Category Archives: Gene Medicine

Boston, MA. May 4, 2022 -- Mass General Brigham today revealed the annual Disruptive Dozen, 12 emerging gene and cell therapy (GCT) technologies with the greatest potential to impact health care in the next several years. The Disruptive Dozen results from interviews of 50 Mass General Brigham senior Harvard faculty followed by a rigorous selection process to identify the 12 most likely to have significant impact on patient care in the next few years.

GCT is widely recognized as a transformational opportunity in medicine, with the potential to stop or slow the effects of disease by targeting it at the genetic level. The Disruptive Dozen were featured as part of this weeks World Medical Innovation Forum in Boston, where clinical experts, industry leaders and venture investors explored how to advance GCT technologies to benefit patients worldwide. The Forum is presented by Mass General Brigham and Bank of America, bringing together two leading organizations with extensive healthcare expertise to advance medical breakthroughs for patients and support Bostons continued growth as a global biotech and investment hub.

The 2022 Mass General Brigham Disruptive Dozen are:

The culture of innovation at Mass General Brigham fosters continuous discussion and debate about emerging technologies and which ones will have the biggest impact for patients, said Ravi Thadhani, MD, Chief Academic Officer, Mass General Brigham. The Disruptive Dozen reflects this spirit of discovery and underpins our work to identify what has the potential to significantly improve health care.

Mass General Brigham is the nations largest academic research enterprise. More than 150 life science and biotechnology companies have been established in Massachusetts as a result of the more than$2 billion in government funded and privately sponsored research that Mass General Brigham attracts every year. The groundbreaking research performed at Mass General Brigham is integral to developing and commercializing life-changing therapies which sustains Massachusetts competitive advantage in the innovation economy.

Following are descriptions of the 2022 Disruptive Dozen:

1. Restoring sight by mending broken genes

Roughly 200 genes are directly linked to vision disorders. In the last several years, groundbreaking new gene therapies have emerged that can compensate for faulty genes in the eye by adding new, healthy copies a molecular fix that promises to restore sight to those who have lost it. The approach, known as CRISPR-Cas-9 gene editing, could open the door to treating genetic forms of vision loss that are not suited to conventional gene therapy, and a host of other medical conditions. A clinical trial is now underway to evaluate a CRISPR-Cas 9 gene-editing therapy for a severe form of childhood blindness, Leber congenital amaurosis type 10, for which there currently are no treatments. Although this treatment is still experimental, it is already historic it is the first medicine based on CRISPR-Cas-9 to be delivered in vivo, or inside a patients body. Similar gene-editing therapies are also under development that correct genes within blood cells.

2. A gene editing solution to increase the supply of donor organs

In the U.S. alone, more than 100,000 people await a life-saving organ transplant. But an inadequate supply of donor organs leads to prolonged waiting times and many patients die before receiving a life-saving organ. The only foreseeable solution to this crisis is xenotransplantation harvesting organs from animals and placing them into human patients. Advances in gene editing technology make it possible to remove, insert, or replace genes with relative ease and precision. This molecular engineering can sidestep the human immune system, which is highly adept at recognizing foreign tissues and triggering rejection. Over the last 20 years, scientists have been working to devise successful gene editing strategies that will render pig organs compatible with humans. The field has taken another major step forward in the past year: transplanting gene-edited pig organs, including the heart and kidney, into humans. While extensive clinical testing is needed before xenotransplantation becomes a reality, that future now seems within reach.

3. Cell therapies to conquer common forms of blindness

The eye has been a proving ground for pioneering gene therapies and is also fueling new cell-based therapies that can restore sight, offering a functional cure by replacing critical cells that have been lost or injured. One approach involves stem cells from the retina that can give rise to light-sensitive cells, called photoreceptors, which are required for healthy vision. Scientists are harnessing retinal stem cells to develop treatments for incurable eye diseases, including retinitis pigmentosa. Because the immune system doesnt patrol the eye as aggressively as other parts of the body, retinal stem cells from unrelated, healthy donors can be transplanted into patients with vision disorders. Other progress includes cell therapies that harness patients own cells, for example, from blood or skin, that can be converted into almost any cell type in the body, including retinal cells. Another novel treatment being tested utilizes stem cells from a patients healthy eye to repair the affected cornea of the other eye.

4. Harnessing the power of RNA to treat brain cancer

RNA is widely known for its helper functions, carrying messages from one part of a cell to another to make proteins. But scientists now recognize that RNA plays a more central role in biology and are tapping its hidden potential to create potent new therapies for a range of diseases, including a devastating form of brain cancer called glioblastoma. This cancer is extremely challenging to treat and highly adaptable. New approaches that either target RNA or mimic its activity could hold promise, including an intriguing class of RNA molecules called microRNAs. One team identified a trio of microRNAs that plays important roles in healthy neurons but is lost when brain cancer develops. These microRNAs can be stitched together into a single unit and delivered into the brain using a virus. Initial studies in mice reveal that this therapeutic can render tumors more vulnerable to existing treatments, including chemotherapy. Another team is also exploring a microRNA called miR-10b. Blocking its activity causes tumor cells to die. Now, scientists are working to develop a targeted therapeutic against miR-10b that can be tested in clinical trials.

5. Realizing the promise of gene therapy for brain disorders

Gene therapy holds enormous promise for serious and currently untreatable diseases, including those of the brain and spinal cord. But some big obstacles remain. For example, a commonly-used vehicle for gene therapy a virus called AAV cannot penetrate a major biological roadblock, the blood-brain barrier. Now, researchers are engineering new versions of AAV that can cross the blood-brain barrier. Using various molecular strategies, a handful of teams have modified the protein shell that surrounds the virus so it can gain entry and become broadly distributed within the brain. These modified viral vectors are now under development and could begin clinical testing within a few years. Scientists are also tinkering with the inner machinery of AAV to sidestep potential toxicities. With a safe, effective method for accessing the brain, researchers will be able to devise gene therapies for a range of neurological conditions, including neurodegenerative diseases, cancers, and devastating rare diseases that lack any treatment.

6. A flexible, programmable approach to fighting viruses

The COVID-19 pandemic has laid bare the tremendous need for rapidly deployable therapies to counteract emerging viruses. Scientists are now developing a novel form of anti-viral therapy that can be programmed to target a range of different viruses from well-known human pathogens, such as hepatitis C, to those less familiar, such as the novel coronavirus SARS-CoV-2. This new approach harnesses a popular family of gene editing tools, known as CRISPR-Cas. While CRISPR-based systems have gained attention for their capacity to modify human genes, their original purpose in nature was to defend bacteria from viral infections. As a throwback to these early roots, scientists are now adapting CRISPR tools to tackle a variety of viruses that infect humans. Researchers are studying the potential of these programmable anti-viral agents in the context of several different viruses, including ones that pose significant threats to global health, such as SARS-CoV-2, hepatitis C, and HIV.

