rigshospitalet, the largest hospital in Denmarkfor further evaluation.

By March, the childs doctors were no closer to figuring out why he wasnt getting better. So they reached out to Trine Mogensen, a professor of immunology at Aarhus University in Denmark. It was really unclear what this infection was. And there was no evidence of bacterial infection or tuberculosis, Mogensen says. Stumped, she and her team sequenced the childs genome to see if this uncovered any clues. It came out, surprisingly, that there was a genetic mutation, she says.

What they had found was a mutation in the gene that codes for IFNAR2, a protein that binds to type I interferons. Interferons are a family of proteins that play an essential role in fighting off viral infections. Without type I interferons working well, the child would be unable to mount any kind of immune response to viruses such as Covid-19 and the flu.

Yet what virus the child was facing was still unclear. So Mogensen got in contact with Christopher Duncan, a clinician-scientist who studies viral immunity and interferons at Newcastle University in the United Kingdom. Duncan had been researching the very same genetic mutation for several years, first documenting it in a 2015 paper in the journal Science Translational Medicine. In that paper, he and his colleagues had found the genetic variant in a family from Ireland. A 13-month-old infant had suffered a severe case of encephalitisinflammation of the brainafter receiving the MMR vaccine, which contains live (but weakened) forms of the measles, mumps, and rubella viruses. The childs illness ultimately proved to be fatal.

Following the publication of that paper, Duncan and his colleagues had been contacted by researchers in Alaska, who had identified a couple of childrenunrelatedwho had run into major problems with multiple viruses and had the same genetic variant. He was also alerted to two children in northern Canada with a similar condition.

Knowing this, Mogensen and Duncan went back to the child from Greenlandand finally uncovered the root of his condition. They discovered that three weeks before falling ill, he had also been vaccinated with the live MMR vaccine. (The child survived and is now healthy.) Duncan and Mogensen published their findings in April in the Journal of Experimental Medicine.

But now the team wanted to know if there were more people carrying this uncatalogued genetic mutation. They had noted that the boy from Greenland and the children from Alaska were all of Inuit or Alaska Native heritage. They trawled through the genetic records of 5,000 Inuit and found the variant was surprisingly common: In fact, 1 in 1,500 people in the Inuit population were carrying it. That was hugely surprising, Duncan says.

Here is the original post:
This Gene Mutation Breaks the Immune System. Why Has It Survived? - WIRED

When it comes to unlocking the secrets of DNA repair, Ranjit Bindra, MD, PhD, doesnt think in terms of just resources. The Harvey and Kate Cushing Professor of Therapeutic Radiology and professor of pathology favors a far mightier word: armamentarium. Based on the Latin word for armory, it describes the collection of medicines, equipment, and techniques utilized by a medical practitioner for a field of study.

Yale Cancer Center has an especially impressive armamentarium in the study of BRCA1 and BRCA2, proteins involved with DNA repair that, when mutated, can cause breast, ovarian, prostate, and pancreatic cancers. So when a $1 million grant became available for BRCA gene research from the Gray Foundation in 2018, a diverse team of Yale experts whose perspectives on BRCA gene-driven malignancies provide a 360-degree view from bench to bedside combined their collective skills to secure the sizable gift.

In the three years since, Yales team has made significant advances in targeting the BRCA gene-dependent DNA repair axis for cancer therapy.

Both the BRCA1 and BRCA2 protein are involved in DNA repair, said Megan King, PhD, associate professor of cell biology and of molecular, cellular and development biology, and co-leader of the Radiobiology and Genome Integrity Research Program at Yale Cancer Center. However, the work weve done has shown us that they have fundamentally different mechanisms. Thats important, because typically in clinical trials we lump together patients with BRCA1 and BRCA2 mutations. We need to think about these patient populations differently.

Those mechanisms affect which kind of therapies might work once cancer patients relapse on PARP inhibitors, a treatment that stops PARP proteins from repairing DNA damage in cancer cells and leads to cell death. For example, King has identified that if BRCA1 tumors stop expressing the 53BP1 or REV7 proteinboth of which play a role in repairing DNA double-strand breaksthey become resistant to PARP inhibitors. Thats because the absence of those proteins allows a third enzyme, called the Bloom syndrome protein (BLM), to not only resume the resection of DNA double-strand breaks, but go into repair overdrive, called hyper-resection.

Kings research identified BLM as a novel therapeutic target. She already has a candidate in mind for the job: a new class of drugs called ATR kinase inhibitors. The ATR kinase communicates DNA damage to the cell and activates DNA damage checkpoints, which arrest the cell cycle to provide time for repairs.

BLMs hyper-resection is a vulnerability that makes it sensitive to ATR inhibitors, King explained. She is working to design a clinical trial for ATR inhibitors in BRCA1 patients with fellow Gray Foundation team member Patricia LoRusso, DO, professor of medicine and associate cancer center director of experimental therapeutics.

The teams expertand a world experton BRCA2 is Ryan Jensen, PhD, associate professor of therapeutic radiology and pathology. He was the first scientist to purify and study the properties of the full-length BRCA2 protein. In collaboration with AstraZeneca, Jensen has focused on three BRCA2 reversion alleles, containing deletions in the BRCA2 gene that reactivate DNA repair functions, in tumor cell DNA from ovarian cancer patients who relapsed on a PARP inhibitor.

Hes currently researching whether these alleles alone cause resistance to PARP inhibitors and other cancer treatmentsand therefore, these studies could impact clinical management of patients harboring BRCA2 mutations. Furthermore, by leveraging genetic changes in BRCA2 directly from patients, Jensens team hopes this reverse translation approach will accelerate our understanding of why BRCA2 plays such a crucial role in responding to PARP inhibitors.

