Low- and middle-income countries (LMICs) can and should play a leading role in dictating the future of the worlds most advanced healthcare technologies, according to the World Economic Forums Accelerating Global Access to Gene Therapies: Case Studies from Low- and Middle-Income Countries white paper.

Gene therapy is at the forefront of modern medicine. By making precise changes to the human genome, these sophisticated technologies can potentially lead to one-time lifelong cures for infectious and non-communicable diseases (e.g. HIV, sickle cell disease) that affect tens of millions of people around the globe, most of whom live in LMICs. However, too often the benefits of advanced healthcare technologies remain restricted to high-income countries (HICs), a reality that could happen to gene therapies.

The narrative that new healthcare technologies are unsuitable for LMICs is a long-standing rationale for excluding a majority of the world from the benefits of modern medicine. Without concerted efforts to build gene therapy capacity in LMICs, the global health divide will continue to widen.

The gene therapy industry is in its infancy, but early clinical successes and substantial funding have generated enormous momentum. This is an ideal moment for LMICs to enter the global market, prioritizing the needs of communities carrying the highest disease burdens.

We asked five clinical researchers from LMICs, who are all co-authors on the recent white paper, what innovations on the ground and changes at policy-level need to happen for gene therapy to make a real impact on global health.

Dr. Cissy Kityo Mutuluza, Executive Director, Joint Clinical Research Centre, Uganda

Although gene therapy has the potential to treat or even cure life-limiting diseases and infections, the full impact will only be realized if we deliver it for the benefit of all people, instead of fueling more health inequity between and within countries.

An essential first step towards maximizing the global impact of gene therapies is to build research and development (R&D) capacity in LMICs. Current gene therapy R&D has mainly excluded LMICs, instead centering pre-clinical and clinical work in HICs. Gene therapy R&D needs to be performed in regions where target diseases are prevalent to ensure that these therapies are safe and effective for those populations. Manufacturing technologies and healthcare infrastructure, which are the cost drivers for gene therapy products in HICs, need to be replaced with innovative and simplified platforms and workflows that bring down costs and are functional and cost-effective within LMIC health systems.

As for policy and regulation, individual countries must establish gene therapy frameworks that enable R&D. The construction of such frameworks should be guided by recommendations from the World Health Organization, emphasizing safety, effectiveness and ethics.

A critical component in effective global health interventions is community outreach. Treatment acceptability is essential for future clinical trials, thus it is important for scientists and clinicians to be clear about the risks and benefits of gene therapies. Communication and education activities should be made accessible to a broad range of stakeholders. Gene therapy and gene editing technologies are complex and it can be difficult for the public to understand their possible benefits or side effects. However, patient and public support is critical for the successful adoption of any new technology.

Professor Johnny Mahlangu, University of the Witwatersrand, South Africa

The ongoing COVID-19 pandemic is accelerating innovation, implementation and acceptance of molecular therapeutics (e.g. mRNA vaccines) globally. As a result, there is escalating interest in developing molecular interventions for many other conditions, such as gene therapies for genetic diseases. Strategically leveraging infrastructure that is being developed for molecular therapeutics will be critical in manufacturing, testing, and delivering gene therapies across diverse settings. Three critical areas of consideration include:

The application of precision medicine to save and improve lives relies on good-quality, easily-accessible data on everything from our DNA to lifestyle and environmental factors. The opposite to a one-size-fits-all healthcare system, it has vast, untapped potential to transform the treatment and prediction of rare diseasesand disease in general.

But there is no global governance framework for such data and no common data portal. This is a problem that contributes to the premature deaths of hundreds of millions of rare-disease patients worldwide.

The World Economic Forums Breaking Barriers to Health Data Governance initiative is focused on creating, testing and growing a framework to support effective and responsible access across borders to sensitive health data for the treatment and diagnosis of rare diseases.

The data will be shared via a federated data system: a decentralized approach that allows different institutions to access each others data without that data ever leaving the organization it originated from. This is done via an application programming interface and strikes a balance between simply pooling data (posing security concerns) and limiting access completely.

The project is a collaboration between entities in the UK (Genomics England), Australia (Australian Genomics Health Alliance), Canada (Genomics4RD), and the US (Intermountain Healthcare).

Professor Vikram Mathews, Christian Medical College, Vellore, India

Gene therapy is on course to revolutionize medical care for several conditions. The hope is that gene therapy will be a one-time curative therapeutic intervention for diseases ranging from inherited hemoglobinopathies, such as sickle cell disease and thalassemia, to acquired diseases such as HIV.

A primary challenge limiting access to these life-saving therapies is their astronomical costs, making them inaccessible even in developed countries where most gene therapies have originated. Due to economic challenges, there is often a mismatch between regions in the world where development and clinical research happens versus regions in the world where the incidence of the disease target is the highest. Classic examples of these are sickle cell disease and HIV with the highest incidence rates in Africa.

Moving the manufacturing of gene therapy products to local regions and point of care settings (within hospitals) are strategies that can both significantly reduce the cost of these products and improve accessibility. Additionally, current gene therapy approaches use expensive ex vivo procedures that require removal of a patients cells from their body. Instead, researchers must develop novel in vivo methods that simplify the procedure to a single injection directly into the patient, saving time and money.

Professor Julie Makani, Muhimbili University of Health and Allied Sciences, Tanzania

In order for gene therapy to have an impact on global health, changes in innovation and policy must occur at several levels: individual, institutional, national, continental and global.

At the individual level, patients and personnel are the primary focal points. Taking a patient-centered approach will ensure that the community is involved in research and will have a say in receiving a particular health intervention when it is available. For personnel working in areas pertinent to gene therapy including healthcare, research and education, there is a need to increase knowledge and to change perspectives regarding the advancements and achievements made within the field of gene therapy.

At the national, continental and global levels, genomic research is catalyzed by strategic partnerships and often occur in Centers of Excellence (CoE). Many countries in Africa have established CoEs in academic settings, which integrate health and science programmes. These innovative environments help maximize resources (physical and human) and provide settings that facilitate research and translation of research findings to health interventions to be done contemporaneously, in the appropriate population and geographical region.

At the policy-level, investments in global health and research in gene therapy must change. This can be done in three ways: direct investment to institutions in Africa; increase in the level of investment through funding partnerships; and recognition that the duration of investment needs to be longer than the normal funding cycles of three to five years.

Professor Suradej Hongeng, Mahidol University, Thailand

Gene therapy has received global attention over the last few years, recognition that continues to grow with each new clinical success. The field is constantly evolving, with disruptive innovation across public and private sectors. However, access to these life-saving treatments remain restricted due to a number of technical and policy challenges.

First, researchers must continue to develop cost-effective ways to administer gene therapies into patients, an area of R&D where the private sector can play an important role. Yet many LMICs have weak ecosystems to support the emergence of new companies or entice collaborations with multinational companies. Stronger private sector involvement will be critical for penetration into emerging markets.

Second, the unique nature of these personalized treatments makes them difficult to regulate within traditional frameworks, meaning that agencies must update current policies and regulations. As regulation evolves, it must also converge with the frameworks of other countries. This will make it easier for companies to navigate regulations and interact with agencies when performing clinical trials or bringing a therapy to multiple markets.

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Gene therapy can make a real impact on global health but we need equitable access, say experts - World Economic Forum

Charles River will manufacture both plasmid DNA and viral vectors for late phase clinical trials targeting degenerative ocular diseases with no known cure

WILMINGTON, Mass. and DALLAS, Oct. 18, 2022 /PRNewswire/ -- Charles River Laboratories International, Inc. and Nanoscope Therapeutics, Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced a comprehensive manufacturing collaboration utilizing Charles River's extensive contract development and manufacturing (CDMO) services in both plasmid DNA and viral vectors.

"We are excited to continue to support Nanoscope Therapeutics' efforts in the production of gene therapies that are focused on restoring vision for people suffering from retinal degenerative diseases with no known cure. Their work is incredibly important to patients, and we look forward to continuing to serve as a partner," said Kerstin Dolph, Corporate Senior Vice President of Biologics Solutions at Charles River.

"Nanoscope looks forward to working with Charles River to support our accelerated development program. We have high expectations for our program and are counting on Charles River leadership, resources and scale to deliver in this partnership," said Anil Lalwani, Vice President of CMC at Nanoscope Therapeutics, Inc.

A Robust Manufacturing Collaboration

Through this partnership, Nanoscope will have access to established manufacturing platforms and multiple Charles River CDMO centers of excellence, leveraging a comprehensive range of services including but not limited to GMP cell banking, High Quality (HQ) and GMP-grade plasmid DNA manufacture, and GMP adeno-associated virus (AAV) production.

This gene therapy manufacturing partnership builds on Charles River's acquisitions of Cognate BioServices, Cobra Biologics, and Vigene Biosciences in 2021 that expanded its comprehensive cell and gene therapy (C>) portfolio to span each of the major CDMO platforms cell therapy, viral vector, and plasmid DNA production.

Treating Degenerative Retinal Diseases

Nanoscope Therapeutics is developing gene-agnostic, sight restoring Multi-Characteristic Opsin (MCO) optogenetic therapies for the millions of patients blinded by retinal degenerative diseases, for which no cure exists. Nanoscope's optogenetic therapy uses a proprietary AAV2 vector to deliver MCO genes into retinal cells to enable vision in different color environments. The therapy is administered as a single intravitreal injection for in-office delivery without the need for any other devices or interventions.

About Charles River

Charles River provides essential products and services to help pharmaceutical and biotechnology companies, government agencies and leading academic institutions around the globe accelerate their research and drug development efforts. Our dedicated employees are focused on providing clients with exactly what they need to improve and expedite the discovery, early-stage development and safe manufacture of new therapies for the patients who need them. To learn more about our unique portfolio and breadth of services, visitwww.criver.com.

About Nanoscope Therapeutics Inc.