7. On the move: Cell therapies to restore gut motility

The human digestive tract or gut has its own nervous system. This second brain, known as the enteric nervous system, is comprised of neurons and support cells that carry out critical tasks, like moving food through the gut. When enteric neurons are missing or injured, gut motility can be impaired. Now, scientists are developing an innovative cell replacement therapy to treat diseases of gut motility. Donor cells can be isolated from a patients own gut or from a more readily available source, such as subcutaneous fat. These cells are then cultivated in the laboratory and coaxed to form the progenitors that give rise to enteric neurons. Researchers are also devising off-the-shelf approaches, which could create a supply of donor cells that are shielded from the immune system and can therefore be transplanted universally across different patients. Early research shows that transplanted enteric neurons can also take up residence in the brain. That means these forays in cell therapy for the gut could also help pave a path toward cell therapies for the brain and spinal cord.

8. CAR-T cell therapies take aim at autoimmune diseases

CAR-T cells have emerged as powerful treatments for some forms of cancer, especially blood cancers. By harnessing the same underlying concept rewiring patients own T cells to endow them with therapeutic properties scientists are working to develop novel CAR-T therapies for a variety of autoimmune diseases. Several research teams are engineering CAR-T cells so they can seek out and destroy harmful immune cells, such as those that produce auto-antibodies immune proteins that target and attack the bodys own tissues. For example, one team is using CAR-T cells to destroy certain immune cells, called B cells, as a potential treatment for lupus, a serious autoimmune disease that mainly affects women. Scientists are also developing CAR-T therapies that take aim at other rogue members of the immune system. These efforts could yield novel treatments for diseases with clear auto-immune mechanisms.

9. Regrowing cells in the inner ear to treat hearing loss

In the U.S. alone, some 37 million people suffer from a hearing deficit. Currently, there are no drugs that can halt, prevent, or even reverse hearing loss. Scientists are working on a novel regenerative approach that could restore the cells in the inner ear required for normal hearing, offering hope to millions of patients who grapple with hearing loss. Healthy hearing requires specialized cells in the inner ear called hair cells, which have fine, hair-like projections. If the cells are damaged or lost, which often happens with age or after repeated exposure to loud sounds, the body cannot repair them. But researchers have discovered a potential workaround that can stimulate existing cells in the ear to be converted and give rise to new hair cells. Scientists are now working to convert this molecular strategy, which is being studied in animal models, into a therapeutic that is safe and effective for hearing loss patients.

10. New technologies for delivering gene therapies

A formidable challenge in the field of gene therapy is delivery getting gene-based therapeutics into the body and into the right target cells. Researchers are exploring the potential of new delivery methods that could expand the reach of gene therapy, including microneedles. When applied to the skin, a microneedle patch can penetrate the outermost layer with minimal pain and discomfort. This novel delivery method can readily access the legion of immune cells that reside in the skin -- important targets for vaccines as well as for the treatment of various diseases, including cancer and autoimmune conditions. Another emerging technology involves an implantable device made of biodegradable materials. When placed inside the body, this device can provide localized, sustained release of therapeutics with few side effects. The approach is now being tested for the first time in cancer patients using standard chemotherapy drugs administered directly at tumor sites. In the future, this method could be customized for the delivery of gene therapy payloads, an advance that could revolutionize cancer treatment, particularly for difficult-to-treat tumors like pancreatic cancer.

11. Engineering cancer-killing cells that target solid tumors

Despite great leaps in cancer treatment, solid tumors remain the most challenging tumors to treat, in part due to the hostile environment in which they grow, which suppresses the immune system. Now, scientists are devising innovative cell therapies that promise to open new therapeutic opportunities for solid tumors. One approach involves making CAR-T cells more like computers, relying on simple logic to decide which cells are cancer and which are not. By building several logic gates and combining them together, researchers are hoping to pave the way toward targeting new tumor types. Scientists are also devising other groundbreaking forms of cancer-killing cell therapy, including one that uses cancer cells themselves. This approach exploits a remarkable feature: once disseminated within the body, cancer cells can migrate back to the original tumor. Researchers are now harnessing this rehoming capability and, with the help of gene editing and other molecular engineering technologies, turning tumor cells into potent cancer killers. An early version of this technology uses patients own cells. Now, the scientists are developing an off-the-shelf version with dual properties killing cancer cells and modulating the immune system that can be universally applied to patients.

12. Reawakening the X-chromosome: a therapeutic strategy for devastating neurodevelopmental diseases

The X chromosome is one of two sex-determining chromosomes in humans, and it carries hundreds of disease-causing genes. These diseases often affect males and females differently. In females, one X chromosome is naturally, and randomly, chosen and rendered inactive. Although X-inactivation was once thought to be permanent, scientists are uncovering ways to reverse it. Scientists are now exploiting this unusual biology to reawaken the dormant X chromosome a strategy that could yield much-needed treatments for a group of rare, yet devastating neurodevelopmental disorders, which predominantly affect females. This new approach could hold promise for females with Rett syndrome, a severe X-linked disorder. A similar strategy could also hold promise for other serious X-linked disorders, including fragile X syndrome and CDKL5 syndrome.

Click here for detailed information on each of the Disruptive Dozen technologies, including video updates. Click here for more information on gene and cell therapy at Mass General Brigham.

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The World Medical Innovation Forum was established in 2015 in response to the intensifying transformation of health care and its impact on innovation. The Forum is rooted in the belief that no matter the magnitude of change, the center of health care needs to be a shared, fundamental commitment to collaborative innovation industry and academia working together to improve patient lives.In 2022, Bank of America joined with Mass General Brigham as presenting sponsor of the Forum, bringing together two leading organizations with extensive healthcare expertise and a shared commitment to support Bostons continued growth as a global biotech and investment hub.

Mass General Brigham is an integrated academic healthcare system, uniting great minds in medicine to make life-changing impact for patients in our communities and people around the world. Mass General Brigham connects a full continuum of care across a system of academic medical centers, community and specialty hospitals, a health insurance plan, physician networks, community health centers, home care, and long-term care services. Mass General Brigham is a non-profit organization that is committed to patient care, research, teaching, and service to the community. In addition, Mass General Brigham is one of the nations leading biomedical research organizations and a principal teaching affiliate of Harvard Medical School. For more information, please visitmassgeneralbrigham.org.