Enter Bindra, whose expertise in drug development drives the translation of these laboratory targets into patient therapies. His high-throughput testing capabilities enable him to conduct 96- and 384-well plate-based screening assays in PARP-nave and resistant cell lines. Where it used to take one day to analyze one well of a microplate, Bindra can now look at 384 tiny wells overnight and analyze the images and discover patterns automatically.

Of even greater excitement is Bindras comprehensive library of DNA repair inhibitor and damaging agents. He mixes and matches them in new therapeutic combinations to create novel compounds that can synergize or replace current PARP inhibitors.

When we do this testing in an academic setting instead of a pharmaceutical one, were able to profile all drug candidates out there and focus in an unbiased manner on the best combinations to move forward, Bindra said. This is not pie-in-the-sky scientific inquiry. Because they are clinical focused, these new combinations can be tested in clinics in a matter of one to two years.

Bindras cell lines have proven invaluable in Yales DNA repair research beyond the bounds of the Gray Foundation grant.

Faye Rogers, PhD, associate professor of therapeutic radiology, contributes her knowledge in DNA damage repair to the Gray Foundation team but is also pursuing numerous other research endeavors. She tapped the library for a cell line in her research on the use of endophytes to develop novel cancer-fighting compounds. Endophytes are fungi or bacteria that live symbiotically with plants and can produce the same natural products as their plant host. Theyre known as an untapped source for finding novel bioactive natural products.

An undergraduate student in Rogers lab collected endophytes for study while in Ecuador with Yales Rainforest Expedition and Laboratory Course. Rogers has identified one that produces a compound that inhibits DNA double-strand break repair in cancers with repair deficiencies, such as PTEN-deficient glioblastomas. Were now moving forward to come up with a synthetic version of this compound and conducting some medicinal chemistry to improve its efficacy, she said.

Rogers has returned the favor to the Bindra library. She has advised Bindas students in how to synthesize new classes of DNA repair inhibitors and damaging agents that will further expand Bindras testing capabilities of new compounds. Their teamwork is an example of the cross-disciplinary collaboration exemplified by the Gray Foundation team.

When you bring together people with different skills and perspectives, Bindra said, it adds so much more value to the conversation. And adds yet more invaluable tools to Yales DNA repair armamentarium.

Originally published Feb. 25, 2021; updated May 16, 2022.

More:
BRCA Experts Rally to Research DNA Repair for Better Breast, Ovarian and Other Cancer Treatments - Yale School of Medicine

Presentations convey essential technological and scientific knowledge regarding AAV gene therapy and advancements across key disease areas

CEO Sheila Mikhail and President of Therapeutics, Katherine A. High will be featured speakers

RESEARCH TRIANGLE PARK, N.C., May 16, 2022 /PRNewswire/ --Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, today announced that the Company will present 11 abstracts at the upcoming American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting being heldMay 16 19, 2022 at the Walter E. Washington Convention Center in Washington, D.C.

The ASGCT Annual Meeting is the premier event for professionals in gene and cell therapy where noted industry professionals gather to learn from the latest advances in scientific and clinical research and cell and gene technology. Abstracts being presented by AskBio team members include new data and insights regarding Adeno Associated Virus (AAV) gene therapy, T-cell immune response to empty capsid technologies, inducible promoters and gene expression, and doggybone DNA (dbDNA) as well as data for AskBio's key clinical development programs, including Pompe Disease, Parkinson's Disease and Congestive Heart Failure.

AskBio will make seven oral presentations and four poster presentations. CEO Sheila Mikhail and President of Therapeutics, Katherine A. High will be featured speakers during the event.

"Having 11 abstracts accepted for presentations reflects the significant progress made by our teams across a broad front," commented Kathy High, President, Therapeutics for AskBio. "I am very proud of the groundbreaking work by our research and clinical teams as we continue to advance our therapeutic pipeline and AAV gene therapy research and manufacturing."

Jude Samulski, Chief Science Officer and Co-Founder for AskBio said, "These presentations underscore our commitment to advancing the science of gene therapy to tackle many of the biggest challenges in the space today, including manufacturing, dosing, immune response and treatment efficacy. We hope that, together with our many colleagues in cell and gene therapy space, we can make a profound difference in the lives of patients around the world who are waiting for transformative gene therapies."

AskBio's presentations at ASGCT include:

Monday, May 16

Oral PresentationAbstract 37: Functional Assessment of T-cell Responses to AAV8 Empty Capsids in Healthy VolunteersSession: Immune Responses to AAV Vectors10:30 am 11:45 am, Room: 102

Oral PresentationAbstract 28: A First-in-Human Phase 1 Clinical Gene Therapy Trial for the Treatment of Heart Failure Using a Novel Re-Engineered Adeno-Associated VectorSession: Cardiovascular and Pulmonary Diseases11:45 am 12 PM, Room 206

Featured SpeakerLaunching Innovation Into Gene Therapy CompaniesSession: Part 2: Translating Science Into Medicine: Moving from Bench to Startup (Organized by the Bioindustry & Translational Science Committees)Sheila Mikhail, JD, CEO and Co-Founder, AskBio1:302:18 PM Room: Salon G

Tuesday, May 17Oral Presentation:Abstract 434: Characterization of Alternative Reading Frame Proteins Generated from AAV CassettesSession: Discoveries in Fundamental AAV Biology4:004:15 PM, Ballroom A

Poster Presentation:Abstract 796: Safety and preliminary efficacy of neurosurgical AAV2-GDNF delivery for Parkinson's diseaseSession: Gene and Cell Therapy Trials in Progress5:30PM, Hall D

