Gene Therapy - Competitive Landscape, 2022

Gene Therapy - Competitive Landscape, 2022

DelveInsight’s, “Gene therapy - Competitive landscape, 2022,” report provides comprehensive insights about 250+ companies and 300+ drugs in Gene therapy Competitive landscape. It covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space. Geography Covered • Global coverage Gene therapy: Understanding Gene therapy: Overview Gene therapy is defined as the treatment of disease by transfer of genetic material into cells. This review will explore methods available for gene transfer as well as current and potential applications for craniofacial regeneration, with emphasis on future development and design. Though non-viral gene delivery methods are limited by low gene transfer efficiency, they benefit from relative safety, low immunogenicity, ease of manufacture, and lack of DNA insert size limitation. Report Highlights • In August 2022, The US Food and Drug Administration approved Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions. • In March 2022, REGENXBIO announced that the US Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation for RGX-202, which may entitle REGENXBIO to receive a priority review voucher which can be redeemed to obtain priority review for any subsequent marketing application and may be sold or transferred should a new BLA for RGX-202 be approved. • In January 2022, REGENXBIO Inc. announced the clearance of its Investigational New Drug application by the United States Food and Drug Administration (FDA) to evaluate RGX-202, a potential one-time gene therapy for the treatment of Duchenne muscular dystrophy (Duchenne) in a first-in-human clinical trial. RGX-202 is designed to deliver a transgene for a novel microdystrophin that includes the functional elements of the C-Terminal (CT) domain found in naturally occurring dystrophin. RGX-202 uses REGENXBIO's proprietary NAV® AAV8 vector • In November 2021, REGENXBIO Inc. announced that the US FDA granted Orphan Drug Designation for RGX-202, a potential one-time gene therapy for the treatment of Duchenne muscular dystrophy Company Profiles & their Late-stage Drug Profiles 1. Company Overview: Pfizer Pfizer's mission is to deliver medicines that can make a real difference for people living with a rare disease. We pledge to maintain the safety of patients who take part in our rare disease clinical trials and to uphold the highest ethical standards in all of our research initiatives. Our Research and Development (R&D) Scientists are committed to delivering new treatments. Product Description: Fordadistrogene movaparvovec Fordadistrogene movaparvovec is an investigational recombinant adeno-associated virus serotype 9 (AAV9) capsid carrying a shortened version of the human dystrophin gene (mini-dystrophin) under the control of a human muscle-specific promotor. The AAV9 capsid was chosen as the delivery mechanism because of its potential to target muscle tissue Data from the Phase Ib study of PF-06939926 in ambulatory for boys with DMD showed the gene therapy displayed durable and statistically significant improvements in multiple efficacy endpoints measured 12 months following infusion. 2. Company Overview: Lexeo Therapeutics LEXEO Therapeutics is a New York City-based, fully integrated biotechnology company currently headquartered at the Alexandria Center for Life Science that aims to apply the transformational science of gene therapy to address some of the world’s most devastating genetic and acquired diseases. LEXEO Therapeutics’ pipeline consists of adeno-associated virus (AAV) - mediated therapies primarily developed at Weill Cornell Medicine’s Department of Genetic Medicine. Beyond LEXEO Therapeutics’ lead programs – which are focused on both rare and non-rare monogenic (single gene mutation) diseases – the company’s pre-clinical pipeline spans monogenic diseases, as well as hereditary and acquired diseases across a spectrum of patient population sizes and a range of unmet medical needs. Importantly, LEXEO Therapeutics will focus on advancing clinical programs through to commercialization, with the goal of maintaining an ongoing research collaboration with Weill Cornell Medicine’s Department of Genetic Medicine to help advance the company’s pre-clinical pipeline. Product Description: LX2006 LX2006 is an AAV-based gene therapy candidate delivered intravenously for the treatment of FA cardiomyopathy, the most common cause of mortality in patients with Friedreich’s ataxia. LX2006 is designed to target the cardiac manifestations of FA by delivering a functional frataxin gene to promote the expression of the frataxin protein and restore mitochondrial function in myocardial cells. In preclinical studies, LX2006 reversed the cardiac abnormalities in FA disease models and showed improvement in cardiac function and survival while demonstrating a favorable safety profile. The FDA has granted Rare Pediatric Disease designation and Orphan Drug designation to LX2006 for the treatment of Friedreich’s ataxia. The Phase I/II study is a 52-week, dose-ascending, open-label