Bank of America is one of the worlds leading financial institutions, serving individual consumers, small and middle-market businesses and large corporations with a full range of banking, investing, asset management and other financial and risk management products and services. The company provides unmatched convenience in the United States, serving approximately 66 million consumer and small business clients with approximately 4,300 retail financial centers, approximately 17,000 ATMs, and award-winning digital banking with approximately 41 million active users, including approximately 32 million mobile users. Bank of America is a global leader in wealth management, corporate and investment banking and trading across a broad range of asset classes, serving corporations, governments, institutions and individuals around the world. Bank of America offers industry-leading support to approximately 3 million small business households through a suite of innovative, easy-to-use online products and services. The company serves clients through operations across the United States, its territories and approximately 35 countries. Bank of America Corporation stock (NYSE: BAC) is listed on the New York Stock Exchange.www.bankofamerica.com

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Top 12 emerging gene and cell therapy technologies likely to impact patient care announced in annual Disruptive Dozen from Mass General Brigham -...

Company continues to optimize cell-targeted lipid nanoparticle (ctLNP) delivery system for nonviral genetic medicine applications in liver, retina and vaccines

Cash balance of $337.0M expected to fund operations into 2024

CAMBRIDGE, Mass., May 05, 2022 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq: GBIO), a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, reported business highlights and first quarter 2022 financial results.

We are making steady advances in optimizing our cell-targeted lipid nanoparticles, or ctLNPs, for use in our liver and retina programs as well as in developing our platform technologies for novel vaccine applications, said Geoff McDonough, M.D., president and chief executive officer of Generation Bio. We believe our approach to genetic medicine has the potential to create highly differentiated therapies capable of fulfilling significant unmet need, and we look forward to providing updates on our progress.

Key priorities across core therapeutic areas:

First Quarter 2022 Financial Results

About Generation Bio

Generation Bio is innovating genetic medicines to provide durable, redosable treatments for people living with rare and prevalent diseases. The companys non-viral genetic medicine platform incorporates a novel DNA construct called closed-ended DNA, or ceDNA; a unique cell-targeted lipid nanoparticle delivery system, or ctLNP; and a highly scalable capsid-free manufacturing process that uses proprietary cell-free rapid enzymatic synthesis, or RES, to produce ceDNA. The platform is designed to enable multi-year durability from a single dose, to deliver large genetic payloads, including multiple genes, to specific cell types, and to allow titration and redosing to adjust or extend expression levels in each patient. RES has the potential to expand Generation Bios manufacturing scale to hundreds of millions of doses to support its mission to extend the reach of genetic medicine to more people, living with more diseases, around the world.

For more information, please visitwww.generationbio.com.

Forward-Looking Statements

Any statements in this press release about future expectations, plans and prospects for the company, including statements about our strategic plans or objectives, our technology platform, our research and clinical development plans, applications and preclinical data and other statements containing the words believes, anticipates, plans, expects, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the conduct of research activities, the initiation and completion of preclinical studies and clinical trials and clinical development of the companys product candidates; uncertainties as to the availability and timing of results from preclinical studies and clinical trials; whether results from earlier preclinical studies will be predictive of the results of later preclinical studies and clinical trials; uncertainties regarding the timing and ability to complete the build-out of the companys manufacturing facility and regarding the RES manufacturing process; challenges in the manufacture of genetic medicine products; whether the companys cash resources are sufficient to fund the companys operating expenses and capital expenditure requirements for the period anticipated; the impact of the COVID-19 pandemic on the companys business and operations; expectations for regulatory approvals to conduct trials or to market products; as well as the other risks and uncertainties set forth in the Risk Factors section of our most recent annual report on Form 10-K, which is on file with the Securities and Exchange Commission, and in subsequent filings the company may make with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the companys views as of the date hereof. The company anticipates that subsequent events and developments will cause the companys views to change.However, while the company may elect to update these forward-looking statements at some point in the future, the company specifically disclaims any obligation to do so.These forward-looking statements should not be relied upon as representing the companys views as of any date subsequent to the date on which they were made.

Investors and Media ContactMaren KillackeyGeneration Bio857-371-4638mkillackey@generationbio.com

GENERATION BIO CO.CONSOLIDATED BALANCE SHEET DATA (Unaudited)(In thousands)

GENERATION BIO CO.CONSOLIDATED STATEMENTS OF OPERATIONS (Unaudited)(in thousands, except share and per share data)

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Generation Bio Reports Business Highlights and First Quarter 2022 Financial Results - GlobeNewswire

Ongoing Clinical Development Program Represents Largest and Longest Development Program for any Gene Therapy in Hemophilia A, Demonstrates Commitment to Advancing Care for People with Hemophilia A

SAN RAFAEL, Calif., May 5, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced five platform presentations and one poster presentation on valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, at the World Federation of Hemophilia (WFH) 2022 World Congress from May 8-11 in Montreal, Canada.

BioMarin Pharmaceutical logo (PRNewsfoto/BioMarin Pharmaceutical Inc.)

"We're pleased to provide updated data on durability of effect in patients treated over two years ago, on molecular contributors to variability, on hepatotoxicity and the role of immunosuppression, on the potential for integration-related oncogenicity, and on health-related quality of life. We continue to learn about the potential for investigational valoctocogene roxaparvovec to transform lives and the optimal way to manage patients through their journey," said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. "We are progressing our regulatory efforts with the intent to deliver a therapy that may represent an important and valuable treatment choice for patients with severe Hemophilia A, as no presently available treatment offers such freedom from prophylaxis and reduced bleeding frequency at the same time."

"BioMarin is generating valuable data about investigational valoctocogene roxaparvovec that potentially could enable shared decision making between physicians and patients on what is the best therapy in each personal situation," said Professor Wolfgang Miesbach, Head of the Department of Coagulation Disorders and the Comprehensive Care Haemophilia Centre at the Goethe University Hospital in Frankfurt/Main, Germany.

Presentation of data at WFH follows positive two-year results from the ongoing, global phase 3 GENEr8-1 study of valoctocogene roxaparvovec presented at a medical meeting earlier in the year, as well as publication of one-year results from the pivotal clinical trial in the New England Journal of Medicine in March 2022.

