Category Archives: Genetic Therapy

BOSTON--(BUSINESS WIRE)--Emulate, Inc., a leading provider of next-generation in vitro models, today announced the launch of its new adeno-associated virus (AAV) transduction application for the Liver-Chip that enables gene therapy researchers to test the delivery efficiency and safety of AAV vectors in a validated, human-relevant model of the liver and get results in weeks, not months, as with animal models. This technology allows them to rapidly iterate on AAV design to optimize delivery of gene therapies and accelerate development.

The ability to more rapidly optimize AAV design using this application is a game-changer for the gene therapy industry, said Emulate CEO Jim Corbett. We are excited to offer this novel application to researchers in biopharmaceutical, academic, and government entities around the world who are exploring gene therapy as a potential to treat a wide range of diseases such as cancer, cystic fibrosis, heart disease, diabetes, and metabolic disease.

Gene therapy involves replacing a faulty or missing gene by adding a new one inside the bodys cells to treat or prevent a genetic disease or disorder. Currently, this technique is primarily available in clinical trials, testing its potential to treat inherited disorders, cancer, and HIV/AIDS. The AAV vector is the most versatile and popular viral vector that researchers use as a delivery vehicle for gene therapy, as it efficiently targets different cell and tissue types and has been demonstrated to be safe and well-tolerated.

Due to the lack of suitable non-clinical models, scientists often struggle when designing new AAV vectors to ensure that the vector effectively and safely delivers the therapy to the right cells, in the right organ. Animal models are slow, costly, and tightly regulated, which limits the number of AAV delivery vehicles that can be tested and the ability to look at the individual contribution of each cell type. In addition, conventional in vitro models restrict the number of AAVs that can be tested as they are only a single cell type in a static petri dish and do not accurately reflect how cells behave inside the body.

The Emulate Liver-Chip provides the specific 3D multicellular architecture, physiological functions and mechanical forces necessary to recapitulate the relevant aspects of the liver, said Emulate Chief Scientific Officer Lorna Ewart, PhD. Now we have demonstrated it to be a more human-relevant model that researchers can use to assess and discriminate between the transduction efficiency of various AAV-based gene therapies in a concentration- and time-dependent manner, as well as evaluate the toxicity.

Information about the Emulate AAV transduction application for the Liver-Chip is available on the companys website and will be presented in a live webinar on October 6th.

About Emulate, Inc.

Emulate is igniting a new era in human health with industry-leading Organ-on-a-Chip technology. The Human Emulation System provides a window into the inner workings of human biology and diseaseoffering researchers an innovative technology designed to predict human response with greater precision and detail than conventional cell culture or animal-based experimental testing. Pioneered at the Wyss Institute for Biologically Inspired Engineering at Harvard University and backed by Northpond Ventures, Founders Fund, and Perceptive Advisors, Organ-on-a-Chip technology is assisting researchers across academia, pharma, and government industries through its predictive power and ability to recreate true-to-life human biology. To learn more, visit emulatebio.com or follow us on LinkedIn and Twitter.

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Emulate Announces New Gene Therapy Application Enabling Accelerated Development of Treatments for Genetic Diseases with Organ-on-a-Chip Technology -...

Rejuvenate Bio is a gene therapy startup that seeks to reverse aging. In response to emailed questions, CEO and Co-founder Daniel Oliver talked about how he and his co-founder started the company and their vision for treating age-related conditions.

Why did you start this company?

The idea was formed in the George Church lab at Harvard Medical School. Our Co-founder and Chief Scientific Officer Noah Davidsohn was doing his postdoc in Georges lab where he got his dog Bear, his best friend. He immediately decided that he needed to make him live a longer healthier life because 10-12 years just isnt long enough.

Noah realized that these treatments were applicable across both animal and human health. As the science progressed, it was clear that the only way to properly advance this technology and create novel therapeutics to help both pets and human patients was to form a company to move it from the bench to the bedside and into the real world. I joined from another startup to help commercialize Rejuvenate Bios current technology.

How did you meet your co-founder?

We are friends from college at Caltech. We met at a point in life where the most important thing on our minds was making sure we got our applied mathematics class problem sets done before Thursday so we could hang out with our friends! We have been friends ever since, and we luckily took complimentary paths where Noah went the traditional science route and I went to business school.