Nanoscope Therapeutics is developing gene-agnostic, sight restoring optogenetic therapies for the millions of patients blinded by retinal degenerative diseases, for which no cure exists. The company's lead asset, MCO-010, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top line data expected H1 2023. The company has also fully enrolled a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA Fast Track designation for RP and FDA orphan drug designations for RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Nanoscope Investor Contact:Argot Partners212-600-1902[emailprotected]

Charles River Investor Contact:Todd SpencerCorporate Vice President,Investor Relations781.222.6455[emailprotected]

Charles River Media Contact:Amy CianciarusoCorporate Vice President,Chief Communications Officer781.222.6168[emailprotected]

SOURCE Nanoscope Therapeutics

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Charles River and Nanoscope Therapeutics Announce Multifaceted Gene Therapy Manufacturing Partnership - PR Newswire

Evernorth's Aimee Tharaldson discussed the growing number of gene therapies in her keynote talk today on specialty drug at AMCP Nexus 2022. She listed 12 approved gene therapies, eight in the near-term pipeline for hemophilia and another seven for other diseases that may be approved next year.

Gene therapy, once the stuff of scientific aspiration, is fast becoming a clinical reality in U.S. healthcare but with staggering tags. In her keynote talk today on specialty drugs at AMCP Nexus 2022, Aimee Tharaldson, Pharm.D., listed 12 approved gene therapies and another seven that could be approved next year.

Tharaldson, senior clinical pharmacist, emerging therapeutics, at Evernorth, told the large audience for her morning talk that 500 gene therapies are in clinical trials and that as many 1 million American will be treated with a gene therapy by 2034.

But Tharaldson also noted the cost, with the one-time gene replacement therapies priced at more in the millions of dollars. Citing a paper from the National Bureau of Economic Research, Tharaldson said the cumulative annual cost of gene therapies could reach $25 billion by 2034.

It is going to be interesting to see what happens because they are very expensive, Tharaldson said in a brief interview after her talk, which drew a large audience on the third day of the AMCP Nexus meeting in National Harbor, Maryland. Some of them are coming to market with pretty good data and some will likely come to market with not the best data. They are just going to need to be evaluated carefully to make sure they are effective and safe and have a durable effect.

Some of the gene therapies have been promoted as curative but Tharaldson sounded a note of caution :I dont think we have enough information at this point to say there are curative.

Tharaldson listed 12 approved gene therapies in her talk and another seven that could be approved by the FDA in 2023, including a gene therapy for Duchenne muscular dystrophy that could be priced at $3 million.

Three of the gene therapies that might be approved next year are for dystrophic epidermolysis bullosa, a rare skin disease.

Tharaldson also listed eight hemophilia gene therapies that are in late-stage development and could be approved over the next several years. One, etranacogene dezaparvovec, may be approved next month.

Value-based contracts have been discussed as one way to possibly hedge some of the cost impacts of the gene therapies. Tharaldson mentioned that Bluebird Bio has proposed a value-based contract for Zynteglo (betibeglogene autotemcel), its gene therapy for beta thalassemia, a blood disorder that results in hemoglobin levels. Bluebird priced the one-time gene replacement therapy, which was approved by the FDA in August 2022, at $2.8 million, but the company has also said that it would reimburse payers for perhaps as much 80% of that cost if patients do not stay "transfusion independent" for two years.

The FDA approved another Bluebird gene replacement therapy, Skysona (elivaldogene autotemcel), a treatment for cerebral adrenoleukodystrophy, a rare neurological condition, in September 2022. The price of $3 million is similar to price Zynteglo but Tharaldson said Bluebird hasn't offered any value-based terms for Skysona.

Here are some of the other highlights from Tharaldsons information-dense talk

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Gene Therapy Has Arrived. But So Will the $$$$$ Bills. | AMCP Nexus 2022 - Managed Healthcare Executive

Carmine Therapeutics has completed series A financing to support the development of its innovative, non-viral gene therapy for cystic fibrosis (CF) and other diseases.

The company is working to advance a novel type of disease-modifying gene therapy, which is accomplished using its proprietary Red Cell EV Gene Therapy or REGENT platform. This technology allows the delivery of therapeutic payloads of pieces of genetic information both DNA and RNA to several tissues, including in the central nervous system (involving the brain and spinal cord).

It also enables the transport of shorter and larger segments of genetic information, as well as multiple payloads simultaneously, without inducing an immune or inflammatory response that often render gene therapies less effective, according to Carmine.

REGENTs ability to deliver very large genetic medicines to a broad variety of tissues, without triggering an immune response or excessive inflammation has the potential to extend the promise of gene therapy to numerous human diseases that have been inaccessible to viral based approaches, Don Haut, PhD, Carmines CEO, said in a press release.

CF is causedby mutations in the geneCFTR, which result in an abnormally thick mucus in the bodys organs, particularly in the lungs, pancreas, liver, and intestine.

Gene therapy involves correcting a genetic defect by introducing a normal copy of the affected gene into the patients cells or by silencing a faulty gene. Current gene therapies commonly involve the use of adeno-associated viruses, which are modified in the lab not to cause disease, as delivery vehicles. However, existing approaches pose manufacturing challenges, limit the size of the therapeutic gene, and may induce an immune response.

The REGENT platform uses red-blood-cell-derived extracellular vesicles (RBCEVs), tiny sacs surrounded by a fat layer that can be used to carry genetic material. Since red blood cells naturally lack genetic material, RBCEVs do not represent a risk of triggering an immune response and therefore enable re-dosing, according to Carmine.

Also, large amounts of RBCEVs can be obtained from a single unit of blood and are highly amenable for manipulation. The REGENT platform was based on research led by the companys co-foundersMinh Le, PhD, and Jiahai Shi, PhD, at theNational University of Singapore, andHarvey Lodish, PhD, at the Massachusetts Institute of Technology.

The Cystic Fibrosis Foundation and Huagai Capital are two new investors in this financing round. They join existing investors EVX Ventures and Simcere Pharmaceutical.

We are delighted to bring a terrific group of new investors who share our vision of the next generation of gene therapies into Carmine, said Haut.

The funding will first be used to advance the clinical development of the gene therapy for retinal (eye) and pulmonary diseases.

We are thrilled to join [the] Carmine family, said Deng Liang, of Huagai Capital. As an early-stage investor in biotech industry, we value Carmines RBCEV platform as a novel and advantageous delivery system in gene therapies, especially considering its payload capacity, costs, and safety. Combined with Carmines patented payload engineering methods, we believe Carmine would bring patients with promising curative therapies.

Added XQ Lin, Carmines chairman and founder: We are pleased to welcome Huagai Capital and the Cystic Fibrosis Foundation as new investors into Carmine and thank our existing shareholders for their continued support.

Non-viral gene therapy is a promising new modality with the potential to address many unmet medical needs, he said.

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Carmine Gets Series A Funding to Develop Non-viral Gene Therapy |... - Cystic Fibrosis News Today

CBER maps modernization plan to handle surge in research and applications.

FDAs Center for Biologics Evaluation and Research (CBER) is updating how it manages a growing volume of cellular and gene therapy development programs, seeking added resources and revisions in its oversight of these cutting-edge therapies. Most visible in the elevation of CBERs Office of Tissues and Advanced Therapies (OTAT) into a new super Office of Therapeutic Products (OTP). The change aims to improve functional alignment, increase review capabilities, and add expertise on new cell and gene therapies by establishing multiple branches and divisions in the expanded regulatory unit, as announced in the Federal Register on Sept. 28, 2002.

Stated goals are to help CBER address the substantial growth in innovative, novel products that present new scientific, medical and regulatory challenges that require changes to its structure, including strategies to advance the Regenerative Medicine Advanced Therapy (RMAT) program. The added resources are needed to oversee more than 2000 development programs involving cellular and gene therapies, many involving innovative testing and manufacturing processes. This soaring workload has over-taxed CBER staffers, resulting in serious difficulties in retaining and hiring capable scientists.

The structural changes at CBER reflect agreed-on plans to hire new staffers with funding from recently reauthorized user fee programs. The PDUFA VII commitment letter calls for an additional 132 new hires for CBER in this coming year and another 48 employees the following year, most to support cell and gene therapy reviews at OTP. The reorganization plan calls for OTP to have seven officesfor therapeutic products, clinical evaluation, review management, pharmacology/toxicology, and two for CMCfor gene therapy and for cellular therapy and human tissues. There will be 14 divisions and 32 branches within those offices, providing attractive supervisory opportunities for both new and experienced staffers.

These changes come in the wake of FDA approval of two new gene therapies that have drawn wide attention for both their therapeutic potential and for million-dollar price tags. Bluebird bios Zynteglo was approved by FDA in August for patients with beta thalassemia, an inherited blood disorder causing serious anemia. That was followed a few weeks later with approval of Bluebirds Skysona to treat a rare neurological disorder afflicting young boys. Zynteglo carries a $2.8 million price tag, Skysonas list price is $4 million, but both therapies are expected to target fewer than 1500 patients, limiting the overall cost impact for the US healthcare system. A greater spending effect would come from FDA approval of a new treatment for sickle cell disease from Vertex Pharmaceuticals and CRISPR Therapeutics, which plan to begin a rolling review by FDA in the coming months. The important potential benefits of these treatments, along with concerns about their impact on healthcare spending and access, speaks to the need for a highly capable and sufficiently resourced FDA oversight program.

These developments also highlight the importance of sound testing and production methods for therapies made from living organisms, which are inherently variable and difficult to control and measure to assure product safety, identify, quality, purity, and strength. The surge in applications from a broad range of firms, moreover, has made it difficult for CBER staffers to schedule formal meetings with each sponsor seeking advice on how best to perform manufacturing and testing processes. And publishing new guidance on these changing and emerging issues also takes time and resources.

In response, FDA looks to engage a broad range of sponsors on topics related to product development through a series of virtual town hall meetings. The first was held Sept. 29, 2022 and addressed how manufacturers should describe and inform FDA about chemistry, manufacturing, and controls (CMC) in applications for gene therapies. Wilson Bryan, OTAT (now OTP) director, opened the session by describing plans for establishing OTP as a super office to increase review capabilities and enhance expertise on gene and cellular therapies and set the stage for OTP branch chiefs to field a broad range of queries, ranging from basic CMC policies for various stages of development, to the scope of potency assays and impact of delivery devices on dose potency and quality [a recording of the town hall meeting is available at the FDA events link].

Main topics were comparability testing, assays for product characterization, and process controls. OTP staffers emphasized the importance of determining process requirements early in development to avoid late changes and analytical method variability that could raise uncertainties likely to delay clinical trials. Products with complex mechanisms of action, they advised, stand to benefit from early product characterization and potency assay development. And developers of gene therapies should use multiple production lots during a clinical study to ensure product consistency and quality, even for treatments for very small patient populations.