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BioMarin's presentations at WFH include:

Platform Presentations

Immune suppression following gene therapy in HemophiliaProfessor Wolfgang Miesbach, Head of the Department of Coagulation Disorders and the Comprehensive Care Haemophilia Centre, Goethe University Hospital, Frankfurt/Main, GermanyMonday, May 9, 2022, 1:30 -2:30 PM ET

Exploratory analyses of healthy liver biopsies and a single case of parotid acinar cell carcinoma do not identify a role for valoctocogene roxaparvovec vector insertion in altering cell growthKevin Eggan, Group Vice President, Head of Research and Early Development / BioMarinMonday, May 9, 2022, 1:30 -2:30 PM ET

Health-related quality of life over 2 years following valoctocogene roxaparvovec adeno-associated virus gene transfer for severe hemophilia A: Results from GENEr8-1Dr. Amy Dunn, Director of Pediatric Hematology, Nationwide Children's Hospital, Columbus, OhioTuesday, May 10, 2022, 1:30-2:30 PM ET

Human liver biopsy analysis showed interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A Sylvia Fong, Head of Hematology Research, BioMarinWednesday, May 11, 2022, 1:30 -2:30 PM ET

Interim 52-week analysis of immunogenicity to the vector capsid and transgene-expressed human FVIII in GENEr8-1, a phase 3 clinical study of valoctocogene roxaparvovec, an AAV5-medicated gene therapy for hemophilia A Brian Long, Principal Scientist, Clinical Immunology, BioMarinWednesday, May 11, 2022, 4-5 PM ET

Poster Presentation

Use of immunosuppressives in patients with hemophilia receiving gene therapy: Evidence generation using a mixed-methods approach Professor Wolfgang Miesbach, Head of the Department of Coagulation Disorders and the Comprehensive Care Haemophilia Centre, Goethe University Hospital, Frankfurt/Main, Germany

BioMarin-Sponsored Symposia

Gene Therapy Clinical Trial Patient Journey: A Look Into Shared Decision MakingMonday, May 9, 12:15 1:15 PM ET

About Hemophilia A

People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A (FVIII levels <1%) often experience painful, spontaneous bleeds into their muscles or joints. Individuals with the most severe form of hemophilia A make up approximately 50 percent of the hemophilia A population. People with hemophilia A with moderate (FVIII 1-5%) or mild (FVIII 5-40%) disease show a much-reduced propensity to bleed. The standard of care for individuals with severe hemophilia A is a prophylactic regimen of replacement Factor VIII infusions administered intravenously up to two to three times per week or 100 to 150 infusions per year. Despite these regimens, many people continue to experience breakthrough bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life.

Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have Hemophilia A.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare diseases and medical conditions. The Company selects product candidates for diseases and conditions that represent a significant unmet medical need, have well-understood biology and provide an opportunity to be first-to-market or offer a significant benefit over existing products. The Company's portfolio consists of seven commercial products and multiple clinical and preclinical product candidates for the treatment of various diseases. For additional information, please visit http://www.biomarin.com.

Forward Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including without limitation, statements about: the data presented at WFH, including the five platform presentations, one poster, and one BioMarin sponsored symposia, the development of BioMarin's valoctocogene roxaparvovec program generally, the impact of valoctocogene roxaparvovec gene therapy for treating patients with severe hemophilia A and the potential to transform the lives of these patients and the ongoing clinical programs generally. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned preclinical studies and clinical trials of valoctocogene roxaparvovec, including final analysis of the above data and additional data from the continuation of these trials and the entire development program, including further assessment of safety events, any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the FDA, the EMA and other regulatory authorities; the content and timing of decisions by local and central ethics committees regarding the clinical trials; our ability to successfully manufacture valoctocogene roxaparvovec; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended March 31, 2022 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc.

Contacts:

Investors

Media

Traci McCarty

Debra Charlesworth

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

(415) 455-7451

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BioMarin to Present Findings from Ongoing Studies of Valoctocogene Roxaparvovec, Investigational Gene Therapy, at the World Federation of Hemophilia...

Athenex, Inc.

Data presented by investigators from the Texas Childrens Cancer Centers Center for Advanced Innate Cell Therapy and Baylor College of Medicine

Data demonstrate expansion of CAR-NKT cells post-transfer in all patients, and objective responses in patients with relapsed/refractory neuroblastoma

Evidence of therapeutic efficacy with 25% (3/12) Overall Response Rate (ORR), 58% (4 stable disease, 2 partial responses, 1 complete response/12) Disease Control Rate (DCR), and 2 out of 3 responses at dose level 4 (1x108/m2)

Durable Complete Response persisting 12 months

Analysis found that responders correlate with CD62L+ NKT frequency in the pre-infusion products as well as CAR-NKT area under the curve (AUC) in the peripheral blood post-infusion

Well tolerated with no dose limiting toxicity; no immune associated neurotoxicity syndrome (ICANS) at the first four dose levels and one case of grade 2 CRS

BUFFALO, N.Y., May 02, 2022 (GLOBE NEWSWIRE) -- Athenex (NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies for the treatment of cancer and related conditions, today announced that the Company will present at the upcoming ASGCT 25th Annual Meeting, taking place May 16-19, 2022, in Washington, D.C. The abstract associated with the presentation is now available on the conference website.

Details of the presentations are as follows:

Oral Presentation Details

Abstract 54: Anti-GD2 CAR NKT cells are safe and produce antitumor responses in patients with relapsed/refractory neuroblastomaDate/Time: Monday, May 16, 2022, 2:30 PM 2:45 PM ETSession: Gene and Cell Therapy Trials in ProgressPresenter: Dr. Andras Heczey, Baylor College of MedicineLocation: Room 201, Walter E. Washington Convention Center, 801 Mt Vernon Pl NW Washington, D.C.

Observing evidence of a dose response is extremely encouraging, and we are excited to see two out of three responses at 100 million cells/m2, said Dan Lang, M.D., President of Athenex Cell Therapy. We are hopeful that we will see additional responses as we move to higher doses. We and our partners at Baylor College of Medicine (BCM) and Texas Childrens Hospital (TCH) continue to be motivated by the data and look forward to our next update.

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About KUR-501

KUR-501, is an autologous NKT cell CAR product that targets GD2, a tumor-associated antigen that is expressed on almost all neuroblastomas as well as melanomas, some sarcomas, and a variety of other tumors. GINAKIT2, a phase 1 single arm clinical study (NCT03294954) of KUR-501, in patients with refractory or recurrent high-risk neuroblastoma will evaluate up to six dose levels of KUR-501 following lymphodepletion with cyclophosphamide and fludarabine.