Poster Presentation:Abstract 711: Effect of tolerogenic ImmTOR nanoparticles on the formation of anti-AAV8 antibodies in mice, nonhuman primates, and healthy human volunteersSession: Immunological Aspects of Gene Therapy and Vaccines I5:30PM, Hall D

Poster Presentation:

Abstract 708: ImmTOR blunts AAVrh32.33 capsid-specific immune responses in C57BL/6 albino miceSession Immunological Aspects of Gene Therapy and Vaccines I5:30PM, Hall D

Wednesday, May 18

Presidential SymposiumTurning Genes into Medicines: The Long and Winding Road from Gene Discovery to Gene Therapeutics

Session: Presidential Symposium and Presentation of Top AbstractsKatherine A. High, MD, President, Therapeutics, AskBio

1:302:15 PM, Hall E

Oral PresentationAbstract 866: Long Term Stability Profiles of AAV Vectors at Ambient Temperature within a Film MatrixSession: Vector Manufacturing and Engineering 3: Improving Vector Design and System Performance5:00PM, Room 201

Poster PresentationAbstract 897: Identification of plasmid backbone-derived antisense RNAs in AAV transduced animalsSession: AAV Vectors - Virology and Vectorology III5:30PM, Hall D

Thursday, May 19

Oral Presentation:Abstract 1203: Inducible Gene Expression for Gene Therapy: Design and Exemplification of Powerful, Small, Modular and Tightly Controlled Regulatable PromotersSession: New Technologies for AAV Gene Therapy10:3010:45 AM, Ballroom C

Oral Presentation:Abstract 1211: Phase 1 Study of Gene Therapy in Late-onset Pompe Disease: Initial 104 Week Experience for Cohort 1Session: AAV Vectors - Clinical Studies10:45AM, Ballroom B

Oral Presentation:Abstract 1213: Rationally Designed Cardiotropic AAV Capsid Demonstrates 30 Fold Higher Efficiency in Human vs Porcine HeartSession: AAV Vectors Clinical Studies11:1511:30 AM, Ballroom B

Abstracts and additional information for the ASGCT 2022 Annual Meeting are available on the ASGCT Annual Meeting web site.

About AskBioAsklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG acquired in 2020,is a fully integrated gene therapy company dedicated to developing life-saving medicines that cure genetic diseases. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson's disease and congestive heart failure, as well as out-licensed clinical indications for hemophilia and Duchenne muscular dystrophy. AskBio's gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, the company has generated hundreds of proprietary capsids and promoters, several of which have entered clinical testing. Founded in 2001 and an early innovator in the gene therapy field, the company holds more than 750 patents in areas such as AAV production and chimeric and self-complementary capsids. Learn more atwww.askbio.comor follow us onLinkedIn.

About BayerBayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2021, the Group employed around 100,000 people and had sales of 44.1 billion euros. R&D expenses before special items amounted to 5.3 billion euros. For more information, go towww.bayer.com.

Find more information at https://pharma.bayer.com/Follow us on Facebook: http://www.facebook.com/pharma.bayerFollow us on Twitter: @BayerPharma

AskBio Forward-Looking StatementsThis press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include without limitation statements regarding AskBio's pipeline of development candidates, manufacturing technology and process. These forward-looking statements involve risks and uncertainties, many of which are beyond AskBio's control. Known risks include, among others: AskBio may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, its reliance on third-parties, clinical development plans, manufacturing processes and plans, and bringing its product candidates to market, due to a variety of reasons, including the ongoing COVID-19 pandemic, possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office. Any of the foregoing risks could materially and adversely affect AskBio's business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. AskBio does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

SOURCE AskBio

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AskBio to Present 11 Abstracts at Upcoming American Society of Gene and Cell Therapy's 25th Annual Meeting - PR Newswire

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Finding therapies for fragile X may depend on understanding the many ways proteins loss affects brain

Researchers at Washington University School of Medicine in St. Louis have identified a previously unknown function for the fragile X protein, the loss of which is the leading inherited cause of intellectual disability. The researchers showed that the protein modulates how neurons in the brains memory center process information, a central part of learning and memory.

Fragile X syndrome, the leading inherited cause of intellectual disability, is due to a genetic mutation that largely eliminates the fragile X protein, a critical element of normal brain development and function.

The fragile X protein modulates neuronal functions, including neurons within the so-called GABAergic system that regulates the activity of neural circuits. The proteins absence throws that system off kilter, and so far, experimental therapies designed to reset the system by compensating for the missing proteins functions have not been effective in clinical trials.

Now, researchers at Washington University School of Medicine in St. Louis have identified a previously unknown role for the fragile X protein in the GABAergic system. They have shown that the protein regulates the opening and closing of the GABA-A receptor in neurons from the brains memory center, thereby influencing how such neurons process information, a central part of learning and memory.

The findings, published May 17 in Cell Reports, indicate that the fragile X proteins role is more complex than previously thought, and that finding effective therapies may depend on a more nuanced understanding of the myriad ways loss of this protein affects the brain.

People think that since fragile X is related to the loss of a single protein, it is a simple disease that we can quickly understand and fix, said senior author Vitaly A. Klyachko, PhD, a professor of cell biology & physiology. But the reality is that the more we study, the more we understand its not simple at all. I think part of the reason why clinical trials fail may be because we dont really understand whats going on very well. It is possible that we need to fix more than one mechanism at the same time for patients to see any meaningful improvement.

People with fragile X syndrome have intellectual or learning disabilities, social and behavioral problems, and characteristic physical features such as large ears and long faces. They also often are noted for their friendly dispositions. The condition affects about 1 in 7,000 males and 1 in 11,000 females, with males typically more severely affected. The fragile X gene is located on the X chromosome, so females inherit one good and one bad copy of the gene, but males have only the bad copy. There are no treatments that address the underlying cause.