Manufacturers raised questions about differing CMC issues between early Phase I and late-stage clinical trials and voiced concerns about product characterization related to autologous cell-based gene therapies. A main theme from FDA was the importance of sponsors establishing a well-controlled manufacturing process and qualified analytical testing well before administering any new gene product. While CBER plans to issue guidance on manufacturing changes and comparability for cellular and gene therapy products, the information provided at this session provides unofficial guidance for implementing changes in product manufacturing and the scope of comparability assessments and development studies expected to support such changes.

Jill Wechsler is Washington editor for Pharmaceutical Technology.

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FDA Expands Oversight of Cell and Gene Therapies - Pharmaceutical Technology Magazine

The IND for DB-OTO provides clearance for the Company to initiate a pediatric Phase 1/2 clinical trial in the U.S. in children and infants, and is part of an international regulatory strategy for clinical development

One-time administration of DB-OTO has resulted in production of otoferlin protein and durable auditory brainstem responses to sound in a congenitally deaf rodent disease model

DB-OTO is Decibels second hearing therapeutic candidate to enter clinical investigation

BOSTON, Oct. 17, 2022 (GLOBE NEWSWIRE) -- Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, today announced that it has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to initiate a Phase 1/2 clinical trial in pediatric patients of DB-OTO, its lead gene therapy product candidate. DB-OTO is designed to provide durable hearing in individuals born with profound congenital hearing loss due to an otoferlin deficiency.

We are thrilled to work with families, advocacy groups and clinicians in the deaf and hard of hearing community to advance DB-OTO into the clinic, said Laurence Reid, Ph.D., Chief Executive Officer at Decibel. Decibel has assembled a compelling preclinical data package showing that DB-OTO demonstrated a favorable tolerability profile and an ability to stably generate full-length otoferlin transcript, express otoferlin protein and provide hearing in animal models. We are at an exciting time in the development of a new wave of precision gene therapies for children who are deaf and hard of hearing.

DB-OTO is being developed in collaboration with Regeneron Pharmaceuticals and is an adeno-associated virus (AAV)-based, dual-vector, gene therapy product candidate. Otoferlin is a protein expressed in cochlear inner hair cells that enables communication between the sensory hair cells of the inner ear and the auditory nerve. Newborns born with mutations in the otoferlin gene have fully developed structures within the inner ear. However, these newborns have profound hearing loss because signaling between the ear and the brain is disrupted. DB-OTO uses a proprietary, cell-selective promoter to express the otoferlin transgene in hair cells, with the goal of enabling the ear to transmit sound to the brain and provide hearing. DB-OTO received Orphan Drug and Rare Pediatric Disease designations from the FDA in 2021. Currently, there are no approved pharmacologic treatment options for individuals with otoferlin-related hearing loss.

In preclinical studies, Decibel observed that delivery of DB-OTO to the inner ear resulted in production of otoferlin protein and durable auditory brainstem responses to sound in a congenitally deaf, rodent otoferlin disease model. Preclinical studies in non-human primates demonstrated that the local delivery procedure for DB-OTO, an intra-cochlear injection using the surgical approach employed by neurotologists and pediatric otolaryngologists during a standard cochlear implantation procedure, resulted in successful distribution and expression of otoferlin protein across the cochlear length.

The Phase 1/2 dose escalation clinical trial is designed to evaluate the safety, tolerability and efficacy of DB-OTO in pediatric patients with congenital hearing loss due to an otoferlin deficiency. In addition to safety and tolerability endpoints, established, clinically relevant, objective and behavioral measurements of hearing will be used as efficacy endpoints in the clinical trial. The auditory brainstem response, which was used to characterize dose-response of DB-OTO after intra-cochlear delivery in translational studies, will serve as an early, objective, clinically accepted readout of hearing thresholds in the clinical trial.

Otolaryngologists, audiologists and auditory scientists have long awaited the clinical realization of the promise of biological therapies for hearing loss. Gene therapy for congenital deafness represents one such intervention and it would be an understatement to say that clinicians in the field of hearing loss are quite excited to see its advancement into clinical trials, said Jay Rubinstein, M.D., Ph.D., Professor and Virginia Merrill Bloedel Chair in Otolaryngology, Head and Neck Surgery at the University of Washington School of Medicine.

Based on discussions with the FDA during the IND review period, Decibel expects the first two participants in the U.S. portion of the Phase 1/2 trial will be as young as seven years of age and that subsequent participants will include children as young as two years of age and infants younger than two years of age. The Company intends to provide an update on the design of the clinical trial in the future. The DB-OTO IND is part of an international regulatory strategy for development of DB-OTO, which also includes plans to submit one or more Clinical Trial Applications (CTAs) in Europe.

DB-OTO is the second product candidate in Decibels pipeline to advance into clinical testing. In June 2022, Decibel reported positive data from the interim analysis of the Companys Phase 1b clinical trial of DB-020, a novel, proprietary formulation of sodium thiosulfate (STS) designed to protect against hearing loss in cancer patients receiving cisplatin chemotherapy. In the data from the interim analysis, 88% of patients experienced ototoxicity in their placebo-treated ear, and of these patients, 87% were partially or completely protected from ototoxicity in their DB-020-treated ears.

About Decibel TherapeuticsDecibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, one of the largest areas of unmet need in medicine. Decibel has built a proprietary platform that integrates single-cell genomics and bioinformatic analyses, precision gene therapy technologies and expertise in inner ear biology. Decibel is leveraging its platform to advance gene therapies designed to selectively replace genes for the treatment of congenital, monogenic hearing loss and to regenerate inner ear hair cells for the treatment of acquired hearing and balance disorders. Decibels pipeline, including its lead gene therapy product candidate, DB-OTO, to treat congenital, monogenic hearing loss, is designed to deliver on our vision of creating a world of connection for people with hearing and balance disorders. For more information about Decibel Therapeutics, please visit http://www.decibeltx.com or follow us on Twitter.

Forward-Looking Statements

This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release, including statements regarding Decibels strategy, future operations, prospects, plans, objectives of management, the therapeutic potential for Decibels product candidates and preclinical programs, the potential benefits of cell-selective expression, plans to submit one or more CTAs in Europe and the expected timeline for initiating a Phase 1/2 clinical trial of DB-OTO constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. The words anticipate, believe, continue, could, estimate, expect, intend, may, might, objective, ongoing, plan, predict, project, potential, should, or would, or the negative of these terms, or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Decibel may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the timing of and Decibels ability to obtain approval to initiate clinical development of its program candidates, whether results from preclinical studies will be predictive of the results of later preclinical studies and clinical trials, whether Decibels cash resources are sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements, uncertainties related to the impact of the COVID-19 pandemic on Decibels business and operations, as well as the risks and uncertainties identified in Decibels filings with the Securities and Exchange Commission (SEC), including those risks detailed under the caption Risk Factors in Decibels Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2022 and in other filings Decibel may make with the SEC. In addition, the forward-looking statements included in this press release represent Decibels views as of the date of this press release. Decibel anticipates that subsequent events and developments will cause its views to change. However, while Decibel may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Decibels views as of any date subsequent to the date of this press release.

Investor Contact:Julie SeidelStern Investor Relations, Inc.julie.seidel@sternir.com212-362-1200

Media Contact:Chris RaileyTen Bridge CommunicationsChris@tenbridgecommunications.com617-834-0936

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Decibel Therapeutics Receives FDA Clearance of IND Application for DB-OTO, a Gene Therapy Product Candidate Designed to Provide Hearing to Individuals...

SEOUL, South Korea, Oct. 18, 2022 /PRNewswire/ --On the Oct 17,2022, GenScript ProBio (Brian Ho-sung Min, CEO), a global CDMO, and GeneCraft (Suk Chul Bae, CEO),a global research team led by Professor Suk Chul Bae of lung cancer mechanism research, announced that they had entered into a strategic partnership MOU concerning the development and production of anew drugs needed for RX001.GenScript ProBio and GeneCraft have agreed to strengthen their cooperation in the AAV gene therapy field through this MOU.

Left Prof. Suk Chul Bae, CEO of GeneCraft / Right Dr. Brian H. Min CEO of GenScript ProBio

GenScript ProBio and GeneCraft are in the process of signing a contract for plasmid and AAV development and production for the GeneCraft's own new drug candidate Pan-KRAS non-small cell lung cancer anti-cancer gene therapy (RX001) developed as a new drug.With this agreement, GenScript ProBio has become a global partner that can support GeneCraft's various gene therapy pipelines.

Brian H. Min, CEO of GenScript ProBio said, "We are very happy to cooperate with GeneCraft in strategic partnership, and we are looking forward to accelerating the development of GeneCraft's gene therapy pipelines as a global partner through our accumulated technology."

Suk Chul Bae, CEO of GeneCraft Inc. said, "Through this strategic partnership with GeneScript ProBio, we look forward to successfully conducting the upcoming clinical trials of RX001, a first-in-class anticancer gene therapy. By establishing a long-term partnership we hope to become the global leaders in the gene therapy market."

About GeneCraftInc.GeneCraftInc.isaninnovativegenetherapydeveloperwithitsrootsincancerdefensemechanismsresearchedbyProfessorSukChulBae, a world renowned cancer research scholar. Genecraftisagloballeaderindevelopinggenetherapyproductswithitscontractdevelopment(CDO) platform specialized in improving the expression vector through viral expression recombination andcapsidmodification.

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About GenScript ProBio

GenScript ProBio is the subsidiary of GenScript Biotech Corporation, proactively providing end-to-end CDMO service from drug discovery to commercialization with proactive strategies, professional solutions and efficient processes in cell and gene therapy (CGT), vaccine, biologics discovery and antibody protein drug to accelerate drug development for customers. GenScript ProBio has established companies in the United States, the Netherlands, South Korea, Shanghai, Hong Kong, Nanjing and other places to serve global customers, and supported customers in the United States, Europe, Asia Pacific and other regions to obtain more than 30 IND approvals. Toward the mission of "Innovation through Collaboration", GenScript ProBio is committed to helping customers shorten the timeline for the development of biological drugs from discovery to commercialization, significantly lowering R&D costs and building a healthier future.

http://www.genscriptprobio.com

SOURCE GenScript ProBio

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GenScript ProBio Signs MOU to Form Strategic Partnership with GeneCraft To Development and Production of New AAV gene therapies - Yahoo Finance

Three in 10 respondents consider their SMA controlled, but 69% are hopeful additional treatment breakthroughs will occur in their lifetime

PHILADELPHIA, Oct. 18, 2022 /PRNewswire/ -- People with spinal muscular atrophy are relying on recently approved condition-specific treatments and are hopeful the treatment pipeline can continue to have a positive impact, according to a recent survey conducted by Health Union, the leader in social health. The inaugural Spinal Muscular Atrophy In America survey illuminates the perspectives and experiences of people living with spinal muscular atrophy.