Neuroblastoma, a pediatric cancer of the sympathetic nervous system, typically the adrenal gland, is associated with a poor prognosis in children with high-risk disease and is a significant unmet medical need. The KUR-501 development program will provide autologous proof-of-concept for CAR-NKT cells in solid tumors using a validated target.

The GINAKIT2 study is supported by Athenex, Inc., which acquired Kuur Therapeutics in May 2021, as well as by Alexs Lemonade Stand Foundation. This study is currently recruiting patients at BCM/TCH.

About Athenex, Inc.

Founded in 2003, Athenex, Inc. is a global clinical-stage biopharmaceutical company dedicated to becoming a leader in the discovery, development, and commercialization of next generation cell therapy drugs for the treatment of cancer. In pursuit of this mission, Athenex leverages years of experience in research and development, clinical trials, regulatory standards, and manufacturing. The Companys current clinical pipeline is derived mainly from the following core technologies: (1) Cell therapy based on NKT cells, (2) Orascovery, based on a P-glycoprotein inhibitor, and (3) Src Kinase Inhibition. Athenexs employees worldwide are dedicated to improving the lives of cancer patients by creating more active, accessible and tolerable treatments. For more information, please visit http://www.athenex.com.

Forward-Looking Statements

Except for historical information, all of the statements, expectations, and assumptions contained in this press release are forward-looking statements. These forward-looking statements are typically identified by terms such as believe, look forward to, potential, and similar expressions. Actual results might differ materially from those explicit or implicit in the forward-looking statements. Important factors that could cause actual results to differ materially include: our history of operating losses and the substantial doubt about our ability to continue as a going concern; our strategic pivot to focus on our cell therapy platform and our plan to dispose of non-core assets; our ability to obtain financing to fund operations, successfully redirect our resources and reduce our operating expenses; our ability to refinance, extend or repay our substantial indebtedness owed to our senior secured lender; the development stage of our primary clinical candidates, including NKT Cell Therapy and related risks involved in drug development, clinical trials, regulation, uncertainties around regulatory reviews and approvals; the preclinical and clinical results for Athenexs drug candidates, which may not support further development of such drug candidates; the Companys ability to successfully demonstrate the safety and efficacy of its drug candidates and gain approval of its drug candidates on a timely basis, if at all; the uncertainty of ongoing legal proceedings; risks related to our ability to successfully integrate the business of Kuur into our existing businesses, including uncertainties associated with maintaining relationships with customers, vendors and employees, as well as differences in operations, cultures, and management philosophies that may delay successful integration and our ability to support the added cost burden of Kuurs business; risks related to counterparty performance, including our reliance on third parties for success in certain areas of Athenexs business; risks and uncertainties inherent in litigation, including purported stockholder class actions; the impact of the COVID-19 pandemic and other macroeconomic factors, like the war in Ukraine, and their ongoing impact on our operations, supply chain, cash flow and financial condition; competition; intellectual property risks; risks relating to doing business internationally and in China; the risk of development, operational delays, production slowdowns or stoppages or other interruptions at our manufacturing facility as well as our ability to find alternative sources of supply to meet our obligations and requirements; and the other risk factors set forth from time to time in our SEC filings, copies of which are available for free in the Investor Relations section of our website at http://ir.athenex.com/phoenix.zhtml?c=254495&p=irol-sec or upon request from our Investor Relations Department. All information provided in this release is as of the date hereof and we assume no obligation and do not intend to update these forward-looking statements, except as required by law.

Athenex Contacts

Investors

Daniel Lang, MDAthenex, Inc.Email: danlang@athenex.com

Caileigh DoughertyAthenex, Inc.Email: cdougherty@athenex.com

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Athenex, Texas Childrens Cancer Center, and the Center for Cell and Gene Therapy at Baylor College of Medicine to Present Phase 1 Clinical Data for...

The version used in Maryland came from a pig with 10 gene modifications developed by Revivicor, a subsidiary of United Therapeutics.

Following promising tests of such pig organs in baboons, three US transplant teams launched the first human studies starting in late 2021. Surgeons at New York University and the University of Alabama each attached pig kidneys to brain-dead people, but the University of Maryland went a step further when Griffith stitched a pig heart into Bennetts chest in early January.

Transferring pig viruses to humans has been a worrysome fear xenotransplantation could set off a pandemic if a virus were to adapt inside a patients body and then spread to doctors and nurses. The concern could be serious enough to require lifelong monitoring for patients.

However, the specific type of virus found in Bennetts donor heart is not believed capable of infecting human cells, says Jay Fishman, a specialist in transplant infections at Massachusetts General Hospital. Fishman thinks there is no real risk to humans of its spreading further.

Instead, the problem is that pig cytomegalovirus is linked to reactions that can damage the organ and the patientwith catastrophic results. Two years ago, for instance, German researchers reported that pig hearts transplanted into baboons lasted only a couple of weeks if the virus was present, while organs free from the infection could survive more than half a year.

Those researchers said they found astonishingly high virus levels in pig hearts removed from baboons. They think the virus could go haywire not just because the baboons immune systems were suppressed with drugs, but also because the pig immune system was no longer there to keep the virus in check. It seems very likely the same may happen in humans, they warned at the time.

UNIVERSITY OF MARYLAND SCHOOL OF MEDICINE

Joachim Denner of the Institute of Virology at the Free University of Berlin, who led that study, says the solution to the problem is more accurate testing. The US team appears to have tested the pigs snout for the virus, but often it is lurking deeper in the tissues.

Its a latent virus and hard to detect, says Denner. But if you test the animal better, it will not happen. The virus can be detected and easily removed from pig populations, but unfortunately they didnt use a good assay and didnt detect the virus, and this was the reason. The donor pig was infected, and the virus was transmitted by the transplant.

Denner says he still thinks the experiment was a great success. For instance, the first human-to-human heart transplant, in 1967, lasted only 18 days and, two years later, one in Germany endured just 27 hours.

Denner says that Bennetts death cannot be blamed on the virus alone. This patient was very, very, very ill. Do not forget that, he says. Maybe the virus contributed, but it was not the sole reason.

Bennetts cause of death matters, because if his heart failed as the result of immune rejection, researchers might need to return to the drawing board. Instead, its now expected that companies like United Therapeutics and eGenesis, or academics working with them, will launch clinical trials of their pig organs within a year or two.

Bennett was offered a pig heart after Griffith applied to the US Food and Drug Administration for special permission to try an animal organ in a one-off transplant. He was considered a good candidate for the daring attempt because he was nearing death from heart failure and was ineligible for a scarce human heart for transplant owing to a history of disregarding medical advice.