trial of LX2006 in patients who have FA cardiomyopathy. LX2006 will be administered as a one-time intravenous infusion to patients in two ascending-dose cohorts of five patients each. 3. Company Overview: Helixmith Helixmith is a gene therapy company headquartered in Seoul, Korea, developing new and innovative biopharmaceuticals for previously untreated diseases, and is listed on KOSDAQ. The company has an extensive gene therapy pipeline, including a CAR-T program targeting several different types of solid tumors and an AAV vector program targeting neuromuscular diseases. Product Description: Engensis (VM202) Engensis (VM202) is an innovative gene therapy drug that provides fundamental treatment through tissue regeneration. Helixmith’s non-viral plasmid DNA product, Engensis, is designed to express recombinant HGF protein in nerve and Schwann cells to promote nerve system regeneration and induce the formation of microvascular blood vessels. HGF has a short half-life (5 minutes or less) and is quickly removed from the body by the liver, creating an obstacle to effective treatment with previous injectable recombinant HGF protein products. 4. Company Overview: bluebird bio bluebird bio is pursuing curative gene therapies to give patients and their families more bluebird days. With a dedicated focus on severe genetic diseases, bluebird has industry-leading clinical and research programs for sickle cell disease, beta-thalassemia and cerebral adrenoleukodystrophy and is advancing research to apply new technologies to these and other diseases. We custom design each of our therapies to address the underlying cause of disease and have developed in-depth and effective analytical methods to understand the safety of our lentiviral vector technologies and drive the field of gene therapy forward. Product Description: Zynteglo ZYNTEGLO is a first-in-class, one-time ex-vivo LVV gene therapy approved for the treatment of beta-thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions. ZYNTEGLO works by adding functional copies of a modified form of the beta-globin gene (βA-T87Q-globin gene) into a patient’s own hematopoietic (blood) stem cells to enable the production of a modified functional adult hemoglobin (HbAT87Q). Once a patient has the βA-T87Q-globin gene, they have the potential to increase ZYNTEGLO-derived adult hemoglobin (HbAT87Q) and total hemoglobin to normal or near normal levels that can eliminate the need for regular RBC transfusions Further product details are provided in the report…….. Future of Competitive Landscape of Gene Therapy is estimated to be very strong. Key emerging drugs include bluebird bio Zynteglo and many other. Gene Therapy Analytical Perspective by DelveInsight • In-depth Gene Therapy analysis: Assessment of Products The Report provides in-depth commercial assessment of drugs that have been included, which comprises collaboration, agreement, licensing and acquisition – deals values trends. The sub-segmentation is described in the report which provide company-company collaboration (licensing/partnering), company academic collaboration and acquisition analysis in tabulated form. • Gene Therapy clinical assessment of products The report comprises of comparative clinical assessment of products by development stage, product type, and route of administration, molecule type. Gene Therapy Report Assessment • Company Analysis • Therapeutic Assessment • Pipeline Assessment • Inactive drugs assessment • Unmet Needs Key Questions Current Treatment Scenario and Emerging Therapies: • How many companies are developing Gene Therapy drugs? • How many Gene Therapy drugs are developed by each company? • How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Gene Therapy? • What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Gene Therapy therapeutics? • What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies? • What are the clinical studies going on for Gene Therapy and their status? • What are the key designations that have been granted to the emerging and approved drugs?   Key Players • Alnylam Pharmaceuticals • Novartis • Johnson & Johnson • Fibrocell Technologies • Pfizer • HELIXMITH Co., Ltd. • Sarepta Therapeutics • REGENXBIO • Solid Biosciences Inc. • Lexeo Therapeutics • Spark Therapeutics • Xalud Therapeutics • uniQure • Ultragenyx Pharmaceutical • Nanoscope Therapeutics • Homology Medicines • Freeline therapeutics • Amicus Therapeutics • REGENXBIO • Gyroscope Therapeutics • CSL • Sangamo Therapeutics • Spark therapeuics • LYSOGENE • Asklepios BioPharmaceutical • Alcyone Therapeutics • Taysha Gene Therapies • Gensight Biologic • Vivet Therapeutics • BridgeBio Key Products • Zolgensma • Carvykti • Laviv • Provenge • Fordadistrogene movaparvovec • Engensis • SRP-9001 • RGX-202 • SGT-001 • LX2006 • SPK 3006 • XT-150 • AMT-161 • UX810 • MCO 010 • HMI-102 • FLT190 • EtranaDez • FLT180a • GT005 • RGX-121 • AT GAA • Giroctocogene fitelparvovec • SPK-8011 • LYS-SAF302 • ACTUS-101 • ACTX-401 • TSHA 120 • GS030 • VTX-801 • BBP-631

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