These findings also support and fuel content and engagement for the recent launch of SpinalMuscularAtrophy.net, one of Health Union's 40 condition-specific online health communities.

Spinal muscular atrophy, or SMA, is a rare chronic condition, with research suggesting up to 25,000 people in the United States are living with the condition. According to the National Institute of Neurological Disorders and Stroke, SMA is an inherited condition that affects nerves and muscles, caused by a mutation in the survival motor neuron, or SMN, gene. Without the protein produced by the SMN gene, people with SMA lose motor neurons in their spinal cord, leading to weakness in the skeletal muscles and often making it harder to speak, walk, breathe and swallow.

Over the past six years, the U.S. Food and Drug Administration has approved the first three treatments specifically for spinal muscular atrophy. These gene therapy treatments increase the amount of SMN protein in the body. In fact, three-fourths of Spinal Muscular Atrophy In America survey respondents said they currently use an "SMN-enhancing" treatment, while another 19% said they previously used one.

SpinalMuscularAtrophy.net patient leader Allie Williams is using one of these SMN-enhancing treatments, which she says has stopped the progression of her spinal muscular atrophy while also helping her make "tremendous gains physically and mentally."

"It has improved my quality of life by letting me do things I wasn't able to do before, like feeding myself, drawing, painting, playing video games and doing my own makeup," Williams said. "Being more independent and being able to do more things I enjoy by myself has improved my happiness beyond measure."

Despite this progress in treatment, experiences differ, especially when factoring in condition progression and symptoms experienced. Only three in 10 respondents consider their SMA controlled under their current treatment plan, and 74% remain worried about their condition progressing.

With this in mind, it is unsurprising that the treatment pipeline remains a priority for people with SMA. When seeking information about their condition, respondents's top topics of interest - with the exception of assistive devices to improve daily life - all revolved around treatment, including new treatments in the pipeline, other SMA patients' experiences with treatments, research and clinical trials for SMA. On top of that, seven in 10 respondents said they are hopeful future treatment breakthroughs will happen in their lifetime.

Fortunately, two-thirds of respondents agree they have reliable access to medical care or treatment, although 44% said finances prevent them from receiving the care they need. Luckily, 44% said they are confident they're doing everything necessary - a mixture of treatments, specialists and other healthcare professionals, assistive devices and other life modifications - to manage their SMA on a regular basis.

"With a number of recently approved treatments already having an impact on quality of life and an intriguing treatment pipeline, people with spinal muscular atrophy are actively looking for accurate information, including the treatment experiences of others," said Olivier Chateau, Health Union's co-founder and CEO. "SpinalMuscularAtrophy.net provides a safe, supportive environment where people with the condition can find the information, connection and support they're looking for."

The inaugural Spinal Muscular Atrophy In America survey, which was fielded from April 19, 2021 to April 8, 2022, included responses from 54 people living with spinal muscular atrophy. Additional survey results may be available upon request. More information about living with spinal muscular atrophycan be found on SpinalMuscularAtrophy.net.

About Health Union

Health Unionis the proven industry leader driving and amplifying social health. As the premier social health company, only Health Union encourages the dynamic, real-time action people take to find meaningful connections and share information that impact their health journey. The company reaches millions of people through the largest portfolio of condition-specific online health communities (e.g., Migraine.com, MultipleSclerosis.net, LungCancer.net) and health leaders - addressing virtually every condition and providing the information, connection and support they need.

SOURCE Health Union

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Survey Reveals Most People with Spinal Muscular Atrophy Using New Gene Therapies, Focused on Treatment Pipeline - PR Newswire

DORVAL, QC, Oct. 17, 2022 /CNW Telbec/ - Novartis Pharmaceuticals Canada Inc. is pleased to announce Luxturna (voretigene neparvovec) is now available to Quebec patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations under the province's public health plan. This announcement follows a positive reimbursement recommendationfrom theInstitut national d'excellence en sant et en services sociaux(INESSS) recognizing the need for treatment options to slow the progression of the disease.

"It has been a long journey for patients waiting for public access to Luxturna. We are pleased with the Quebec government's decision and hope that eligible Canadians in other provinces have the same chance to be treated with Luxturna," said Doug Earle, President & CEO of Fighting Blindness Canada. "To progressively lose your vision to blindness, especially at a young age, is shattering. Our hope is that other provinces move quickly, because for these Canadians, time is of the essence."

Luxturnareceived Health Canada approval in 2020 and was eagerly anticipated by the vision community as the first pharmacological treatment option for people living with this rare, progressive genetic condition1. For most of these patients, progressive vision loss and total blindness were part of the life-long burden of the disease.

"This is a pivotal moment for the IRD community and for patients who could benefit from treatment with Luxturna," said Andrea Marazzi, Country President, Novartis Canada. "We believe the recognition by INESSS to fund Luxturna is vital and gives children and adults with biallelic RPE65 mutation-associated retinal dystrophy a chance for improved vision. We will continue to work towards broad reimbursement through public drug plans across Canada to ensure other Canadians have the opportunity to thrive."

Details regarding the INESSS recommendation are availablehere. The reimbursement criteria for Luxturna (voretigene neparvovec) on Quebec's public health plan are available here.

AboutRPE65mutation-associated inherited retinal dystrophy

Mutations in both copies of theRPE65gene affect approximately 1 in 200,000 people and can lead to blindness3,4. Early in the disease patients can suffer from night blindness (nyctalopia), loss of light sensitivity, loss of peripheral vision, loss of sharpness or clarity of vision, impaired dark adaptation, and repetitive uncontrolled movements of the eye (nystagmus)4. Patients with mutations in both copies of theRPE65gene may be diagnosed, for instance, with subtypes of either retinitis pigmentosa or Leber congenital amaurosis5.

About Novartis in Gene Therapy and Rare Disease

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

About Novartis in Canada

Novartis Pharmaceuticals Canada Inc., a leader in the healthcare field, is committed to the discovery, development and marketing of innovative products to improve the well-being of all Canadians. Over the last 5 years, our average annual research and development investment in Canada was $47 million. Located in Dorval, Quebec, Novartis Pharmaceuticals Canada Inc. employs approximately 1,000 people in Canada and is an affiliate of Novartis AG, which provides innovative healthcare solutions that address the evolving needs of patients and societies. The company prides itself on its commitment to diversity and to nurturing an inclusive and inspiring environment. Novartis is recognized as a Great Place to Work, ranked among the Top 50 Best Workplaces in the country and is proudly named on the 2021 Best Workplaces for Women in Canada and Best Workplace for Mental Wellness lists. For further information, please consultwww.novartis.ca.

About Novartis

Novartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach more than 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 108,000 people of more than 140 nationalities work at Novartis around the world. Find out more atwww.novartis.com.

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

References

SOURCE Novartis Pharmaceuticals Canada Inc.

For further information: Novartis Media Relations: Katia Kononova, Novartis Pharmaceuticals Communications, + 1 514 633 7873, E-mail: [emailprotected]

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Gene therapy Luxturna now reimbursed in Quebec for people with previously untreatable inherited vision loss(1) - Canada NewsWire

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces data presentations at the 29th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) in Edinburgh, United Kingdom, taking place October 11-14, 2022. Presentations will include clinical data from Rockets lentiviral vector (LV)-based gene therapy programs for Leukocyte Adhesion Deficiency-I (LAD-I), Fanconi Anemia (FA) and Pyruvate Kinase Deficiency (PKD). Donald B. Kohn, MD, Distinguished Professor of Microbiology, Immunology & Molecular Genetics, Pediatrics, and Molecular & Medical Pharmacology at University of California, Los Angeles (UCLA) and Director of the UCLA Human Gene and Cell Therapy Program, will also give an Invited Talk incorporating previously disclosed data from the RP-L201 trial for LAD-I.

Positive Updated Safety and Efficacy Data from Phase 2 Pivotal Trial for Fanconi Anemia (FA)

The poster and presentation include updated safety and efficacy data from the Phase 2 pivotal trial of RP-L102, Rockets ex-vivo lentiviral gene therapy candidate for the treatment of FA.

Positive Top-line Clinical Data from Phase 2 Pivotal Trial for Severe Leukocyte Adhesion Deficiency-I (LAD-I)

The oral presentation includes previously disclosed efficacy and safety data at three to 24 months of follow-up after RP-L201 infusion for all patients and overall survival data for seven patients at 12 months or longer after infusion. RP-L201 is Rockets ex-vivo lentiviral gene therapy candidate for the treatment of severe LAD-I.

Interim Data from Ongoing Phase 1 Trial for Pyruvate Kinase Deficiency (PKD)

The poster and presentation include previously disclosed safety and efficacy data from the Phase 1 trial of RP-L301, Rockets ex-vivo lentiviral gene therapy candidate for the treatment of PKD.

Details for Rockets Invited Talk and poster presentations are as follows:

Title: Interim Results from an ongoing Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)Session: Clinical Trials (Plenary 2)Presenter: Donald B. Kohn, MD - University of California, Los Angeles, Distinguished Professor of Microbiology, Immunology & Molecular Genetics (MIMG), Pediatrics, and Molecular & Medical Pharmacology; Director of the UCLA Human Gene and Cell Therapy ProgramSession date and time: Wednesday, 12 October at 11:10-13:15 BSTLocation: Edinburgh International Conference Centre (EICC)Presentation Number: INV20

Title: Lentiviral-Mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Results from Global RP-L102 Clinical TrialsSession: Poster Session 1Presenter: Julin Sevilla MD, PhD - Fundacin para la Investigacin Biomdica, Hospital Infantil Universitario Nio JessSession date and time: Wednesday, 12 October at 19:30-21:00 BSTLocation: Edinburgh International Conference Centre (EICC)Poster Number: P139

Title: Preliminary Conclusions of the Phase I/II Gene therapy Trial in Patients with Fanconi Anemia-ASession: Blood Diseases: Haematopoietic Cell DisordersPresenter: Juan Bueren, PhD - Unidad de Innovacin Biomdica, Centro de Investigaciones Energticas, Medioambientales y Tecnolgicas (CIEMAT)Session date and time: Thursday, 13 October at 15:30-17:30 BSTLocation: Edinburgh International Conference Centre (EICC)Presentation Number: INV41

Title: Interim Results from an Ongoing Global Phase 1 Study of Lentiviral-Mediated Gene Therapy for Pyruvate Kinase DeficiencySession: Poster Session 2Presenter: Jos Luis Lpez Lorenzo, MD, Hospital Universitario Fundacin Jimnez DazSession date and time: Thursday, 13 October at 17:30-19:15 BSTLocation: Edinburgh International Conference Centre (EICC)Poster Number: P128

Abstracts for the presentations can be found online at: https://www.esgct.eu/.