Daniel Oliver

What need/problem are you seeking to address in healthcare?

Aging costs our healthcare system in the U.S. $1.3 trillion dollars annually. Aging is driven by a dysregulation in gene expression. You have the same DNA the day you are born as you do the day you die, but what genes are turned on or off change dramatically through time. These changes can be beneficial. They drive one through development and leave people in early adulthood, but there is no evolutionary mechanism to keep these systems regulated long term. That is why we see very similar changes in gene regulation patterns across humans and subsequently see similar prevalence of age-related conditions.

What does your technology do?

At Rejuvenate Bio, we are using combination gene therapy to reverse aging in people, increasing their quality of life while treating multiple age-related conditions. Rejuvenate Bio is creating gene therapies to address issues of gene expression. By utilizing gene therapy Rejuvenate Bio can precisely deliver genetic material to patients to re-regulate certain genes whose expression levels change through age.

Rejuvenate Bios technology originated in the lab of co-founder George Church, Ph.D., as part of co-founder Noahs postdoctoral research into the genetics of aging. Our initial pipeline products are based on three longevity genes with proven efficacy and unprecedented safety profiles validated by big pharma: FGF21, sTGFR2, and Klotho are the cornerstone of Rejuvenate Bios approach. They have previously been shown to increase health and lifespan in mice that were bred to overexpress them. By delivering combinations of these three genes we have treated multiple models of age-related disease, heart failure, kidney failure, diabetes and obesity in mice and heart failure and obesity in large animals.

We have also created key tools for the delivery and control of epigenetic modifying factors that hold the promise of re-regulating the entire gene expression profile.

What is your lead therapeutic?

RJB-01 is a combination liver directed AAV gene therapy that expresses the secreted factors FGF21 and TgfBr2. RJB-01 has shown the ability to increase heart function and decrease damage against three types of heart failure in animals. RJB-01 has also been shown to have positive metabolic effects across both mice and dogs. RJB-01 will initially go into cynical trials for Arrhythmogenic Right Ventricular Cardiomyopathy(ARVC) and Familial Partial Lipodystrophy (FPL). As RJB-01 enters clinical trials for humans, a canine version will become available for the treatment of Mitral Valve Disease in dogs through a partnership with Phibro Animal Health.

Do you have any clinical validation of your product?

Weve conducted extensive preclinical studies in mice and dogs. We now have more than two years of safety data in dogs as well as multiple experiments demonstrating the efficacy of the treatment in both cardiac and metabolic conditions.

What are your upcoming milestones?

We are driving towards initial clinical trials with RJB-01 and commercialization of the canine version of RJB-01.

Photo: tylim, Getty Images

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Rejuvenate Bio CEO highlights ambitious approach of using gene therapy to reverse aging - MedCity News

DUBLIN--(BUSINESS WIRE)--The "Hemophilia Gene Therapy Market, by Hemophilia Type, and by Region - Size, Share, Outlook, and Opportunity Analysis, 2022 - 2030" report has been added to ResearchAndMarkets.com's offering.

Hemophilia is a genetic bleeding disorder in which an individual lacks or has low levels of proteins called clotting factors. There are around 13 types of clotting factors that work with blood platelets, which are necessary for clotting process to initiate. There are three forms of hemophilia - A, B, and C.

Hemophilia A is the most common form and is caused due to deficiency in clotting factor VIII. Hemophilia B occurs due to deficiency of clotting factor IX and Hemophilia C is caused due to clotting factor XI deficiency. Hemophilia is incurable with current therapeutic options, which only reduces symptoms such as spontaneous bleeding in muscles and joints as well as increased risk for intracranial hemorrhage.

These treatment options lasts only for a day and is costly. As against conventional clotting factor replacement therapy, gene therapy is expected to offer sustainable cure for hemophilia by correcting defective gene sequence (F8 or F9 gene) that codes for clotting factor VIII or IX in the patient's body.

In this therapy, recombinant Adeno - associated virus is most commonly utilized to deliver a codon optimized version of the clotting factors genes (VIII or IX) to patients affected by hemophilia. This gene is shortened by deleting a discrete portion of the gene to better fit the coding sequence into the viral vector. There is dose dependent gene expression level observed which means genetic expression for clotting factor increases with increased dose of gene therapy.