The GABAergic system is based on the transmission of gamma aminobutyric acid (GABA) from one neuron to another. When it arrives, GABA binds to a receptor molecule and triggers a cascade of events in the receiving neuron that results in suppression of the activity of that neuron. An overactive GABAergic system puts people to sleep; an underactive one is linked to depression, anxiety and epileptic seizures.

To better understand the role of fragile X protein in the GABAergic system, Klyachko and first author Pan-Yue Deng, MD, PhD, an associate professor of cell biology & physiology, studied neurons from the brains of mice with and without the fragile X protein. Specifically, they recorded the activity of key information-processing neurons controlled by the GABAergic system in the hippocampus, the brains learning and memory center. Such neurons sense the presence of GABA primarily by using the so-called GABA-A receptor.

The receptor is a channel that can open to allow negatively charged chloride ions to flow into the cell to modulate its activity. The researchers found that fragile X protein influences how much time the GABA-A receptor spends open and how much chloride it allows into the cell, thereby setting the baseline electrical charge inside the neuron. This baseline charge, in turn, affects the neurons ability to distinguish between multiple signals that arrive at nearly the same time, a critical mechanism of pattern separation essential for learning and memory formation.

The fragile X protein directly interacts with receptors that play a major role in the way neurons process information, Klyachko said. This is an additional function for the fragile X protein, and it may be an important one. These neuronal receptors are everywhere, and they control many critical brain functions.

But Klyachko cautions against assuming that these findings can be easily translated into therapies for people living with fragile X syndrome. The GABAergic system is complex, and small tweaks can have unexpected and far-reaching effects on brain function, he said.

I think there is a very strong desire an understandable one to immediately translate each discovery into a clinical trial, Klyachko said. But if we dont understand all of the functions this protein has and we try to go after one specific mechanism, it may destabilize the other ones, and the end result is that people dont get better. An entirely different approach to treating this disease may be possible, but I think we need to first understand much more about how it works. This is just the first stepping stone in a new direction.

Deng PY, Kumar A, Cavalli V, Klyachko VA. FMRP regulates GABAA receptor channelactivity to control signal integration in hippocampal granule cells. Cell Reports. May 17, 2022. DOI: 10.1016/j.celrep.2022.110820

This work was supported by the National Institutes of Health (NIH), grant numbers R35NS111596, R01NS111719 and R35NS122260.

Washington University School of Medicines 1,700 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, and currently is No. 4 in research funding from the National Institutes of Health (NIH). Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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Protein linked to intellectual disability has complex role Washington University School of Medicine in St. Louis - Washington University School of...

NEW YORK--(BUSINESS WIRE)--Emendo Biotherapeutics presented the results of its next generation CRISPR-based gene editing approaches for several indications in an oral presentation and three posters at the 25th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) held May 16-19, 2022, in Washington, D.C.

Emendo presented pre-clinical data for the treatment of ELANE-related Severe Congenital Neutropenia using an allele-specific editing approach, demonstrating the power of Emendos dual technology platforms that enable the development of a highly specific editing composition that demonstrates no off-targets and complete allele specificity. Significantly, the lack of off-target achieved by Emendos engineered and optimized OMNI nuclease also eliminated any translocations. Edited patient derived CD34+ cells differentiated normally into neutrophils both in-vitro and in-vivo, showing full engraftment and reconstitution of all blood lineages, as required for the desired therapeutic effect.

ELANE-based Severe Congenital Neutropenia, Emendos lead indication, is a devastating disease affecting pediatric patients that until now has been incurable, said David Baram, Ph.D., President & CEO of Emendo Biotherapeutics. We are excited by the potentially curative treatment developed by our team and were pleased to present our pre-clinical results for this program and other diverse applications of our dual gene editing technology platforms at this years ASGCT meeting. And of course, we look forward to the discussion generated by our discoveries around Type II CRISPR nuclease classifications that promise to be ground-breaking in the field.

Senior members of the Emendo Biotherapeutics R&D team including Chief Technology Officer Lior Izhar, Ph.D. and Executive Vice-President Research & Development Rafi Emmanuel, Ph.D. presented Emendos research on-site at the conference.

Session Presentation

Title: A Novel Engineered CRISPR-Associated Nuclease Accurately Removes ELANE Mutated Allele and Shifts HSC Differentiation Towards Neutrophils in Severe Congenital Neutropenia

Session Title: Gene Therapy for Immunologic DiseasesSession Date/Time: Tuesday May 17, 2022 3:45 PM - 5:30 PMPresentation Time: 3:45pm - 4:00pmRoom: Room 202Final abstract number: 482

Poster Presentations

Title: A Unique CRISPR-Based Nuclease with a Non-NGG PAM Efficiently Targets Multiple Exclusive Genomic Sites for Immuno-Oncology Based Therapy

Session Title: Cancer - Targeted Gene and Cell Therapy ISession Date/Time: Monday May 16, 2022 5:30 PM - 6:30 PMPoster Board Number: M-215Room: Hall DFinal abstract number: 334

Title: CRISPR-Based Gene Editing Enhances LDLR Expression and Boosts LDL-C Uptake in Familial Hypercholesterolemia

Session Title: Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases IISession Date/Time: Wednesday May 18, 2022 5:30 PM - 6:30 PMPoster Board Number: W-125Room: Hall DFinal abstract number: 999

Title: Challenges and Inconsistencies in Type II CRISPR-Associated Nuclease Subtype Classification

Session Title: Gene Targeting and Gene Correction IISession Date/Time: Tuesday May 17, 2022 5:30 PM - 6:30 PMPoster Board Number: Tu-61Room: Hall DFinal abstract number: 556

About Emendo Biotherapeutics

Emendo Biotherapeutics, a subsidiary of AnGes, Inc., is a next generation CRISPR gene editing company leveraging dual proprietary technology platforms to enable high precision gene editing throughout the genome. Emendos novel nuclease discovery platform broadens the targetable range of the genome while its target-specific optimization platform enables highly precise editing, including allele specific editing, while maintaining high efficiencies. The capabilities of the OMNI technology platforms, along with deep expertise in genomic medicine, protein engineering and therapeutic development, provide Emendo with a unique advantage when addressing indications within hematology, oncology, ophthalmology and other disease areas. For more information please visit http://www.emendobio.com.