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutations in the FANCA gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Increased sensitivity to DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is a gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natural gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells.

About Leukocyte Adhesion Deficiency-I

Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California.

About Pyruvate Kinase Deficiency

Pyruvate kinase deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway. Mutations in the PKLR gene result in increased red cell destruction and the disorder ranges from mild to life-threatening anemia. PKD has an estimated prevalence of 4,000 to 8,000 patients in the United States and the European Union. Children are the most commonly and severely affected subgroup of patients. Currently available treatments include splenectomy and red blood cell transfusions, which are associated with immune defects and chronic iron overload.

RP-L301 was in-licensed from the Centro de Investigaciones Energticas, Medioambientales y Tecnolgicas (CIEMAT), Centro de Investigacin Biomdica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigacin Sanitaria de la Fundacin Jimnez Daz (IIS-FJD).

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare childhood disorders. The Companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rockets future expectations, plans and prospects, including without limitation, Rockets expectations regarding its guidance for 2022 in light of COVID-19, the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), and Danon Disease, the expected timing and data readouts of Rockets ongoing and planned clinical trials, the expected timing and outcome of Rockets regulatory interactions and planned submissions, Rockets plans for the advancement of its Danon Disease program and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rockets ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rockets dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rockets Annual Report on Form 10-K for the year ended December 31, 2021, filed February 28, 2022 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Announces Presentations Highlighting Lentiviral Gene Therapies at the 29th Annual Congress of the European Society of Gene...

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BioMagnetic Solutions will supply research and cGMP manufacturing grade FerroSelect reagents to Lonza for use in developing CAR-T and other immunotherapeutics.

10.14.22

The collaboration provides a framework for additional clinical grade reagents to be developed and opens up the possibility of other collaborations as Gamma Biosciences expands its portfolio of tools and technologies supporting the bioprocessing, vaccine, cell and gene therapy industries.

"We are looking forward to working together with the team at Gamma Biosciences to offer cell and gene therapy developers added cell selection functionality on the Cocoon Platform" said Adam Bryan, Vice President of Personalized Medicines at Lonza. "The BioMagnetic Solutions ferrofluid technology provides efficient, high-performance cell selection reagents for research, clinical, and commercial-scale cell separations in a workflow that can be easily integrated into our automated, closed-system Cocoon manufacturing platform. This collaboration speaks to Lonza's commitment to expanding the flexibility and functionality of our automated manufacturing technology to address a broader range of needs for the cell therapy industry."

"We are thrilled to collaborate with Lonza in further developing and adapting FerroSelect reagents for use with the Cocoon Platform," said Phil Vanek, Chief Technology Officer at Gamma Biosciences. "The Lonza platform can be an alternative to our FerroSelect Array cell selection platform, demonstrating that our proprietary reagents can be applied to other automated manufacturing technologies. The agreement gives BioMagnetic Solutions technical direction on new applications, and access to some of the best process developers in the industry. In return, Lonza gains early access to certain BioMagnetic Solutions FerroSelect reagents to complement the growing demand for their Cocoon Platform technology."

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Gamma Biosciences, Lonza Enter Cell and Gene Therapy Alliance - Contract Pharma

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Dublin, Oct. 17, 2022 (GLOBE NEWSWIRE) -- The "AAV Vector Market: Industry Trends and Global Forecasts, 2022-2035" report has been added to ResearchAndMarkets.com's offering.

This report features an extensive study on the current market landscape, offering an informed opinion on the likely evolution and future opportunities associated with the adeno-associated viral vector market. The study underlines an in-depth analysis, highlighting the key drivers and the market trends related to this evolving segment of the pharmaceutical industry.

Given the potential of gene therapies in targeting the underlying cause of a disease at cellular level, the demand for such therapies has increased considerably over the past few years. Currently, more than 285 gene therapies are being evaluated in different phases of clinical development.

Further, various gene therapy developers have raised more than USD 10 billion capital during the year 2021. With the growing interest in such therapies, the demand for novel delivery vectors has also increased. Among various gene delivery vectors available, adeno-associated viral (AAV) vectors have emerged as one of the most efficient viral vectors. Till now, the USFDA has approved two adeno-associated viral vectors based drugs, LUXTURNA and ZOLGENSMA.

A number of adeno-associated viral vector-based therapies are also being evaluated in different clinical trials. In fact, it has been observed that clinical trials evaluating adeno-associated viral vector based therapies have increased at ~30% growth rate over the last few years. Further, it is worth mentioning that over 50 trials are expected to complete in the next three years.

In order to cater to the demand, close to 100 players, across the globe, have emerged for the development and manufacturing of adeno-associated viral vectors.

In fact, a number of these companies also offer advanced technology platforms, enabling the processing of adeno-associated viral vectors and related therapies across different scales of operation. Various industry and non-industry players are actively engaged in research and development of novel gene delivery technologies, which are safe and effective. This is evident from the patents filed for protection of intellectual property related to such technologies.

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Additionally, multiple partnerships and collaborations have been established between the stakeholders in order to enhance development activity in this industry. Considering the prevalent trends and projected opportunity associated with the overall adeno-associated viral vector / AAV vector domain, we believe that the market is anticipated to witness substantial growth in the foreseen future.

Key Questions Answered

Which adeno-associated viral vector-based therapy candidates are present in the current development pipeline? Which disease indications are targeted by such products?

Who are the leading players (contract service providers and in-house manufacturers) engaged in the development of adeno-associated viral vectors?

Which type of adeno-associated viral vector related technologies are presently offered / being developed by the players in this domain?

Which geographies are the most active in conducting clinical trials related to adeno-associated viral vectors?

Which partnership models are commonly adopted by industry and non-industry stakeholders?

Which companies are likely to partner with adeno-associated viral vector and gene therapy product manufacturers?

What are the different initiatives undertaken by start-ups for the development of adeno-associated viral vectors in the recent past?

Which factors are likely to influence the decision of manufacturing the adeno-associated viral vector in-house or via outsourcing?

How is the current and future market opportunity likely to be distributed across key market segments?

Key Topics Covered:

1. PREFACE

2. EXECUTIVE SUMMARY

3. INTRODUCTION3.1. Chapter Overview3.2. Viral and Non-Viral Vectors3.2.1. Viral Vectors3.2.1.1. Adenovirus Vectors3.2.1.2. Adeno-Associated Viral Vectors3.2.1.3. Lentivirus Vectors3.2.1.4. Retrovirus Vectors3.2.1.5. Other Viral Vectors3.2.1.5.1. Alphavirus3.2.1.5.2. Foamy Virus3.2.1.5.3. Simian Virus3.2.1.5.4. Vaccinia Virus3.2.1.5.5. Chimeric Viral Vectors3.2.1.5.6. Herpes Simplex Virus3.2.1.5.7. Sendai Virus3.2.2. Non-Viral Vectors3.2.2.1. Plasmid DNA3.2.2.2. Oligonucleotides3.2.2.3. Liposomes, Lipoplexes, and Polyplexes3.2.2.4. Other Non-Viral Vectors3.3. Adeno-Associated Viral Vectors3.3.1. Structure and Design3.3.2. Adeno-Associated Viral Vector Life Cycle3.3.3. Applications of Adeno-Associated Viral Vectors3.3.3.1. Gene Therapy3.3.3.2. Vaccination3.3.4. Advantages of Adeno-Associated Viral Vectors3.3.5. Challenges Related to Adeno-Associated Viral Vectors3.4. Concluding Remarks

4. ADENO-ASSOCIATED VIRAL VECTOR BASED THERAPY: MARKET LANDSCAPE4.1. Chapter Overview4.2. Adeno-Associated Viral Vector Based Therapy: Overall Market Landscape4.2.1. Analysis by Phase of Development4.2.2. Analysis by Therapeutic Area(s)4.2.3. Analysis by Type of Gene / Molecule Targeted4.2.4. Analysis by Type of Therapy4.2.5. Analysis by Type of Gene Delivery Method Used4.2.6. Analysis by Route of Administration4.3. Adeno-Associated Viral Vector Based Therapy Candidates: Special Designations4.3.1. Analysis by Special Designation(s) Awarded4.4. Adeno-Associated Viral Vector Based Therapy: List of Developers4.4.1. Analysis by Year of Establishment4.4.2. Analysis by Company Size4.4.3. Analysis by Location of Headquarters4.4.4. Adeno-Associated Viral Vector Based Therapy: Leading Developers

5. ADENO-ASSOCIATED VIRAL VECTOR MANUFACTURERS: MARKET LANDSCAPE5.1. Chapter Overview5.2. Adeno-Associated Viral Vector Manufacturers: Overall Market Landscape5.2.1. Analysis by Year of Establishment5.2.2. Analysis by Company Size5.2.3. Analysis by Location of Headquarters5.2.4. Analysis by Type of Product(s) Manufactured5.2.5. Analysis by Location of Vector Manufacturing Facilities5.2.6. Analysis by Type of Manufacturer and Company Size5.2.7. Analysis by Scale of Operation5.2.8. Analysis by Location of Headquarters and Scale of Operation5.2.9. Analysis by Application Area(s)

6. ADENO-ASSOCIATED VIRAL VECTOR MANUFACTURING TECHNOLOGIES: MARKET LANDSCAPE6.1. Chapter Overview6.2. Adeno-Associated Viral Vector Manufacturing Technologies6.2.1. Analysis by Type of Technology6.2.2. Analysis by Scale of Operation6.2.3. Analysis by Application Area(s)6.2.4. Analysis by Therapeutic Area6.3. Adeno-Associated Viral Vector Technology: List of Developers6.3.1. Analysis by Year of Establishment6.3.2. Analysis by Company Size6.3.3. Analysis by Location of Headquarters6.3.4. Analysis by Company Size and Location of Headquarters6.3.5. Adeno-Associated Viral Vector Technology: Leading Developers