Market Dynamics

Regulatory bodies such as the U.S. Food and Drug Administration (FDA) are supporting the product development for gene therapy by allotting breakthrough designation status to investigational gene therapy.

For instance, in 2017, the U.S. FDA granted breakthrough therapy designation to valoctocogene roxaparvovec, which is an Adeno -associated virus 5 factor VIII vector designed to restore factor VIII plasma concentrations in patients with hemophilia A.

Key features of the study:

Detailed Segmentation: Global Hemophilia Gene Therapy Market, By Hemophilia Type:

Global Hemophilia Gene Therapy Market, By Region:

Company Profiles

Key Topics Covered:

1. Research Objectives and Assumptions

2. Market Purview

3. Market Dynamics, Regulations, and Trends Analysis

4. Global Hemophilia Gene Therapy Market - Impact of Coronavirus (Covid-19) Pandemic

5. Global Hemophilia Gene Therapy Market, By Hemophilia Type, 2017 - 2030, (US$ Million)

6. Global Hemophilia Gene Therapy Market, By Region, 2017 - 2030, (US$ Million)

7. Competitive Landscape

8. Section

For more information about this report visit https://www.researchandmarkets.com/r/dodq7x

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Global Hemophilia Gene Therapy Market 2022: Advantages Over Traditional Treatments Driving Growth - ResearchAndMarkets.com - Business Wire

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to AVR-RD-04, an investigational gene therapy for the treatment of cystinosis, a life-threatening disease that causes progressive multi-organ damage, including early, acute kidney disease progressing to end-stage kidney disease.

FDAs Rare Pediatric Disease Designation and Voucher Program is intended to facilitate the development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. Companies that receive approval for a New Drug Application (NDA) or Biologics License Application (BLA) for a rare pediatric disease may be eligible to receive a voucher for a priority review of a subsequent marketing application for a different product. The priority review voucher may be used by the company or sold to a third party.

AVR-RD-04 is designed to genetically modify patients own hematopoietic stem cells (HSCs) to express the gene encoding cystinosin, the protein that is critically deficient in people living with cystinosis.

Preliminary data from the ongoing University of California San Diego Phase 1/2 clinical trial suggest that this approach is well tolerated, with no adverse events (AEs) related to the drug product reported to date. All AEs reported were related to myeloablative conditioning, stem cell mobilization, underlying disease or pre-existing conditions. The majority of AEs were mild or moderate and resolved without clinical sequelae. Clinical data to date indicate this investigational approach provides benefits in multiple tissues evaluated, including the eyes, skin, gastrointestinal mucosa and the neurocognitive system. The collaborator-sponsored Phase 1/2 clinical trial is funded in part by grants to University of California San Diego from the California Institute for Regenerative Medicine (CIRM), Cystinosis Research Foundation (CRF) and National Institutes of Health (NIH).

About CystinosisCystinosis is a rare, progressive disease that impacts approximately 1,600 patients in the U.S., Europe and Japan and is marked by the accumulation of cystine in cellular organelles known as lysosomes. Untreated cystinosis is fatal at an early age. The current SOC for cystinosis, a treatment regimen that can require dozens of pills per day, does not prevent overall disease progression and carries side effects, such as breath and body odor and gastrointestinal symptoms, which can impede compliance. More than 90% of treated cystinosis patients require a kidney transplant in the second or third decade of life.

About AVROBIOOur vision is to bring personalized gene therapy to the world. We target the root cause of genetic disease by introducing a functional copy of the affected gene into patients own hematopoietic stem cells (HSCs), with the goal to durably express the therapeutic protein throughout the body, including the central nervous system. Our first-in-class pipeline includes clinical programs for cystinosis and Gaucher disease type 1, as well as preclinical programs for Gaucher disease type 3, Hunter syndrome and Pompe disease. Our proprietary plato gene therapy platform is designed to be scaled to support late-stage clinical development and commercialization globally. We are headquartered in Cambridge, Mass. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