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The gene-edited pig heart given to a dying patient was infected with a pig virus - MIT Technology Review

As CEO of Rubius Therapeutics, Pablo Cagnoni oversees development of a type of cell therapy made with red blood cells, but he was at Novartis when that pharmaceutical giant struck the deal for the T cell research that proved to be a beacon for the cell therapy field. That CAR T therapy offered a novel way to treat some of the toughest cases of blood cancer, but Cagnoni also acknowledged the limitations of this type of treatment.

These personalized therapies made from a patients own T cells are not scalable. For all of their therapeutic benefits, CAR T still poses dangerous safety risks for patients. Also, the effectiveness of this type of treatment on liquid tumors has proven elusive in solid tumors. Now, 10 years after Novartis licensed the University of Pennsylvania CAR T technology that would become Kymriah and five years since that cell therapy won a landmark FDA approval, Cagnoni says the progress he hoped the field would make has been slower than he expected.

Those three obstacles remain, at least for CAR T cells, Cagnoni said.

Cagnoni spoke Monday as part of a panel discussion on the potential of cell and gene therapies during the World Medical Innovation Forum in Boston. While the panelists acknowledged the challenges, they also pointed to approaches that the cell and gene therapy research is taking to overcome them.

Bristol Myers Squibb is working to improve CAR T in several different ways, said Kristen Hege, the pharma giants senior vice president of early clinical development, hematology/oncology & cell therapy. The company is optimizing autologous cell therapy by automating the process and improving how these cells are engineered. BMSs development of next-generation cell therapies use gene editing techniques to knock out parts of the genome that might make a cell cause a dangerous immune response, or to knock in the properties that you would want to make it more effective as treatment, Hege said. Many of the edits for next-generation cell therapies build off of what has been learned from the first generation of CAR T treatments, she said.

Cell therapy research is going beyond T cells. Noting the encouraging early clinical data that Nktarta Therapeutics reported last week for its engineered and off-the-shelf natural killer cells, Cagnoni said that other types of cells might be able to overcome some of the challenges facing T cell-based therapies. Andrew Plump, Takeda Pharmaceuticals president of R&D, said that his companys cell therapy efforts are focused on innate immune cells. Takeda hasnt done as much gene editing research as others. But Plump said that similar to BMS, Takedas approach is to use editing approaches to improve on some property of the cell therapy, such as ensuring delivery to the right tissue.

Cell and gene therapies are opening doors to even more complex diseases with treatments, said Catherine Stehman-Breen, CEO of Chroma Medicine. These therapies also hold the potential to address a broader patient population, she added. Her Cambridge, Massachusetts-based startup is developing epigenetic medicines that treat disease by turning genes on or off. Chroma launched last fall backed by a $125 million Series A round of funding. Stehman-Breen said that Chromas approach builds on the earlier work done in cell and gene therapy research.

Plump said that Takedas approach involves partnering with small biotechs, such as Chroma, to get access to capabilities it does not have in house. Takedas partnership with bispecific T cell engager developer Maverick Therapeutics led to that biotechs acquisition last year. Plump said the deal was the product of an ongoing relationship between the companies.

Brisbane, California-based Maverick had a platform technology for making its bispecific drugs, but it did not have the capability to manufacture cell lines. Takeda provided those capabilities, leading the companies to work more closely together. The partnership, which included an equity investment, gave the pharmaceutical giant the option to acquire Maverick. Biotech company innovation in the kinds of things that Takeda does not do well in house will continue to drive the companys investments. Offering a prediction, Plump said we imagine that 50% of our clinical-stage pipeline in seven to 10 years will be in cell and gene therapy.

Image by Flickr user NIAID via a Creative Commons license

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WMIF panel: How cell and gene therapy can overcome limitations of CAR T - MedCity News

Article Highlight | 5-May-2022

With gene therapy, a cure for growth-hormone resistant dwarfism may be on the horizon.

National University of Singapore, Yong Loo Lin School of Medicine

Researchers from the NUS Yong Loo Lin School of Medicine (NUS Medicine) have shown that gene therapy using a single-dose injection of a virus carrying the good gene can potentially be used to cure growth-hormone resistant dwarfism, also known as Laron Syndrome.

People with Laron syndrome are very short as their bodies are unable to use the growth hormone, a substance that helps the body to grow. The team led by Prof Lee K O, from Department of Medicine at NUS Medicine, attempted to increase a hormone called insulin-like-growth factor 1 (IGF1) in a laboratory model by replacing the defective growth hormone receptor gene, using a virus containing instructions specifically targeted at the liver. The liver is the main organ producing the IGF1 hormone. Previously, the only treatment available was injection of genetically-engineered recombinant insulin-like-growth factor 1 (rhIGF1), administered daily or even twice daily. Yet, the results from this original treatment have not been ideal.

The team previously generated a specific gene delivery tool, called the AAV8 carrier that expressed the human insulin gene in the liver. When this showed potential to be used by diabetic patients as a long-term basal insulin gene therapy, the team of researchers which included Dr Sia Kian Chuan and Dr Gan Shu Uin, from the Department of Surgery, attempted to further explore the potential of the AAV8 gene delivery tool to understand the extent of its efficacy. The results showed the significant size and weight which increased steadily throughout the course of 26 weeks, in comparison to the untreated laboratory model.

Other researchers have shown that there is persistent gene expression for up to 10 years to produce proteins in the liver necessary for growth, following a single dose of AAV administration. This makes AAV gene therapy an attractive treatment with potentially significantly good therapy outcomes.

Using an AAV8 gene delivery tool, gene therapy has emerged as a safe and efficient treatment with the potential to treat a variety of inherited or rare mutation disorders such as hemophilia and spinal muscular atrophy. There were no treatments available for these disorders previously. With these optimistic results, the AAV vector can be potentially used to treat people with Laron syndrome.

Prof Lee said, Gene therapy, has given us a promising start in managing dwarfism. This is far more favourable compared to rhIGF1 injections, once or twice daily for many years, which causes side effects of pain and discomfort to patients and comes as a huge financial burden to those involved.

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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Promising results shown in the treatment of growth-hormone resistant dwarfism - EurekAlert

One of the most important systems in our bodies is the immune system. It defends our bodies from germs, infections, bacteria, viruses, and more. But, would you have imagined that about 1 in 58,000 babies are born with little to no Immune system? This is a very rare genetic disorder called Severe Combined Immunodeficiency (SCID). A baby can be diagnosed with SCID before birth through screenings, but most babies are diagnosed with SCID within the first six months of life.