7. DRUG PROFILES7.1. Chapter Overview7.2. Marketed Gene Therapies7.2.1. LUXUTRNA (Spark Therapeutics)7.2.1.1. Company Overview7.2.1.2. Development Timeline7.2.1.3. Mechanism of Action7.2.1.4. Target Indication(s)7.2.1.5. Current Status of Development7.2.1.6. Manufacturing, Dosage and Sales7.2.2. ZOLGENSMA (Novartis)7.2.2.1. Company Overview7.2.2.2. Development Timeline7.2.2.3. Mechanism of Action7.2.2.4. Target Indication(s)7.2.2.5. Current Status of Development7.2.2.6. Manufacturing, Dosage and Sales7.3. Late Stage (Phase II/III and Above) Gene Therapies7.3.1. AGTC 501: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results7.3.2. LYS-SAF302: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results7.3.3. NFS-01: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results7.3.4. RGX-314: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results7.3.5. AMT-061: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results7.3.6. OAV-101: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results7.3.7. PF-06838435: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results7.3.8. PF-06939926: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results7.3.9. PF-07055480: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results7.3.10. SPK-8011: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results7.3.11. SRP-9001: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results7.3.12. BMN 270: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results7.3.13. Lumevoq (GS010): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results7.3.14. PTC-AADC: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results

8. COMPANY PROFILES8.1. Chapter Overview8.2. Abeona Therapeutics8.2.1. Company Overview8.2.2. Financial Information8.2.3. AAV Vector Manufacturing related Capabilities8.2.4. Recent Developments and Future Outlook8.3 Aldevron (Acquired by Danaher)8.3.1. Company Overview8.3.2. Financial Information8.3.3. AAV Vector Manufacturing related Capabilities8.3.4. Recent Developments and Future Outlook8.4 Oxford BioMedica8.4.1. Company Overview8.4.2. Financial Information8.4.3. AAV Vector Manufacturing related Capabilities8.4.4. Recent Developments and Future Outlook8.5 Sanofi (CEPiA, Sanofi Pasteur, Genzyme)8.5.1. Company Overview8.5.2. Financial Information8.5.3. AAV Vector Manufacturing related Capabilities8.5.4. Recent Developments and Future Outlook8.6. WuXi AppTec8.6.1. Company Overview8.6.2. Financial Information8.6.3. AAV Vector Manufacturing related Capabilities8.6.4. Recent Developments and Future Outlook8.7. YPOSKESI8.7.1. Company Overview8.7.2. AAV Vector Manufacturing related Capabilities8.7.3. Recent Developments and Future Outlook

9. COMPANY COMPETITIVENESS ANALYSIS9.1. Chapter Overview9.2. Methodology and Key Parameters9.3. Adeno-Associated Viral Vector Manufacturers: In-house Manufacturers9.3.1. Players based in North America9.3.2. Players based in Europe9.4. Adeno-Associated Viral Vector Manufacturers: Contract Manufacturing Organizations9.4.1. Players based in North America9.4.2. Players based in Europe9.4.3. Players based in Asia-Pacific9.5. Adeno-Associated Viral Vector Manufacturers: Both In-House and Contract Manufacturing Organizations9.5.1. Players based in North America9.5.2. Players based in Europe9.5.3. Players based in Asia-Pacific

10. TECHNOLOGY COMPETITIVENESS ANALYSIS10.1. Chapter Overview10.2. Methodology and Key Parameters10.3. Adeno-Associated Viral Vector Technology Platforms10.3.1. Adeno-Associated Viral Vector Technology Platforms Developed by Companies10.3.2. Adeno-Associated Viral Vector Technology Platforms Developed by Companies based in Europe and Asia-Pacific

11. CLINICAL TRIAL ANALYSIS11.1. Chapter Overview11.2. Scope and Methodology11.3. Adeno-Associated Viral Vector Based Therapies: Clinical Trial Analysis

12. PARTNERSHIPS AND COLLABORATIONS12.1. Chapter Overview12.2. Partnership Models12.3. Adeno-Associated Viral Vector based Therapies: List of Partnerships and Collaborations12.4. Adeno-Associated Viral Vector Manufacturers: List of Partnerships and Collaborations

13. STRATEGIC PARTNER ANALYSIS

14. PATENT ANALYSIS

15. START-UP HEALTH INDEXING

16. OUTSOURCING: GO / NO-GO FRAMEWORK

17. MARKET SIZING AND OPPORTUNITY ANALYSIS

18. CONCLUDING REMARKS

19. EXECUTIVE INSIGHTS

20. APPENDIX 1: TABULATED DATA

21. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS

For more information about this report visit https://www.researchandmarkets.com/r/6ev168

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Worldwide AAV Vector Industry Report 2022 - by Type of Therapy, Type of Gene Delivery Method Used, Target Therapeutic Area, Application Area, Scale of...

Researchers have been experimenting with different ways to deliver genes to the brain to treat central nervous system diseases and tumors. One of the obstacles, however, is the ability to penetrate the blood-brain barrier while having minimal effect on the other organs in the body.

Hong Chen, associate professor of biomedical engineering at the McKelvey School of Engineering and of radiation oncology at the School of Medicine, both at Washington University in St. Louis, and her team found an effective method to overcome that obstacle using focused ultrasound intranasal delivery (FUSIN). In new research, they found that the intranasally delivered gene therapy had comparable or better outcomes than existing methods while having minimal effect on the bodys other organs.

Results of the research, led by Chen and Dezhuang Ye, a postdoctoral research associate, and collaborators, were published online in the journal eBioMedicineSept. 21. It is the first study to evaluate the potential of FUSIN to deliver adeno-associated viral vectors, small viruses used to deliver gene therapy, in a mouse model.

Read more on the engineering website.

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A sound approach for effective gene therapy delivery to brain - The Source - Washington University in St. Louis - Washington University in St. Louis

Following is a summary of current health news briefs.

Serum Institute to produce Ebola vaccine for use in Uganda outbreak

The Serum Institute of India plans to manufacture 20,000 to 30,000 doses of an experimental Ebola vaccine by the end of November for use in trials against an outbreak in Uganda, its developers and a company source said. The response to Uganda's outbreak has been blunted by the absence of a proven vaccine against the Sudan strain of the virus.

Eli Lilly to buy gene therapy company Akouos for $487 million

Eli Lilly and Co will acquire genetic medicine developer Akouos Inc for about $487 million in cash as the drugmaker aims to bolster its arsenal of gene therapies that target a range of disabilities. Shares of Akouos, which is developing gene therapies for sensorineural hearing loss, soared 84% to trade 40 cents above Lilly's offer price of $12.50 before the opening bell.

J&J results beat estimates on cancer drug strength

Johnson & Johnson on Tuesday beat Wall Street estimates for quarterly revenue and profit on strong demand for its cancer drug Darzalex while projecting an easing of supply-chain pressures next year on its consumer unit. The U.S. health conglomerate, which also sells medical devices and consumer health products such as Band Aid bandages and painkiller Tylenol, tightened its full-year adjusted profit forecast range. Its shares rose 1.5% to $169.01 in premarket trading.

Moderna signs deal on variant-adapted COVID shots for world's poorest

Moderna Inc (MRNA.O) has agreed to provide its new variant-adapted COVID-19 vaccine to the global scheme aiming to deliver shots to the world's poorest people. The biotech company and vaccine alliance GAVI will cancel their existing supply deal for vaccines based on the original coronavirus strain. Instead, Moderna will supply up to 100 million doses of its new, variant-adapted vaccines at its lowest available price from 2023.

S.Korea's LG Chem to invest $566 million to acquire U.S. Aveo Pharmaceuticals

South Korea's LG Chem Ltd said in a regulatory filing on Tuesday that it is investing $566 million to acquire U.S. firm Aveo Pharmaceuticals Inc and enter the U.S. anti-cancer drug market. The deal is expected to close in the first quarter of 2023, LG Chem said.

Bill & Melinda Gates Foundation pledge $1.2 billion to eradicate polio

Bill & Melinda Gates Foundation said on Sunday that it will commit $1.2 billion to support efforts to end all forms of polio globally. Polio is a highly infectious disease spread mainly through contamination by faecal matter, used to kill and paralyse thousands of children annually. While there is no known cure, three injections of the vaccine provide nearly 100% immunity.

U.S. nears record poultry deaths from bird flu; virus type complicates fight

A near-record number of U.S. chickens and turkeys have died in this year's outbreak of avian flu, as a different form of the virus than farmers battled before has infected more wild birds that then transmit the disease, officials said. More than 47 million birds have died due to infections and cullings. This has spurred export bans, lowered egg and turkey production, and contributed to record prices of the staples ahead of the U.S. holiday season. The outbreak exacerbates economic pain for consumers grappling with soaring inflation.

Trump administration blocked CDC transit mask mandate, report shows

Former President Donald Trump's administration at a crucial time in the COVID-19 pandemic in 2020 blocked the U.S. Centers for Disease Control and Prevention (CDC) from adopting a federal mandate requiring face masks on airline flights and other forms of transit, a congressional report released on Monday said. Marty Cetron, a senior CDC official, is cited in the report as saying the federal public health agency began working on the proposed order in July 2020 after its experts determined that there was scientific evidence to support requiring masks in public and commercial transportation.

Hair-straightening products linked with uterine cancer risk -study

Hair-straightening products may significantly increase the risk of developing uterine cancer among those who use them frequently, a large study published on Monday suggests. "We estimated that 1.64% of women who never used hair straighteners would go on to develop uterine cancer by the age of 70, but for frequent users, that risk goes up to 4.05%," study leader Alexandra White of the U.S. National Institute of Environmental Health Safety (NIEHS) said in a statement.