Forward-Looking StatementsThis press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as aims, anticipates, believes, could, designed to, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will, and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our preclinical and clinical product candidates, including AVR-RD-04 for the treatment of cystinosis, the potential benefits and incentives provided by FDAs rare pediatric disease designation for AVR-RD-04, the design, commencement, enrollment and timing of planned clinical trials, preclinical or clinical trial results, product approvals and regulatory pathways, our plans and expectations with respect to interactions with regulatory agencies, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success, the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs, and the expected safety profile of our preclinical and investigational gene therapies. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, including beneficial effects seen in multiple organs and tissues, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our enrollment and development timelines and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Annual or Quarterly Report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

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AVROBIO Receives Rare Pediatric Disease Designation from U.S. Food and Drug Administration (FDA) for First Gene Therapy in Development for Cystinosis...

Visiongain Reports Ltd

Visiongain has published a new report entitled Gene Therapy R&D 2022-2032. It includes profiles of Gene Therapy R&D and Forecasts Market Segment by Disease {Cancer, Rare Diseases (Oncologic, Non-oncologic), Cardiovascular Diseases, Ophthalmic Diseases, Haematology, Neurological, Diabetes Mellitus, Other Diseases)}, Vector {Viral (Retrovirus, Adenovirus, AAV, Lentivirus, Others), Non-viral (Naked DNA, Gene Gun, Electroporation, Lipofection)}, Techniques (Gene Augmentation Therapy, Gene Replacement Therapy), Participants (Small/Medium Pharma & Biotech, Universities & Research Institutes, Hospitals, Government & Public Bodies, Big Pharma) PLUS COVID-19 Impact Analysis and Recovery Pattern Analysis (V-shaped, W-shaped, U-shaped, L-shaped) Profiles of Leading Companies, Region and Country.

The gene therapy R&D market was valued at US$1,653.0 million in 2021 and is projected to grow at a CAGR of 30.1% during the forecast period 2022-2032.

Gene Therapies Are Projected to Provide Potential Benefits for a Range of Rare DiseasesThere are about 7,000 rare diseases reported, but only a few hundred have therapies approved. Gene therapy is especially important for patients with rare disorders, as more than 80% of them have a documented monogenic (single-gene) cause. Rather than treating the disease, conventional small molecule medications often work by reducing symptoms. When managing a chronic condition, this may indicate that the medication or drugs used to control the condition are administered on a daily basis. Gene therapy, on the other hand, has the ability to remedy structural genetic disorders, rather than merely treating symptoms.

In October 2021, the U.S. FDA, National Institutes of Health (NIH), ten pharmaceutical companies & five non-profit groups joined forces to pace up the development of gene therapies for addressing the 30 million rare diseases patient pool across the North American region. Only two genetic disorders now have FDA-approved gene treatments, despite the fact that there are about 7,000 rare diseases. Hence, partnerships between pharmaceutical companies to tackle rare diseases is likely to fuel the demand for gene therapy in rare diseases treatment during the forecast period.

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Furthermore, gene therapies provide the potential of a one-time cure for a range of rare disorders for which there are actually no clear clinical alternatives. With multiple gene therapy drugs securing FDA clearance, recent developments in genetic engineering and recombinant viral vector production have fuelled interest in the field.

The Asia Pacific Has Witnessed an Increase in Early ApprovalsThe regulatory framework for supporting fast marketing authorizations for advanced medicines to address unmet medical needs has been developed by regulatory agencies as a result of the Asia-Pacific region's rapid growth in advanced therapy research and development. With the introduction of regulatory frameworks by the authorities, the region has witnessed an increase in early approvals of new medicines. These approvals showed that regional regulators are more prepared to review and authorize cutting-edge treatments. To introduce these cutting-edge medications into Asia-Pacific, numerous pharmaceutical companies are making use of these new regulatory paths to take a competitive edge in the market.

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Gene Therapy R&D Market Report 2022-2032

How has COVID-19 had a Significant Impact on the Gene Therapy R&D Market?All biopharmaceutical firms have been impacted by the COVID-19 pandemic, but many CGT companies have been hit particularly severely because of their complex manufacturing and distribution models and financial structures. Businesses' decisions will have a significant impact on both the present patients with CGT and those who stand to gain from the next wave of innovation being explored by CGT companies. The manufacturing and treatment supply of the CGT industry, as well as scientific and clinical advancement and business operations, have all been severely hampered by the COVID-19 problem. The COVID-19 impact, which has been more severe in some nations compared to others, has not affected some CGT enterprises very negatively. However, since the supply chains used to manufacture CGTs are complex and tightly regulated, CGT companies have discovered that they are especially susceptible to interruptions in regions where the new coronavirus has been widespread.