When a child has SCID, it means they lack T cells, Natural Killer Cells, and Functional B cells. T Cells help protect the body from infection and help fight cancer. Natural Killer cells destroy cells infected with a virus. Functional B cells produce antibodies to fight bacteria and viruses.

The process of treating or curing a disease by altering a persons genes is known as gene therapy. In two previous studies conducted, SCID gene therapy consisted of two generations, both using viruses to deliver the genes.The first generation of treatment worked, but patients unfortunately developed leukemia, a cancer of the white blood cells that makes them abnormally large. The research community did deliver a second generation of gene therapies that were safer, but they did not completely restore the immune system.

To treat infants with SCID, stem cells are taken from the bone marrow of siblings, parents, or unrelated donors. Then, a bone marrow transplant introduces these healthy infection-fighting cells into the SCID infants body. The idea is that this will provide a new immune system for the patient. Generally, bone marrow stem cell transplants from family donors are effective but unavailable for more than 80% of patients in the world. This means a higher risk of the non-family donors T cells attacking and damaging the patients healthy cells.

In this study, the researchers used a type of gene therapy involving a lentiviral vector. A lentiviral vector is a type of virus called a lentivirus that inserts its RNA into the hosts cells. They took advantage of this viruss action to insert a corrected gene sequence, as an RNA strand, into the patients own bone marrow stem cells to both fix the genetic error and reduce the chance of rejection. The gene of interest is IL2RG, which instructs the body to make certain immune proteins in the bone marrow. When this gene is broken, SCID results.

The research team had successfully tried this type of treatment before in children and young adults with SCID. They combined the lentiviral vector gene therapy with a chemotherapy agent called nonmyeloablative busulfan, typically given to patients before a stem cell transplant. This drug destroys a patients bone marrow cells in preparation for new stem cells. The researchers hypothesized that lentiviral gene therapy, after a low dose of this drug busulfan, would be a safe and effective treatment for infants with recently diagnosed SCID.

First, the infants bone marrow was collected. The correct gene was inserted into the patients blood stem cells using the lentivirus vector or carrier. The cells were then frozen and went through quality testing in order to detect, reduce, and correct any problems that may have occurred. Importantly, the lentiviral vector contained protectors that prevented the gene therapy from accidentally causing leukemia. The protectors work by blocking the virus from turning on certain oncogenes (or cancer-linked genes) that happen to sit next to the IL2RG gene on the chromosome.

They recruited a group of eight infants newly diagnosed with SCID. The researchers conducted their experiment by giving the infants one to two daily doses of busulfan by injection. They customized the initial dose based on the weight and age of the patient and previous knowledge on how this drug typically moves through the body.

The results of the experiment supported the research teams prediction. Natural Killer cells were restored within the first four months in seven of the eights infants as the T cells. The eighth infant initially developed a low T cell count but improved after a boost of gene-corrected cells without needing additional busulfan pre-treatment. Several types of blood cells such as T, B and natural killer cells made in the bone marrow seemed active within 3-4 months after infusion with the viral lentiviral vector.

The combination of lentiviral gene therapy with busulfan conditioning appeared safe in all eight infants. These results aligned with what the researchers expected. Patients were followed for a range of 6-24 months after the study to assess whether their new immune system remained stable. After the 24 month period, they concluded that this treatment was more effective than current treatments for SCID patients with fewer side effects.

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New treatment for infants with weakened immune systems - Sciworthy

Bioinformatics Marketis valued atUSD 10.82 Billion in 2021and is expected to reachUSD 24.07 Billion by 2028with aCAGR of 12.1%over the forecast period.

The increasing scope in research and development, drug discovery, new advancements in genetics analysis and synthesis methods and techniques are driving the Bioinformatics market.

The healthcare sector has seen a development in the last couple of years with the coronavirus pandemic hurting the world in the hardest ways possible and changing the way people live forever. Healthcare has been the topic of discussions at the macro and micro levels and manufacturers have realized that it is time for them to invest in technology and come up with innovative ways to help people. They get funded extensively with the government initiatives too as the authorities all over the world create commissions to help the healthcare sector. The research initiatives take time and multiple rounds of investment but in the interest of the people at large they make sure that those requirements are met. Bioinformatics market in healthcare depends a lot on how innovative the advancements are and how they are used in the long run as the lock in period is always going to be long.

A trend which is aiding Bioinformatics market in healthcare is the habits of people which are increasingly becoming unhealthier. People, particularly in the developing countries live a life where they work really hard and they do not have the time to focus on their health. That is why there is a rise in number of obesity and other diseases in the world. With technological advancements, many illnesses can be diagnosed before they become serious ailments. Diabetes is a common illness which can now be tapped by checking the blood sugar levels at home through advancements of technology. The technological advancements in Bioinformatics market in healthcare can also help people get healthcare services remotely. It is difficult particularly in the developing nations to manage time for their healthcare requirements. This has increased the prevalence of illnesses that made the world look at innovative ways to come up with solutions. In the pandemic, we saw that testing kits played a key role in preventing the spread of the pandemic.

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Some major key players for global Bioinformatics market are,

Bioinformatics market in healthcare has now seen its future vision and how the world will operate once the pandemic is over. Technology is going to play a key role in how the world moves forward. The challenges are going to increase only with the eating habits increasingly becoming worse. Healthcare sector has also been focusing on the mental health aspect. There are innovative ways where counseling can be given online with the use of technology. Governments are also focusing on finding means of e-healthcare in order to benefit people remotely. E-healthcare has also been aided by the accessibility of social media to the deepest part of countries.

The growth of literacy and awareness is also helping people get their consultancy online. The COVID pandemic showed the world ways in which adoption of technology can be the key to how countries deal with healthcare emergencies. Another factor which is aiding growth is how the insurance is extending its wings to the online consultancy too. This will continuously aid the initiatives of online consultancy and help the healthcare sector see growth in the coming years. Bioinformatics market in healthcare surely has a future which will depend on technological advancements

Bioinformatics Market: Key Features

The report throws light on the competitive landscape, segmentation, geographical expansion, and revenue, production, and consumption growth of the Bioinformatics market. Bioinformatics Market Size, Growth Analysis, Industry Trend, and Forecast, offers details of the factors influencing the global business scope. This report provides future products, joint ventures, marketing strategy, developments, mergers and acquisitions, marketing, promotions, revenue, import, export, CAGR values, the industry as a whole, and the particular competitors faced are also studied in the large-scale market.