Addiction drug shows promise lifting long COVID brain fog, fatigue

Lauren Nichols, a 34-year-old logistics expert for the U.S. Department of Transportation in Boston, has been suffering from impaired thinking and focus, fatigue, seizures, headache and pain since her COVID-19 infection in the spring of 2020. Last June, her doctor suggested low doses of naltrexone, a generic drug typically used to treat alcohol and opioid addiction. After more than two years of living in "a thick, foggy cloud," she said, "I can actually think clearly." Researchers chasing long COVID cures are eager to learn whether the drug can offer similar benefits to millions suffering from pain, fatigue and brain fog months after a coronavirus infection. The drug has been used with some success to treat a similar complex, post-infectious syndrome marked by cognitive deficits and overwhelming fatigue called myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS). Drawing on its use in ME/CFS and a handful of long COVID pilot studies, there are now at least four clinical trials planned to test naltrexone in hundreds of patients with long COVID, according to a Reuters review of Clinicaltrials.gov and interviews with 12 ME/CFS and long COVID researchers. It is also on the short list of treatments to be tested in the U.S. National Institutes of Health's $1 billion RECOVER Initiative, which aims to uncover underlying causes and find treatments for long COVID, advisers to the trial told Reuters.

(With inputs from agencies.)

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Health News Roundup: Serum Institute to produce Ebola vaccine for use in Uganda outbreak; Eli Lilly to buy gene therapy company Akouos for $487...

LX1004 is an adeno-associated virus (AAV) based gene therapy for the treatment of CLN2 Batten disease

In a completed Phase 1/2 clinical trial, LX1004 demonstrated a favorable safety profile and showed statistically significant reduction in rate of decline compared to natural history studies

NEW YORK, Oct. 18, 2022 (GLOBE NEWSWIRE) -- LEXEO Therapeutics (LEXEO), a clinical-stage biotech company advancing a pipeline of adeno-associated virus (AAV)-based gene therapy candidates for genetically defined cardiovascular and central nervous system (CNS) diseases, today announced that the European Commission has granted Orphan Drug Designation to LX1004 for the treatment of CLN2 Batten disease. LX1004 is an AAV-mediated gene therapy designed to deliver a fully functional CLN2 gene to the CNS via intracisternal injection to restore TPP1, the secreted protein that is deficient in patients with CLN2 Batten disease.

CLN2 Batten disease is a fatal autosomal recessive lysosomal storage disease of early childhood caused by a mutation in the CLN2 gene, which results in cognitive impairment, blindness, seizures, and loss of motor function and leads to death at a young age.

An End of Phase 1/2 Meeting with the FDA will be held by the end of 2022. LEXEO has previously been granted Rare Pediatric Disease and Orphan Drug designations by the FDA.

About LEXEO Therapeutics

LEXEO Therapeutics is a New York City-based, clinical-stage gene therapy company focused on addressing some of the most devastating genetically defined cardiovascular and central nervous system diseases affecting both larger-rare and prevalent patient populations. LEXEOs foundational science stems from partnerships and exclusive licenses with leading academic laboratories at Weill Cornell Medical College and the University of California, San Diego. LEXEO is advancing a deep and diverse pipeline of AAV-based gene therapy candidates in rare cardiovascular diseases, APOE4-associated Alzheimers disease, and CLN2 Batten disease, and is led by pioneers and experts with decades of collective experience in genetic medicines, rare disease drug development, manufacturing, and commercialization. For more information, please visit http://www.lexeotx.com or LinkedIn.

Media Contact:

Evan FeeleyEvoke Canale for LEXEO(619) 849-5392 evan.feeley@evokegroup.com

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LEXEO Therapeutics Receives Orphan Drug Designation for LX1004 from European Commission - BioSpace

Researchers from Trinity College Dublin have developed a new gene therapy approach that shows promise for treating the dry form of age-related macular degeneration (AMD) a progressive eye disease that affects up to 10% of adults over 65 years of age and is a leading cause of severe vision impairment and blindness in this age group.

According to a Trinity College Dublin news release, dry AMD cases represent around 85% to 90% of all AMD cases and there are no treatments available to treat or prevent disease progression,underlining the need for developing treatment options for this debilitating disease.

In adults, many diseases of aging have been found to have defects of mitochondrial function, including AMD.

The news release noted that the team, in Trinitys School of Genetics and Microbiology, developed a new gene therapy (ophNdi1) that is the first of its kind to directly target mitochondrial function in cells that are malfunctioning in AMD. Mitochondria are known as the powerhouses of the cell because they manage the production of energy but their performance dips greatly in dry AMD and this is linked to a deterioration in sight.

The new gene therapy cleverly uses a virus to access the cells that are suffering and deliver the code needed to give the failing mitochondria a lifeline, enabling them to generate extra energy and continue to function in supporting vision.

Jane Farrar, PhD, the studys senior author, noted that the therapy has shown benefit in multiple models of dry AMD, offering hope that it could one day progress to a treatment that could help millions across the globe.

Critically, this study provides the first evidence in models that directly modulating bioenergetics in eye cells can provide benefit and improve visual function in dry AMD, Farrar said in a news release. In doing so, the study highlights the energy powerhouses of the cell, mitochondria, as key targets for dry AMD.

Sophia Millington-Ward, PhD, first author and research fellow in Trinitys School of Genetics and Microbiology, pointed out that the novel gene therapy targeting cellular energy, or mitochondrial function, that investigators explored for dry AMD consistently provided benefit in the model systems tested.

Many retinal cells, essential for vision, require particularly high levels of energy compared to most other cells, which makes them particularly vulnerable to mitochondrial dysfunction, she said. The therapy we are developing directly targets mitochondrial function and increases energy production levels in the retina, which leads to better visual function in disease models of dry AMD.

Millington-Wardnoted that although there is further work to be done before this could be made available as a treatment for patients, the results give us hope that we are getting closer to a solution to this challenging, debilitating condition.

The Research team in Trinity is funded by Enterprise Ireland and the European Regional Development Fund under Irelands European Structural and Investment Funds programme 1014-2020, Science Foundation Ireland, Fighting Blindness Ireland Health Research Charities Ireland, EU Marie Curie Innovative Training Network, Health Research Board Ireland, Health Research Charities and the Irish Research Council (StarT).

Reference

1. Sophia Millington-Ward, PhD; Jane Farrar, PhD; Arpad Palfi; G.J. Farrar; Naomi Chadderton; Laura K. Finnegan; Iris J.M. Post; Matthew Carrigan Tom Gardiner; Elisa Peixoto, et.al; AAV-mediated gene therapy improving mitochondrial function provides benefit in age-related macular degeneration models. Clinical and Translational Medicine. Published August 23, 2022. https://doi.org/10.1002/ctm2.952.

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Trinity team's new gene therapy shows promise for treating eye condition affecting millions across the globe - Ophthalmology Times

The Pitt BioForge Biomanufacturing Center will be fully equipped with its enabling technologies, including gene editing, induced pluripotent stem cell (iPSC) and cell, vector, and protein engineering capabilities.

The new facility is expected to generate more than 170 permanent full-time jobs, 900 construction jobs and 360 off-site support jobs.

This announcement supports the region's rise as a leader in cell and gene therapy and advances our vision of bringing an entirely new commercial manufacturing sector to the area," said Patrick Gallagher, Chancellor of the University of Pittsburgh.

In November last year, the Richard King Mellon Foundation announced a $100m grant to the University of Pittsburgh to create the Pitt BioForge Biomanufacturing Center: representing the largest single-project grant in the Foundation's 75-year history.

Now, the 30-year agreement with Cambridge, Massachusetts cell and gene therapy developer ElevateBio will see ElevateBio extend its manufacturing footprint to Pittsburgh, building on its first BaseCamp biomanufacturing facility in Waltham, Massachusetts (the companys new disruptive business model addresses challenges in CGT with a suite of technologies; alongside the BaseCamp manufacturing capability; to create an end-to-end technology platform offering).

To realize our vision of transforming the cell and gene therapy field for decades to come, broadening our footprint across metropolitan areas is a key priority for us, and we are thrilled that the University of Pittsburgh will be home to one of our BaseCamp facilities, said David Hallal, Chairman and Chief Executive Officer of ElevateBio. Weve identified Pittsburgh as an ideal location to extend our BaseCamp presence as it sits at the intersection of science, technology, and talent.

Pitt Senior Vice Chancellor for the Health Sciences, Dr. Anantha Shekhar, said the University of Pittsburgh a top recipient of NIH research funding has exceptional emerging research but to date has lacked access to high-quality process science and manufacturing capabilities.

As we position ourselves to become the next global hub for life sciences and biotech, we were in search of the right partner to help us realize our vision, and ElevateBios expertise and reputation in cell and gene therapy made them the perfect partner to accelerate our ability to build our biomanufacturing center of excellence.

Sam Reiman, Director of the Richard King Mellon Foundation, added:This partnership between two national life-science powerhouses the University of Pittsburgh and ElevateBio - is a consequential step forward in realizing our shared vision to make Pittsburgh a national and international biomanufacturing destination.

Pitt BioForge is a generational opportunity to bring extraordinary economic-development benefits to our region, and life-changing cell and gene therapies to patients - distribution that will be accelerated and enhanced by Pitts partnership with UPMC. ElevateBio could have chosen to locate its next biomanufacturing hub anywhere in the world; the fact they are choosing to come to Pittsburgh is another powerful validation of our region, and the Pitt BioForge project at Hazelwood Green.

The Commonwealth of Pennsylvania and the County of Allegheny also provided incentive grants to ElevateBio in support of this partnership.

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New Pittsburgh biomanufacturing center to accelerate cell and gene therapy innovation - BioPharma-Reporter.com

Ovid Therapeutics has struck a deal with young biotechnology company Gensaic, hoping the startups method of delivering genetic medicines can yield new brain drugs.

Under the deal, the partners will develop up to three gene-based treatments for neurological conditions Ovid is targeting. The New York biotech will get rights to license any gene therapies that emerge from the deal, so long as the two can agree on terms. Ovid also invested $5 million in the startup and committed to participate in future financing rounds.

The deal is the latest step in a rebuilding plan for Ovid, a biotech former Teva and Bristol Myers Squibb executive Jeremy Levin formed seven years ago.

Levins plan in starting Ovid was to grab medicines overlooked elsewhere, license them and develop them for rare brain diseases. That strategy led Ovid to two medicines the company developed for Angelmans syndrome and rare forms of epilepsy, and helped the biotech to go public in 2017.

Ovid hasnt been successful, however. The Angelmans drug failed a Phase 3 trial in 2020, erasing more than half of the companys value. One year later, Ovid, aiming to bolster its dwindling cash reserves, sold rights to the epilepsy drug back to Takeda. Though Ovid can still receive milestone payments and royalties from the drug, which is now in late-stage testing, its only remaining in-house programs are in preclinical testing. At just over $2 apiece, shares trade near all-time lows.