How this Report Will Benefit you?Visiongains 462 page report provides 169 tables and 228 charts/graphs. Our new study is suitable for anyone requiring commercial, in-depth analyses for the gene therapy R&D market, along with detailed segment analysis in the market. Our new study will help you evaluate the overall global and regional market for gene therapy R&D. Get the financial analysis of the overall market and different segments including service type, molecule type, and therapeutic area. We believe that high opportunity remains in this fast-growing gene therapy R&D market. See how to use the existing and upcoming opportunities in this market to gain revenue benefits in the near future. Moreover, the report would help you to improve your strategic decision-making, allowing you to frame growth strategies, reinforce the analysis of other market players, and maximise the productivity of the company.

What are the Current Market Drivers?

Increasing Investments Driving Market GrowthThe pandemic has highlighted the relevance of cell and gene therapies, as well as genetic medicines in specific. If the investment by venture capitalists maintains capital flows, the momentum will be maintained during the forecast years. Large biopharmaceutical firms are anticipated to invest or acquire innovative technologies & support valuation, even if the capital markets funding climate deteriorates; Visiongain anticipates that the gene therapy industry will continue to attract investor interest over the forecast period.

Even as private companies like enGene plan to go public, Generation Bio's valuation has grown to $2 billion due to non-viral gene therapy. Longer term, synthetic biology investments, such as transgene engineering, are expected. In cell-based treatment, we see more investment potential in solid tumors and off-the-shelf pluripotent stem cell technology.

Recognizing the promise of these cutting-edge developments, large pharmaceutical firms sought out partnerships with smaller, more agile biotech start-ups. Janssen (Johnson & Johnson) & Fate Therapeutics agreed to a US$100 million upfront deal to develop cell-based immunotherapies for hematologic and solid tumors. Biogen & Sangamo have agreed to a US$350 million upfront contract to create zinc finger protein-based gene regulation therapies for neurodegenerative diseases. These agreements aided in the receipt of US$3 billion in upfront fees from corporate alliances, as well as clinical and regulatory milestones worth billions more.

Technological Advancements to Fuel Market Growth Through 2032Gene therapy, both as a modern medical technique and as a biomedical business, has a bright future in terms of technology and industry promotion. Researchers may use genome editing technology to break, alter, and edit particular genes in a DNA sequence-specific manner. However, genome editing carries the possibility of unintentional editing of genes with identical DNA sequences, a phenomenon known as the off-target effect. Genome editing has the ability to create lasting changes in the genome.

Furthermore, the genome editing tool CRISPR-Cas9 is making waves in the scientific area. It has a wide range of possible uses and is quicker, less expensive, and more accurate than earlier methods of DNA editing. Animal research has been transformed by CRISPR/Cas9 technology, as has human gene therapy, medical research, and plant science study. This method has become increasingly useful in recent years for carrying out precise gene targeting and alterations, such as gene insertions and deletions, gene replacements, and single-base pair conversions. Over the forecast period, the market for gene therapy R&D is expected to grow as a result of significant breakthroughs in this field.

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Gene Therapy R&D Market Report 2022-2032

Where are the Market Opportunities?

Growing Number of Clinical Trials to Offer Lucrative Growth OpportunitiesWhile 2022 will be a significant year for gene treatments that target rare diseases, we also anticipate that clinical readouts on medicines that target common diseases will garner media attention. It was one of the pivotal events when Vertex Pharmaceuticals' cell therapy effectively cured one patient's type 1 diabetes in 2021. The first gene therapy approval for a prevalent illness in the U.S. & Europe may occur within the next several years due to Phase 3 studies for indications like congestive heart failure, critical limb ischemia, diabetic peripheral neuropathy & macular degeneration. Approximately, 59% of the 2,406 clinical studies in the area focus on prevalent diseases. Additionally, 62% of academic and government-sponsored studies are against 56% for commercial trials, demonstrating the industry's greater involvement in the study of rare diseases. In addition, when compared to university and government sponsors, the industry places more emphasis on rare haematological diseases like hemophilia and sickle cell anaemia as well as rare ophthalmological conditions like retinitis pigmentosa.