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Emendo Biotherapeutics' next generation CRISPR gene editing technologies achieve breakthrough results with allele-specific approach for ELANE-related...

Researchers at theUniversity of Colorado School of Medicine have discovered an enzyme that regulates heart stiffness, setting the stage for developing novel treatments for heart failure.

The enzyme,histone deacetylase 6 (HDAC6), has been studied in the context of many diseases, including heart disease, but CU School of Medicine researchersTimothy McKinsey, PhD, professor of medicine in theDivision of Cardiology, andKathleen Woulfe, PhD, assistant professor of medicine in the Division of Cardiology, recently discovered a new role for HDAC6 in the regulation of myofibrils, the contractile units of the heart. The research was published May 16 in The Journal of Clinical Investigation.

When your heart is pumping and relaxing optimally, it's at a certain stiffness, McKinsey says. Stresses including aging, hypertension, and obesity can cause the heart to become too stiff, preventing it from relaxing and filling with blood efficiently,which leads to something called diastolic dysfunction. In other instances, the heart is not stiff enough, so it can't pump effectively, which leads to systolic dysfunction. Both conditions are life-threatening.

The CU researchers found evidence that HDAC6 acts on titin, a massive myofibril protein that contributes to heart stiffness. HDAC6 appears to remove a chemical modification known as acetylation fromtitin.When HDAC6 is inhibited, titin causes the heart to become stiffer; when HDAC6 is activated, the heart becomes less stiff. In the future, once cardiologists determine which type of dysfunction a patient has, it might be possible to therapeutically adjust HDAC6 enzymatic activity or levels to help the heart to pump and relax at an optimal stiffness.

Heart failure is still a huge problem that affects millions of people worldwide, McKinsey says. Even though there are medications to treat heart failure, people with the condition still often have poor quality of life and die at an alarming rate. We think this discovery could provide a novel avenue for treating heart failure through a distinct mechanism.

The progress of therapeutically manipulating HDAC6 to treat heart failure is helped along by the fact that HDAC6 inhibitors are actively being developed to treat other conditions, including neurodegeneration and cancer, though McKinsey cautionsthat the heart needs to be monitored more carefully in people receiving HDAC6 inhibitors.

"Our data suggest that in some cases, if you inhibit this enzyme, the heart might get too stiff, McKinsey says. Nevertheless, we firmly support continued clinical development of HDAC6 inhibitors, since this class of compounds holds great promise for treating a variety of devastating diseases, including certain forms of heart failure.

The CU researchers plan to continue to study the role of HDAC6 in heart stiffness, including testing HDAC6 inhibitors in preclinical models of systolic heart failure where titin is too compliant, and developing gene therapy to provide activated HDAC6 to hearts that are too stiff. Much of their work takes place in Woulfes laboratory, which is one of a handful of labs in the world that can isolate and study myofibril mechanics.

We are able to isolate proteins that direct contraction and relaxation in the heart in a way that preserves the mechanical function, Woulfe says. We can do this from tissue that's frozen, from our human heart bank, or from animals. We take away everything else except for those proteins that contract and relax. They are the fundamental basis of the function of the heart.This system enabled us to discover that HDAC6 directly regulates myofibril stiffness, most likely by deacetylating titin.

We think this is a major finding, and there's still a lot more to do, McKinsey adds. Scientific discovery is a series of building blocks, and we believe this is a key building block that allows us to understand the mechanics of the heart better at a molecular level, and also suggests therapeutic potential. We're going to keep vigorously working on the details of HDAC6 action in the heart.

This work was partially supported by theConsortium for Fibrosis Research & Translation, a program funded by the CU School of Medicine and co-directed by McKinsey. It aims to improve understanding of fibrotic diseases across various organ systems.

In addition to Woulfe and McKinsey, the other researchers on the study areYing-Hsi Lin, Jennifer Major, Joshua Travers, Sara Wennersten, Cortney Wilson, Korey Haefner, Maria Cavasin, Mark Jeong, Yu Han, Amrut Ambardekar, and Maggie Lam from the CU School of Medicine Division of Cardiology; Scott Ferguson from the Cardiovascular and Pulmonary Research Laboratory in the CU Department of Medicine; Tim Liebner and Chunaram Choudhary from the University of Copenhagen, Denmark; Zaynab Hourani and Henk Granzier from the University of Arizona; and Michael Gotthardt from the Max Delbruck Center for Molecular Medicine in Germany.

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CU School of Medicine Research Defines the Role of HDAC6 in Regulating Heart Stiffness - University of Colorado Anschutz Medical Campus

In the latest version of the Human Cells Atlas, 2,000 researchers in 83 countries have defined, one by one, some 60 million cells.

Our mission is to create a map of all human cells, says Israeli biologist Aviv Regev, who began the project in 2016 and leads the international consortium together with the German biologist Sarah Teichmann, from the Wellcome Sanger Institute in Cambridge (UK).