Recently, Ovid has taken steps to restock its pipeline. One experimental medicine for treatment-resistant epileptic seizures could start human trials later this year, while a licensing deal with AstraZeneca and a related partnership with Tufts University could yield other drug candidates that might follow in 2024.

The alliance with Gensaic adds up to three more prospects, while pushing Ovid into the field of gene therapy.

Gensaic was seeded in 2021 as M13 Therapeutics and is currently housed in Cambridge, Massachusetts biotech startup incubator LabCentral. Over the past two years, the company has won awards in multiple startup competitions for its research into a method of gene therapy delivery designed to overcome the limitations of standard approaches.

Many gene therapies rely on modified viruses to send genetic instructions into the bodys cells. Those delivery vehicles are used in multiple products approved for rare inherited diseases, but they also come with weaknesses, too. One commonly used tool, the adeno-associated virus, can only carry a relatively small amount of genetic cargo and is sometimes shut down by the body. Another, the lentivirus, also has limited packaging capacity and has been linked in rare cases to the development of cancers.

Gensaic instead aims to use tiny particles derived from phages, the viruses that infect bacteria, to deliver genetic material. Gensaic claims these particles can be engineered to target multiple tissue types among them the lung and brain and can carry much larger genes. Gensaic believes they may have the potential to be administered more than once, too, though that hasnt yet been proven.

In a statement, Levin said the approach appears to be optimal for carrying substantial genetic cargo across the blood-brain barrier, a filtering mechanism the body uses to keep foreign substances out of the brain.

We believe it may hold the potential to treat a broad continuum of diseases in the brain, Levin said.

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Ovid turns to gene therapy startup to restock drug pipeline - BioPharma Dive

Steven Pipe, MD: What we've highlighted so far are the benefits of prophylaxis by replacing factor 9 [therapy] with an IV infusion. However, the limitation of that is the need for an IV to deliver that and then the limitations of maintaining sufficient levels so you don't have breakthrough bleeding. The promise of gene therapy is to achieve a steady state, a measurable level of factor that's above key thresholds that would also produce a steady state hemostatic effect sufficient to prevent bleedsall of this following a single treatment intervention. How do we achieve that? The first wave of gene therapies for hemophilia are adenocarcinoma-associated virus-based liver-directed approaches. What we do is a functional copy of factor 9 [therapy] is packaged inside a viral vector, these AV vectors, and the vector contains no propagating viral gene elements. Following a single outpatient IV infusion over as little as 13 hours, these viral particles that contain the factor 9 gene are picked up by the liver cell receptors, and they're taken into the cell. The vector particle uncoats, and it delivers the DNA to the nucleus of the cell. The genetic elements that accompany the gene allow for efficient expression and then secretion of factor 9 protein into the plasma. This reaches a steady state between the rate of secretion from the cell, as well as the clearance from the plasma. That's represented by a factor level that we can measure with traditional blood sampling. If we get those patients up to a sufficient level, they can come off prophylaxis. They may not need factor episodic doses related to trauma if they're at a high enough level, and we can allow them to leave. Hopefully, [this will] allow them to live the life that they choose related to their activity levels, the occupations they want to be involved in, and bring a lot of spontaneity back to their life.

Hemophilia A and hemophilia B gene therapy are similar in concept. However, there are some challenges related to hemophilia A, in particularly factor 8 expression using this strategy, that might make this easier for hemophilia B to come to the forefront of clinical care delivery. One of the biggest differences is the size of the gene itself. The factor 9 gene is considerably smaller than the factor 8 gene that's used for hemophilia A. In fact, the intact factor 8 gene is far too big to fit inside these AV vectors. We use a modified trans-gene in order to package it inside the vectors. In addition, factor 8 has some challenges related to how efficiently the cell can make factor 8. The other aspect, which is interesting from a biological perspective, is that factor 9 is naturally made in the hepatocyte of the cell. However, factor 8 is not primarily made in the hepatocyte; it's made in a number of different tissues in the body, primarily endothelial cells. Trying to express factor 8 within that hepatocyte, which, if you like a non-native cell, has become considerably more challenging. What has this meant in the observations from the trials? We've been getting more consistent and durable expression of factor 9 in the hemophilia B trials than we've seen in hemophilia A. Seeing this get approved at the regulatory level, and then also approved for reimbursement at the payer level, I [now] have a higher level of confidence that hemophilia B might achieve that earlier. We'll be able to incorporate that into our clinical care delivery earlier.

If gene therapy is actually able to come to the forefront as part of a clinical care delivery, this [will] be a game changer, at least for a proportion of the patients we have in our clinicsto be relieved from the need for regular prophylactic therapy, including the burden of the commitment, the need for IV infusions, the economic burden associated with the prophylaxis. You no longer have to worry about doing prophylaxis or even having breakthrough bleeding [from a single treatment event]. You can carry your life forward, not having to think about your hemophilia. These are all significant advantages of this treatment. There's going to be several patients who want to have that kind of transformation and live the life that they choose. We're going to see some limitations in that approach because this is going to be applied according to the eligibility and restrictions associated with clinical trials. Out of the gate, this will be a smaller proportion of patients who are going to benefit from this, a truly transformational treatment.

Transcript edited for clarity.

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Defining the Impact of Gene Therapy - Managed Healthcare Executive

SEATTLE, Aug. 24, 2022 /PRNewswire/ --According to Coherent Market Insights, the global cell and gene therapy market is estimated to be valued at US$ 15,580.3 million in 2022 and is expected to exhibit a CAGR of 24.7 % during the forecast period (2022-2030).

Key Trends and Analysis of the Global Cell and Gene Therapy Market:

Major players operating in market are focusing on adopting strategies such as collaboration for product development, which is expected to drive the market growth over the forecast period. For instance, in May 2021, Biogen, a U.S. based multinational biotechnology company and Ginkgo Bioworks, a U.S. based Biotechnology Company, announced a gene therapy collaboration. The collaboration aimed to redefine the industry standard for manufacturing recombinant adeno-associated virus (AAV)-based vectors.Recombinant adeno-associated virus AAV-based vectors are widely used to develop innovative gene therapies and have the potential to treat certain neurological and neuromuscular diseases as well as other conditions across multiple therapeutic areas.

Increasing collaborations between key market players is expected to drive growth of the global cell and gene therapy market over the forecast period. For instance, in December 2021, BioMarin Pharmaceutical Inc., a U.S. based Biotechnology Company and Skyline Therapeutics, a gene and cell Therapy Company focused on developing novel treatments for unmet medical needs, announced a global strategic collaboration for the discovery, development and commercialization of Adeno-Associated Virus (AAV) gene therapies to treat genetic cardiovascular diseases. The partnership would help Skyline Therapeutics integrated AAV gene therapy platform based on its proprietary vector engineering and design technology and manufacturing capability to develop innovative gene therapies with a focus on genetic dilated cardiomyopathies (DCM), a group of progressively advancing, devastating diseases with no targeted treatment options. Under the agreement, BioMarin and Skyline Therapeutics would collaborate on discovery and research through to an Investigational New Drug Application (IND).

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Key Market Takeaways:

Increasing inorganic strategies by the key market players is expected to boost the global cell and gene therapy market growth over the forecast period. For instance, in November 2021,Entos Pharmaceuticals, Inc., a clinical-stage biotechnology company developing genetic medicines with its Fusogenix proteolipid vehicle (PLV) nucleic acid delivery platform, and BioMarin Pharmaceutical Inc., a U.S. based Biotechnology Company, announced an agreement that would see Entos apply its Fusogenix nucleic acid delivery technology to gene therapy candidates in the BioMarin pipeline. Under the agreement, Entos would create specially formulated product candidates for BioMarin. Under the agreement, Entos would use its Fusogenix nucleic acid delivery system to specially formulate BioMarin products directed at multiple undisclosed genetic disease indications. BioMarin would conduct preclinical studies of the Fusogenix-formulated candidates to evaluate their potential as therapies to prevent or treat these conditions.

Among Therapy Type, the gene therapy segment held a dominant position in the cell and gene therapy market in 2022. For instance, according to the Catalyst report, in December 2018, around 289 novel cell and gene therapies were in development for a variety of diseases, of which around 111 medicines were for cancer and around 28 medicines were for cardiovascular disease. These medicines in development were either in clinical trials or awaiting for approval from the U.S. Food and Drug Administration (FDA).

Among region, North America is expected to hold a dominant position in the global cell and gene therapy market over the forecast period. For instance, according to the U.S. Pharmacist Journal, January 2018, annually, around 1.2 million adults are diagnosed with early symptoms of brain disorders, of which 21% are due to Alzheimer's disease, and the total number of new cases of Parkinson's disease and traumatic brain injuries is around 135 million in the U.S.

Key players operating in the global cell and gene therapy market include Amgen, Biogen, BioMarin Pharmaceuticals, Bristol-Myers Squibb Company, GlaxoSmithKline, Novartis, Pfizer, Regeneron Pharmaceuticals Inc, Spark Therapeutics, Agilis Biotherapeutics, Angionetics AVROBIO, Freeline Therapeutics, Horama, MeiraGTx, Myonexus Therapeutics, Nightstar Therapeutics, Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., MEDIPOST, and Bluebird Bio. Inc.

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Detailed Segmentation:

Global Cell and Gene Therapy Market, By Therapy type:

Global Cell and Gene Therapy Market, By Indication:

Global Cell and Gene Therapy Market, By Scale of Operation:

Global Cell and Gene Therapy Market, By Region:

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Middle East and Asia Pacific Cell and Gene Therapy Market, by Therapy Type (Cell Therapy (Stem Cells, T Cells, Dendritic Cells, and NK Cells) and Gene Therapy (Germline Gene Therapy and Somatic Gene Therapy)), by Indication (Dermatology, Musculoskeletal, Oncology, Immunology, Cardiology & Neurology, and Others), by Technology (Lentiviral Vector, Plasmid DNA, and AAV), and by Region (Middle East and Asia Pacific) - Size, Share, Outlook, and Opportunity Analysis, 2022 - 2028

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Global Cell and Gene Therapy Market to Surpass US$ 90,984.2 Million by 2030 - Coherent Market Insights - PR Newswire