Nearly two-thirds of all trials for rare diseases focus on treating rare malignancies, which continue to be the main goal. Additionally, inherited haematological conditions like SCDs & hemophilia, ophthalmological indications like retinitis choroideremia & pigmentosa, and other rare monogenic disorders like mucopolysaccharidosis, Duchenne muscular dystrophy & Wilson disease have drawn interest from cell and gene therapy developers.

Even while the proportion of trials targeting both common and rare diseases is roughly similar over phases, the prominent diseases being targeted are evolving. Phase 3 studies that target a common disease include 23% of musculoskeletal problems, but just 7% of Phase 1 trials, including bone fractures, osteoarthritis, and sports injuries. Other common disease categories targeted include viral diseases like HIV and CNS disorders such as Alzheimer's & Parkinson's disease.

There is also a change in the predominant disease category toward focusing on more complicated, polygenic diseases. We are witnessing a gradual transition within the CNS disorders, from more complex, polygenic disorders like Alzheimer's disease, autism & even treatment-resistant bipolar disorder & depression, to conditions such as spinal cord injury (SCI), traumatic brain injury (TBI), and neuropathic pain.

Facility Expansion Anticipated to Offer Lucrative Growth ProspectsContract manufacturers, on whom new gene & cell therapy businesses rely for early-stage development, are experiencing a lack of viral vector manufacturing capacity as a result of the increase in clinical-stage start-ups. When these companies reach commercial scale, they frequently prefer to maintain total control over their manufacturing in order to avoid the difficulties of outsourcing. As a result, biotech firms began to create expansion plans, set up internal teams, and/or ask for site consultant guidance. These professionals support the strenuous search for suitable research and development facilities or, increasingly, new construction sites in competitive real estate markets.

These in-house capabilities allow gene and cell therapy companies to rapidly scale up production from clinical batches to commercial scale, even when therapies are still in the research and development stage. This also allows for co-location with drug research and development operations, ensuring smooth technology transfer and minimal disruption, particularly during clinical trials.

As a result, there is a pressing need for time-to-market, so the chosen emphasis is on existing buildings, which have become increasingly difficult to come by in developed biotech hubs due to market demand. These hubs provide benefits such as tailored university programs and the involvement of other gene and cell therapy companies (both rivals and potential collaborators), all of which combine to create a target-rich environment for the talent they are all looking for. While all ventures are cost-sensitive, venture-funded businesses are more concerned with cost, and the need to reduce both upfront and ongoing cash outlay.

Competitive LandscapeThe major players operating in the gene therapy R&D market are Astellas Pharma Inc., American Gene Technologies, Applied Genetic, Bayer, Benitec BioPharma, Biogen, Bluebird Bio, Bristol Myers Squibb, Calimmune, Inc. (CSL Behiring), Cellectis, F. Hoffmann-La Roche Ltd., GeneQuine Biotherapeutics, GenSight Biologics, Gilead Lifesciences, Inc., Novartis AG, OCUGEN, INC., Orchard Therapeutics, Oxford Biomedica, Pfizer, Inc., REGENXBIO Inc., Sangamo Therapeutics, Inc., Sanofi, Sarepta Therapeutics, Inc., Spark Therapeutics (Subsidiary of Roche), Takeda Pharmaceuticals, Taysha GTx, Transgene, UniQure NV, Voyager Therapeutics, and ViGeneron. These major players operating in this market have adopted various strategies comprising M&A, investment in R&D, collaborations, partnerships, regional business expansion, and new product launches.

Recent Developments

In June 2022, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending conditional marketing authorization (CMA) to BioMarin Pharmaceutical Inc. for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. The one-time infusion is planned to be marketed under the brand name ROCTAVIAN (valoctocogene roxaparvovec).

In May 2022, ASC Therapeutics joined as a full partner with the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) including the National Institutes of Health (NIH) and the Food and Drug Administration (FDA), managed by the Foundation for the National Institutes of Health (FNIH).