Regev, who is on leave from the Massachusetts Institute of Technology (MIT) and has a management position at US biotech company Genentech, says she believes the cross-continental team is halfway to its goal.

While children study some examples of cells at school such as neurons, red blood cells, white blood cells, and platelets, nobody knows how many types there really are, explains the scientist.

We didnt know how many genes we had until the Human Genome Project. This is a similar case, she says.

The Human Cells Atlas aims to catalog all cell types and their multiple subtypes, but also to locate them precisely in the human body and have a picture of the exact composition of each organ: what we are made of and why diseases arise.

Spanish immunologist Cecilia Domnguez Conde is among the main authors of the latest version, which was reported last week in the journal Science. The researchers describe the detailed profile of more than a million cells; presenting cross-referenced data from 33 organs of the human body, such as the heart, skin and lungs.

Domnguez Condes team is based at the Wellcome Sanger Institute, and has focused on the cells of the immune system.

We have discovered how cells adapt to different environments, says the immunologist, who will begin leading her own laboratory at the Human Technopole, a new research center in Milan (Italy), in June.

The diversity of cell types within the immune system is absolutely incredible, says Domnguez Conde, adding that the work had previously focused on the blood circulating in the body, while now, we study the cells in different tissues and we see new mechanisms.

The immunologist notes that there is a new generation of cancer treatments, the so-called CAR-T, in which white T lymphocyte blood cells are extracted from the patient. Then, using genetic engineering, they are redesigned in the laboratory to increase their ability to destroy cancer cells.

Biology textbooks have traditionally spoken of about 300 cell types in the human body, but the authors of the atlas have found 500 types in the last million cells analyzed. Understanding this astonishing diversity will make it possible to improve vaccines, increase the efficacy of antitumor therapies, facilitate regenerative medicine and develop new treatments for rare and common diseases, according to Regev, who now has a management position at the US biotech company Genentech.

While biology textbooks usually teach that there are about 300 cell types in the human body, the authors of the Human Cell Atlas have now found some 500 types. This new understanding, says Aviv Regev, will contribute to improving vaccines and antitumor therapies as well as regenerative medicine and new treatments for both rare and common diseases.

A mutation in a gene can cause a disease, but although all cells share the same DNA, the problem will only appear in cells that have that particular gene activated.

We have found many unexpected cells that have active disease-associated genes, says Regev.

For example, we have observed non-muscle cells in muscle tissue expressing genes that cause rare muscle diseases.

This is important, because if we want to develop treatments we need to know the cells in order to target them, explains the biologist.

Regev also highlights possible applications of these findings to regenerative medicine, a specialization that tries to rebuild damaged organs using new cells.

To get it right, we need to generate cells with the right properties. The atlas is a reference to ensure that the cells generated in the laboratory have the desired characteristics, he says.

Neuroscientist Rafael Yuste, a professor at Columbia University in New York, applauds the latest version of the atlas.

This batch of results is historic. It is one of the first salvos of what will be a torrent of studies in the next decade that will classify all cell types in the body, says Yuste, who has not participated in the investigation. The Spanish neuroscientist was the founder of BRAIN, a billion-dollar project sponsored in 2013 by then-US president, Barack Obama, to obtain a map of the human brain.

Yuste is optimistic. The new technologies, called transcriptomics, allow cells to be placed in narrow channels and trapped one by one in oily droplets, analyzing their active genes in a manner that is fast, automated and cheap.

The first steps of this strategy have been spectacular. For example, in the United States, the Allen Institute of Brain Sciences has classified all the cells in a part of the cerebral cortex of the mouse, generating for the first time a list of all the types of neurons in an area of the brain, says Yuste, who has collaborated on that project.

Yuste recalls how the father of neuroscience, Santiago Ramn y Cajal, discovered in 1888, using a rudimentary microscope, that neurons were individual cells. Now, says Yuste, it is giant consortia like his BRAIN initiative and the Human Cell Atlas doing this work.

It is a huge effort, says the professor. It will have a fundamental impact on science and medicine, since, in the end, everything that the brain, or the body, does, is cooked up between cell types.

Originally posted here:
Latest Human Cells Atlas reveals more of the unknown world inside our bodies - EL PAS in English

ReportLinker

Middle East Precision Medicine Market to Reach $8,620. 0 Million by 2032. Middle East Precision Medicine Market Industry Overview. The Middle East precision medicine market was estimated to be at $3,942.

New York, May 17, 2022 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Middle East Precision Medicine Market - Country Analysis: Focus on Ecosystem, Technology, Application, End User, and Country Data - Analysis and Forecast, 2022-2032" - https://www.reportlinker.com/p06277485/?utm_source=GNW 6 million in 2021, which is expected to grow with a CAGR of 7.37% and reach $8,620.0 million by 2032. The Middle East precision medicine market is expected to witness high growth, attributed to the rising prevalence of chronic disease, advancement of sequencing technologies, reducing adverse drug reactions through pharmacogenomics tests, and potential to reduce the overall healthcare cost across the globe. The continued significant investments by healthcare companies to meet industry demand and the growing adoption of precision medicine among major end users are the major factors propelling the growth of the Middle East precision medicine market.

Market Lifecycle Stage

Precision medicine refers to the medicine developed as per an individuals genetic profile.It provides guidance regarding the prevention, diagnosis, and treatment of diseases.

The segmentation of the population is done depending on the genome structure of individuals and their compatibility with a specific drug molecule.In the precision medicine market, the application of molecular biology is to study the cause of a patients disease at the molecular level so that target-based therapies or individualized therapies can be applied to cure the patients health-related problems.