Bioinformatics Market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, production sites and facilities, company strengths and weaknesses, product launch, product trials pipelines, product approvals, patents, product width and breath, application dominance, technology lifeline curve. The data points provided are only related to the companys focus related to Bioinformatics market. Leading global Bioinformatics market players and manufacturers are studied to give a brief idea about competitions.

Latest news and industry developments in terms of market expansions, acquisitions, growth strategies, joint ventures and collaborations, product launches, market expansions etc. are included in the report. The report focuses on the operation and their competitive landscape present within the market. Identification of numerous key players of the market will help the reader perceive the ways and collaborations that players will need to understand the competition within the global Bioinformatics market.

Bioinformatics Market report provides depth analysis of the market recent developments and comprehensive competitive landscape created by the COVID19/CORONA Virus pandemic. Bioinformatics Market report is helpful for strategists, marketers and senior management, And Key Players in Bioinformatics Industry.

Market Dynamics Of Bioinformatics Market

Global Bioinformatics market report has the best research offerings and the required critical information for looking new product trends or competitive analysis of an existing or emerging market. Companies can sharpen their competitive edge again and again with this business report. The report comprises of expert insights on global industries, products, company profiles, and market trends. Users can gain unlimited, company-wide access to a comprehensive catalog of industry-specific market research from this industry analysis report. The market report examines industries at a much higher level than an industry study.

Table of Content: Global Bioinformatics Market Research Report

Chapter 1: Global Bioinformatics Industry Overview

Chapter 2: Global Economic Impact on Bioinformatics Market

Chapter 3: Global Market Size Competition by Industry Producers

Chapter 4: Global Productions, Revenue (Value), according to Regions

Chapter 5: Global Supplies (Production), Consumption, Export, Import, geographically

Chapter 6: Global Productions, Revenue (Value), Price Trend, Product Type

Chapter 7: Global Market Analysis, on the basis of Application

Chapter 8: Bioinformatics Market Industry Value Chain

Chapter 9: Bioinformatics Market Chain, Sourcing Strategy, and Downstream Buyers

Chapter 10: Strategies and key policies by Distributors/Suppliers/Traders

Chapter 11: Key Economic Indicators, by Market Vendors

Chapter 12: Market Effect Factors Analysis

Chapter 13: Global Bioinformatics Market Forecast Period

Chapter 14: Future Of The Market

Chapter 15: Appendix

Substantial research & development activities carry out by some players that comprises offering training to covering recent information on new technology, materials and techniques to innovative practice solutions, will complement the market growth is also explained. Frequent technological advances, superior portability, and ease of handling for Bioinformatics are boosting adoption in home and alternate care settings as well. Furthermore, non-profit and government initiatives, and awareness programs, and an influx of funding for research studies have positively influenced developments within the industry.

Global Bioinformatics Market: Regional Analysis

The research report includes specific segments by region (country), by company, by Type and by Application. This study provides information about the sales and revenue during the historic and forecasted period of 2022 to 2028. Understanding the segments helps in identifying the importance of different factors that aid the market growth.

Thanks for reading this article; you can also get individual chapter wise section or region wise report version like North America, Europe or Asia.

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Bioinformatics Market Size, Share, And Trends Analysis Report, By Application (Drug Development, Protein Function Analysis, Gene Therapy, Molecular...

Retinal regions of preserved photoreceptors identified as targets for subretinal delivery of AAV8-based gene therapy to address mutations in genes that cause forms of Leber congenital amaurosis

Subretinal injection well tolerated in preclinical dose-ranging studies

Data presented at Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting 2022

RESEARCH TRIANGLE PARK, N.C., May 04, 2022 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-focused gene therapy company developing treatments for inherited retinal diseases, today announced promising new preclinical data from studies evaluating the potential of its gene therapies OPGx-001 and OPGx-002 to address forms of Leber congenital amaurosis (LCA), a group of rare inherited retinal diseases characterized by photoreceptor degeneration, due to mutations ofLCA5orRDH12genes, respectively.

In preparation for IND-enabling trials of OPGx-001 and OPGx-002, studies were conducted to determine eligibility, therapeutic window, and possible outcome measures for gene therapy for LCA5 and RDH12 inherited retinal diseases. In addition, safety evaluations for the subretinal delivery of an AAV8 vector containing LCA5 or RDH12 were performed in non-human primates (NHP).

The data demonstrated that despite severe retinal dysfunction, LCA patients exhibited detectable photoreceptor regions that may be targets for gene augmentation, identified in the central and midperipheral retina of LCA5-LCA patients and in the pericentral and peripapillary retina of RDH12-LCA patients. In two dose-ranging studies in NHPs, subretinal delivery of OPGx-001 and OPGx-002 was well tolerated, with mild inflammatory changes observed at the higher dose. The data support the therapeutic potential and tolerability of gene augmentation to address LCA5-LCA and RDH12-LCA and provide guidance for formal preclinical toxicology studies and future human clinical trials.

Patients with Leber congenital amaurosis due to mutations of the LCA5 or RDH12 genes experience rapid retinal degeneration, resulting in vision loss in early childhood, said Ash Jayagopal, Ph.D., Chief Scientific Officer of Opus. The detection of preserved photoreceptors in LCA patients signals a therapeutic opportunity to target the mutation and potentially restore structure and function through gene augmentation. In addition, the encouraging dose-ranging results in the primate model suggest subretinal delivery of Opus AAV8-based gene therapies are safe and inform the doses to be used in our toxicology studies, a key step on our path toward the clinic for OPGx-001 and OPGx-002.

The data were presented today at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting 2022 in Denver, Colo., by Jean Bennett, M.D., Ph.D., Scientific Co-founder, Opus Genetics, and University of Pennsylvania Perelman School of Medicine; and Tomas Aleman, M.D., University of Pennsylvania Perelman School of Medicine.

Also at ARVO 2022, Dr. Jayagopal was awarded the title of ARVO Fellow, an honor established to recognize current ARVO members for their individual accomplishments, leadership and contributions to the ARVO Association.

About Opus GeneticsOpus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindnesss venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous retinal diseases. Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel manufacturing scale and efficiencies. Based in Research Triangle Park, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit http://www.opusgenetics.com.

Media Contact: Heather Anderson6 Degrees919-827-5539handerson@6degreespr.com

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Opus Genetics Announces Promising New Data Highlighting Potential of AAV-based Gene Therapies for the Treatment of Rare Inherited Retinal Diseases -...