In May 2022, Ultragenyx Pharmaceutical Inc. & Abeona Therapeutics Inc. entered into a license agreement for adeno-associated virus gene therapy ABO-102 (UX111) for the treatment of Sanfilippo syndrome type A.

In April 2022, Pfizer Inc. announced the launch of its U.S. sites in the Phase 3 study evaluating the investigational fordadistrogene movaparvovec & mini-dystrophin gene therapy in patients with Duchenne muscular dystrophy.

In April 2022, the U.S. FDA granted commercial approval to Novartis for its Durham, N.C. site, which is a gene therapy manufacturing facility. This authorization enables the cutting-edge, 170,000 square-foot facility to manufacture, test, and commercially market Zolgensma as well as gene therapy products for ongoing and upcoming clinical trials. The achievement of this milestone allows Novartis Gene Therapies to open a second commercially-licensed manufacturing plant, complementing the Libertyville, Illinois, location that received approval for the production and distribution of Zolgensma (onasemnogene abeparvovec) in 2019.

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Gene therapy R&D market is projected to grow at a CAGR of 30.1% by 2032: Visiongain Reports Ltd - Yahoo Finance

DORVAL, QC, Sept. 20, 2022 /CNW/ - Novartis Pharmaceuticals Canada Inc. and the panCanadian Pharmaceutical Alliance (pCPA) have successfully concluded negotiations for Luxturna (voretigene neparvovec),a one-time gene therapy for the treatment of adult and pediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations.

Novartis Pharmaceuticals Canada Logo (CNW Group/Novartis Pharmaceuticals Canada Inc.)

Inherited retinal dystrophies (IRDs) are a major cause of early onset blindness2. RPE65-mediated IRDs are rare, serious, and progressive conditions that ultimately lead to severe visual impairment and blindness. Prior to the approval of Luxturna, there were no available pharmacological treatment options for this form of inherited blindness2.

"We are thrilled to have positively completed these negotiations for Luxturna,oneof two pioneering targeted gene therapies Novartis has introduced in Canada forpatients and families devastated by rare,debilitating orlife-threatening genetic diseases. For all Canadians who urgently need treatment with this innovative therapy, this is an important step to achieve access," said Andrea Marazzi, Country President, Novartis Canada. "We will continue to work collaboratively with provincial and territorial jurisdictions so that patients whose vision is impaired as a result of a mutation in both copies of the RPE65 gene can have access to Luxturnathrough public drug plans as quickly as possible."

"This type of inherited eye disease affects childrenand youngadults and creates a significant impact on the entire family," said Doug Earle, President & CEO of Fighting Blindness Canada.

"For the majority, it can lead to completeblindness as cells in the retina, the light sensitive tissue of the eye, gradually stop working, work less effectively, or die2. Having access to a treatment that can help restore sight, can be life-altering for a child or young adult and their family. We encourage the provinces to recognize the hope Luxturnarepresents and prioritize access as there are no approved alternative treatments available for these Canadians."

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AboutRPE65mutation-associated inherited retinal dystrophyMutations 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 DiseaseNovartis is at the forefront of cell and gene therapies designed to halt diseases in their tracks or reverse their progress rather than simply manage symptoms. The company is collaborating on the cell and gene therapy frontier to bring this major leap in personalized medicine to patients with a variety of diseases, including genetic disorders and certain deadly cancers. Cell and gene therapies are grounded in careful research that builds on decades of scientific progress. Following key approvals of cell and gene therapies by health authorities, new treatments are being tested in clinical trials around the world.

About Novartis in CanadaNovartis Pharmaceuticals Canada Inc., a leader in the healthcare field, is committed to the discovery, development and marketing of innovative products to improve the well-being of all Canadians. 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 NovartisNovartis 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

Novartis Pharmaceuticals Canada Inc. Luxturna(voretigene neparvovec) Product Monograph. April 20, 2022

Patel U BM, de Lsleuc L, et al. Voretigene Neparvovec: An Emerging Gene Therapy for the Treatment of Inherited Blindness. CADTH Issues in Emerging Health Technologies Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2016. 2018 (169)

Novartis. Data on file. 2018.

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

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

SOURCE Novartis Pharmaceuticals Canada Inc.

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