This industry is gaining traction owing to the growing awareness about healthcare among individuals, the integration of smart devices such as smartphones and tablets into healthcare, and the increasing collaborations and agreements of information technology (IT) firms with the diagnostics and biopharmaceutical companies for the development of precision diagnostic tools.The growth of the precision medicine market over the last few years has been monumental.

New technologies are rapidly being introduced, expanding the arsenal of tools accessible to support the development and adoption of precision medicine solutions over one-size-fits-all therapies. Advancements in gene therapies, cell therapies, molecular biomarker analysis, and companion diagnostics have the potential to transform medicine and increase the ability to treat and cure several intractable diseases. Advances in sequencing technologies and non-invasive diagnostics, such as liquid biopsy and non-invasive prenatal testing (NIPT), have facilitated the acquisition of real-time data and gained interest for their usage in acquiring the data by exhibiting the biology of tumors and metastatic tissues.

Impact

The presence of major service providers of precision medicine products in Middle East regions has a major impact on the market. For instance, Illumina, Inc. provides NovaSeq 6000 S2, Reagent Kit, NovaSeq 6000 S4 reagent kit, and NovaSeq Xp 4-lane kit in the Middle East. Companies such as F. Hoffmann-La Roche Ltd provide foundation one liquid. The services are a reliable and convenient way to expand in-house resources with expertise and perfectly tailored bioinformatics services that ensure quality results. The presence of these companies has a positive impact on market growth.

Impact of COVID-19

The current Middle East precision medicine market study comprises products and applications utilized to provide beneficial effects to specific health benefits and treatments.Since the market is primarily application and ecosystem-dominated, the COVID-19 pandemic had a low impact on the growth or the revenue generated from the market.

The current market assessment has considered information provided by key opinion leaders in the market, from the supply side as well as the demand side.A majority of the products, such as kits and assays in the market, are used for infectious disease applications.

In addition, infectious diseases are playing an important role in the growth of the market.

Market Segmentation:Segmentation 1: by Ecosystem Applied Sciences Precision Therapeutics Digital Health and Information Technology Precision Diagnostics

The Middle East precision medicine market is expected to be dominated by the precision therapeutics segment. This is due to an increasing number of drugs or therapeutics offered to their end users.

Segmentation 2: by Application Oncology Infectious Diseases Neurology Endocrinology Cardiology Gastroenterology Others

The Middle East precision medicine market is dominated by the oncology segment owing to an increasing number of patients suffering from cancer. According to the data published by World Health Organization, cancer is a leading cause of deaths, with nearly 10 million deaths reported in 2020.

Segmentation 3: by End User Biopharmaceutical Companies Diagnostic Companies Others

Segmentation 4: by Region Middle East - K.S.A., Israel, Egypt, United Arab Emirates (U.A.E.), Iran, Qatar, Other Countries

Kingdom of Saudi Arabia (K.S.A.) generated the highest revenue of $1,059.7 million in 2021, which is attributed to the R&D advancements in the field of single-cell analysis and the presence of dominating players operating in the precision medicine market.

Recent Developments in Middle East Precision Medicine Market

In October 2020, Bio-Rad Laboratories launched the CFX Opus 96 Dx System and CFX Opus 384 Dx System. The product can multiplex near about five samples to offer effective in-vitro diagnostics (IVD) assay development and testing. The product has been commercialized in the Middle East region. In March 2021, F. Hoffmann-La Roche Ltd released the AVENIO Tumor Tissue CGP Kit, which enables laboratories to extend their oncology research in-house. In June 2019, F. Hoffmann-La Roche Ltd partnered with the Health Authority and an international health insurer, AXA, in Dubai. This partnership would develop funding for diagnostic and treatment for breast, colorectal, and cervical cancers.

Demand Drivers and Limitations

Following are the demand drivers for Middle East precision medicine market: Advancement of Sequencing Technologies Rising Prevalence of Chronic Diseases Shifting the Significance in Medicine from Reaction to Prevention Reducing Adverse Drug Reactions through Pharmacogenomics Test Potential to Reduce the Overall Healthcare Cost Across the Globe

The market is expected to face some limitations too due to the following challenges: Unified Framework for Data Integration Limited Knowledge about Molecular Mechanism/Interaction Lack of Robust Reimbursement Landscape

How Can This Report Add Value to an Organization?

Ecosystem/Innovation Strategy: The ecosystem segment helps the reader understand the four types of products, i.e., applied sciences, precision therapeutics, digital health and information technology, and precision diagnostics. These ecosystems are the major focus of the study as these are the target of market players in terms of revenue generation. Moreover, the study provides the reader with a detailed understanding of the different applications such as oncology, immunology, NIPT, microbiology, and others.

Growth/Marketing Strategy: The Middle East precision medicine market has been dominated significantly by companies such as QIAGEN N.V., PerkinElmer, Inc., F. Thermo Fisher Scientific Inc, and Hoffmann-La Roche Ltd., due to their expansive portfolio and strong presence across the world. The following figure represents the growth-share matrix for some of the major players in the Middle East precision medicine market based on their activities till the end of 2020. Many companies such as Agilent Technologies, Inc., Thermo Fisher Scientific Inc., PerkinElmer, Inc., Beckman Coulter, Inc. (Danaher Corporation), Illumina, Inc., Norgen Biotek Corp., and Omega Bio-tek, Inc. lie in the low growth and low market share segment. Most of the key market players in the Middle East precision medicine market are categorized under the low growth and low market share segment. The low market share of these companies is primarily due to limited products with respect to the Middle East precision medicine market in comparison to other segments of these companies. Also, the lack of synergistic activities with respect to the market is responsible for the low growth of these companies.

Competitive Strategy: Key players in the Middle East precision medicine market analyzed and profiled in the study have involved the precision medicine application-based product manufacturers that provide precision therapeutics and diagnostics.Moreover, a detailed competitive benchmarking of the players operating in the Middle East precision medicine market has been done to help the reader understand how players stack against each other, presenting a clear market landscape.

Additionally, comprehensive competitive strategies such as partnerships, agreements, and collaborations will aid the reader in understanding the untapped revenue pockets in the market.

Key Market Players and Competition Synopsis

The companies that are profiled have been selected based on inputs gathered from primary experts and analyzing company coverage, product portfolio, and market penetration.

Some of the prominent names established in this market are: Abbott Bio-Rad Laboratories, Inc. ASURAGEN, INC. bioMrieux SA Diginova Health Solutions Ltd. F. Hoffmann-La Roche Ltd Diginova Health Solutions Ltd. GlaxoSmithKline plc. Illumina, Inc. Intomics Merck KGaA Novartis AG Thermo Fisher Scientific Inc.

Companies that are not a part of the above-mentioned pool have been well represented across different sections of the report (wherever applicable).

Countries Covered K.S.A. Israel U.A.E. Egypt Iran QatarRead the full report: https://www.reportlinker.com/p06277485/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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Middle-East precision medicine market was estimated to be at $3,942.6 million in 2021, which is expected to grow with a CAGR of 7.37% and reach...

First Study from the CANbridge UMass Chan Medical School Rare Disease Gene Therapy Research Collaboration

BEIJING & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- CANbridge Pharmaceuticals Inc., (CANbridge,1228.HK) a leading China-based global biopharmaceutical company committed to the research, development and commercialization of transformative rare disease and rare oncology therapies, announced that data from its gene therapy research agreement with the Horae Gene Therapy Center, at the UMass Chan Medical School, will be presented at the ASGCT 25th Annual Meeting, being held in Washington D.C. from May 16 - 19th. Researchers will present findings from an animal study exploring a novel second-generation gene therapy for the potential treatment of spinal muscular atrophy (SMA). This is the first data to come out of the CANbridge rare disease gene therapy research collaboration with the UMass Chan Medical School, which is focused on developing gene therapy treatments for neuromuscular conditions with unmet medical needs.

Presentation Details:

Title: Endogenous Human SMN1 Promoter-driven Gene Replacement Improves the Efficacy and Safety of AAV9-mediated Gene Therapy for Spinal Muscular Atrophy in Mice

Poster #:M-144

Category: Neurologic Diseases I

Session Date and Time: Monday, May 16, 5:30-6:30 PMAuthors: Qing Xie, Hong Ma, Xiupeng Chen, Yunxiang Zhu, Yijie Ma, Leila Jalinous, Qin Su, Phillip Tai, Guangping Gao, Jun Xie

Abstracts are available on the ASGCT website: https://annualmeeting.asgct.org

About the Horae Gene Therapy Center at UMass Chan Medical School

The faculty of the Horae Gene Therapy Center is dedicated to developing therapeutic approaches for rare inherited disease for which there is no cure. We utilize state of the art technologies to either genetically modulate mutated genes that produce disease-causing proteins or introduce a healthy copy of a gene if the mutation results in a non-functional protein.

The Horae Gene Therapy Center faculty is interdisciplinary, including members from the departments of Pediatrics, Microbiology & Physiological Systems, Biochemistry & Molecular Pharmacology, Neurology, Medicine and Ophthalmology. Physicians and PhDs work together to address the medical needs of rare diseases, such as Alpha 1-Antitrypsin Deficiency, Canavan Disease, Tay-Sachs and Sandhoff diseases, Retinitis Pigmentosa, Cystic fibrosis, Lou Gehrig's disease, TNNT1 nemaline myopathy, Rett syndrome, N-Gly 1 deficiency, Pitt-Hopkins syndrome, Marple Syrup Urine Disease, Sialidosis, GM3 synthase deficiency, Huntington's disease, ALS and others. More common diseases such as cardiac arrhythmia and hypercholesterolemia are also investigated. The hope is to treat a wide spectrum of diseases by various gene therapeutic approaches. Additionally, the University of Massachusetts Chan Medical School conducts clinical trials on site and some of these trials are conducted by the investigators at the Gene Therapy center.

About CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. (CANbridge, 1228.HK) is a China-based global rare disease-focused biopharmaceutical company committed to the research, development and commercialization of transformative rare disease and rare oncology therapies.

CANbridge has a comprehensive and differentiated pipeline of 13 drug assets with significant market potential, targeting some of the most prevalent rare diseases and rare oncology.

These include Hunter syndrome (MPS II) and other lysosomal storage disorders (LSDs), complement mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases including Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA) as well as glioblastoma multiforme (GBM).

CANbridge strategically combines global collaborations and internal research to build and diversify its drug portfolio and invest in next-generation gene therapy technologies for rare disease treatments. CANbridge global partners include, but are not limited to, Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, the University of Massachusetts Chan Medical School, the University of Washington School of Medicine, LogicBio and Scriptr.

For more on CANbridge Pharmaceuticals Inc., please go to: http://www.canbridgepharma.com.

Forward-Looking Statements

The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, after the data on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220503005199/en/

Contacts

U.S. Investor Relations: Chris BrinzeyICR WestwickeChris.brinzey@westwicke.com

China Investor Relations CANbridge Pharmaceuticals Inc.ir@canbridgepharma.com

Media: Deanne EaglePlanet Communicationsdeanne@planetcommunications.nyc 917.837.5866

Source: CANbridge Pharmaceuticals Inc.

View this news release online at:http://www.businesswire.com/news/home/20220503005199/en

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CANbridge-UMass Chan Medical School Gene Therapy Research to be Presented at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting -...