DelveInsight’s ‘Gene Therapy Competitive Landscape 2023‘ report provides comprehensive global coverage of available, marketed, and pipeline Gene therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the Gene Therapy pipeline domain.
For emerging Gene Therapy, the Gene Therapy pipeline analysis report provides a 360° view of the therapeutics landscape by development point, product type, route of administration, molecule type, and MOA. The pipeline research covers business opportunities, challenges, future partnerships, strong competitors, and growth strategies.
Key Takeaways from the Gene Therapy Pipeline Report
- DelveInsight’s Gene Therapy Pipeline analysis depicts a robust space with 260+ active players working to develop 300+ pipeline gene therapies.
- Leading gene therapy companies such as Novartis, Johnson & Johnson, Fibrocell Technologies, Pfizer, HELIXMITH Co., Ltd., Sarepta Therapeutics, 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, LYSOGENE, Asklepios BioPharmaceutical, Alcyone Therapeutics, Taysha Gene Therapies, Gensight Biologic, Vivet Therapeutics, BridgeBio, BioMarin Pharmaceutical, Neurophth Therapeutics, Abeona Therapeutics, Coave Therapeutics, Actus Therapeutics, Forge Biologics, Passage Bio, Aspa Therapeutics, Adrenas Therapeutics, ESTEVE, 4D Molecular Therapeutics, Bayer, LogicBio Therapeutics, Eli Lilly and Company, Adverum Biotechnologies, Libella Gene Therapeutics, Rocket Pharmaceuticals, Decibel therapeutics, DiNAQOR, Affinia Therapeutics, Lacerta Therapeutics, Atamyo Therapeutics, Apic Bio, Orchard Therapeutics, Nervosave Therapeutics, Maze Therapeutics, SOLA Biosciences, Benitec Biopharma, Hanugen Therapeutics, Exegenesis Bio, CANbridge Life Sciences Ltd, StrideBio, ENZERNA BIOSCIENCES INC., AAVogen, Capsigen, Code Biotherapeutics, Amarna Therapeutics, Poseida Therapeutics, Inc., bluebird bio, Genethon, Voyager Therapeutics, Jaguar Gene Therapy, LLC, Oxford Biomedica, Ocugen, Genprex, Beam Therapeutics, Neurogene Inc., Treeway, Oyster Point Pharma, Elicera Therapeutics AB, Generation Bio Co., Atsena Therapeutics, Precision Biosciences, Precigen, SELECTA BIOSCIENCES, Encoded Therapeutics, Graphite Bio, Nuevocor, M6P Therapeutics, SwanBio Therapeutics, AviadoBio, Myrtelle, Kriya Therapeutics, Inc., Senti Biosciences, Castle Creek Biosciences, Inc., Treadwell Therapeutics, Opus Genetics, Renovacor, and others are evaluating novel gene therapies candidate to improve the treatment landscape.
- Key gene therapies in the pipeline in various stages of development include 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, ADVM-022, AAV- hTERT, RP-A501, and others.
- In March 2023, Lineage Cell Therapeutics, Inc., a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, announced that findings from a Phase 1/2a clinical study of RG6501 (OpRegen) will be presented at the 2023 Retinal Cell and Gene Therapy Innovation Summit, hosted by the Foundation Fighting Blindness and the Oregon Health & Science University Casey Eye Institute.
- In March 2023, Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, announced that the U.S. Food and Drug Administration’s Office of Therapeutics (OTP) determined at its late-cycle meeting for the SRP-9001 (delandistrogene moxeparvovec) biologics license application (BLA) that an advisory committee meeting will be held for SRP-9001 prior to the regulatory action date of May 29, 2023.
- In December 2022, Hemgenix (etranacogene dezaparvovec), the first gene therapy for Hemophilia B, was approved by the US Food and Drug Administration (FDA). A single dose costs $3.5 million and is the most expensive drug ever approved in the United States.
- In October 2022, Astellas and Taysha Gene Therapies announced the strategic investment to support the development of Taysha’s AAV-based gene therapy programs.
- In October 2022, French companies formed a strategic alliance to cover cell and gene therapy (C>) GMP manufacturing.
- In October 2022, AVROBIO announced the patient dosing completion in the first gene therapy clinical trial for cystinosis treatment.
- In October 2022, Xcell Biosciences Inc. (Xcellbio) and aCGT Vector announced a collaboration today to improve manufacturing and analytic procedures used to develop personalized cell and gene therapies for cancer patients.
- In August 2022, the FDA approved Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy for treating adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.
Request a sample and discover the recent breakthroughs happening in the Gene Therapy pipeline landscape @ Gene Therapy Competitive Landscape Report
Gene Therapy Overview
Gene therapy is an experimental technique in which functional genes are introduced into a patient’s body to counteract or replace defective ones, thereby curing disease without the use of pharmaceuticals, radiotherapy, or surgery. Gene therapy can treat genetic defects that are difficult to treat with drugs or antibodies. There are two types of gene therapy based on the type of cells treated: somatic gene therapy and germline gene therapy.
Gene therapy is a promising treatment option for many diseases, and the general gene therapy strategy is straightforward. Gene therapy is a cure for many rare diseases and a ray of hope for patients with complex genetic disorders. Furthermore, it is used to treat cancer and viral infections. Gene therapy for cancer is on the verge of becoming a standard cancer treatment, potentially elevating this method to first-line therapy for neoplastic diseases.
Find out more about gene therapy treatment @ Gene Therapy Market
Company Profiles & their Late-stage Drug Profiles
Company Overview: Pfizer
Pfizer’s mission is to provide medicines that can make a significant difference in the lives of people living with rare diseases. Pfizer promises to keep patients safe in our rare disease clinical trials and to uphold the highest ethical standards in all of our research initiatives. Pfizer’s research and development (R&D) scientists are dedicated to developing new treatments.
Product Description: Fordadistrogene movaparvovec
Fordadistrogene movaparvovec is a recombinant adeno-associated virus serotype 9 (AAV9) capsid that contains a shortened version of the human dystrophin gene (mini-dystrophin) controlled by a human muscle-specific promotor. Because of its ability to target muscle tissue, the AAV9 capsid was chosen as the delivery mechanism. The gene therapy demonstrated durable and statistically significant improvements in multiple efficacy endpoints measured 12 months after infusion in the Phase Ib study of PF-06939926 in ambulatory for boys with DMD.
Company Overview: Lexeo Therapeutics
LEXEO Therapeutics is a New York City-based, a fully integrated biotechnology company based at the Alexandria Center for Life Science that aims to use the transformative science of gene therapy to treat some of the world’s most devastating genetic and acquired diseases. The pipeline of LEXEO Therapeutics consists primarily of adeno-associated virus (AAV)-mediated therapies developed at Weill Cornell Medicine’s Department of Genetic Medicine.
Product Description: LX2006
LX2006 is an AAV-based gene therapy candidate that is given intravenously to patients with Friedreich’s ataxia cardiomyopathy, which is the leading cause of death in Friedreich’s ataxia patients. LX2006 targets Friedreich’s ataxia’s cardiac manifestations by delivering a functional frataxin gene, which promotes frataxin protein expression and restores mitochondrial function in myocardial cells. In preclinical studies, LX2006 reversed cardiac abnormalities in Friedreich’s ataxia disease models, improving cardiac function and survival while demonstrating a favorable safety profile. The FDA has granted LX2006 Rare Pediatric Disease and Orphan Drug designation for the treatment of Friedreich’s ataxia.
Gene Therapy Pipeline Therapies and Key Companies
- Etranacogene dezaparvovec: CSL Behring/uniQure
- Engensis: Helixmith
- Fordadistrogene movaparvovec: Pfizer
- RGX-121: REGENXBIO
- LYS-SAF302: LYSOGENE
- GT005: Gyroscope Therapeutics
- SPK 3006: Spark Therapeutics
- FLT190: Freeline Therapeutics
- LX2006: Lexeo Therapeutics
Learn more about the emerging gene therapies @ Gene Therapy Clinical Trials
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
- Gene Therapy Company Analysis
- Gene Therapy Therapeutic Assessment
- Gene Therapy Pipeline Assessment
- Inactive Gene Therapy drugs assessment
- Gene Therapy Unmet Needs
Scope of the Gene Therapy Pipeline Report
- Coverage: Global
- Key Gene Therapy Companies: Novartis, Johnson & Johnson, Fibrocell Technologies, Pfizer, HELIXMITH Co., Ltd., Sarepta Therapeutics, 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, LYSOGENE, Asklepios BioPharmaceutical, Alcyone Therapeutics, Taysha Gene Therapies, Gensight Biologic, Vivet Therapeutics, BridgeBio, BioMarin Pharmaceutical, Neurophth Therapeutics, Abeona Therapeutics, Coave Therapeutics, Actus Therapeutics, Forge Biologics, Passage Bio, Aspa Therapeutics, Adrenas Therapeutics, ESTEVE, 4D Molecular Therapeutics, Bayer, LogicBio Therapeutics, Eli Lilly and Company, Adverum Biotechnologies, Libella Gene Therapeutics, Rocket Pharmaceuticals, Decibel therapeutics, DiNAQOR, Affinia Therapeutics, Lacerta Therapeutics, Atamyo Therapeutics, Apic Bio, Orchard Therapeutics, Nervosave Therapeutics, Maze Therapeutics, SOLA Biosciences, Benitec Biopharma, Hanugen Therapeutics, Exegenesis Bio, CANbridge Life Sciences Ltd, StrideBio, ENZERNA BIOSCIENCES INC., AAVogen, Capsigen, Code Biotherapeutics, Amarna Therapeutics, Poseida Therapeutics, Inc., bluebird bio, Genethon, Voyager Therapeutics, Jaguar Gene Therapy, LLC, Oxford Biomedica, Ocugen, Genprex, Beam Therapeutics, Neurogene Inc., Treeway, Oyster Point Pharma, Elicera Therapeutics AB, Generation Bio Co., Atsena Therapeutics, Precision Biosciences, Precigen, SELECTA BIOSCIENCES, Encoded Therapeutics, Graphite Bio, Nuevocor, M6P Therapeutics, SwanBio Therapeutics, AviadoBio, Myrtelle, Kriya Therapeutics, Inc., Senti Biosciences, Castle Creek Biosciences, Inc., Treadwell Therapeutics, Opus Genetics, Renovacor, and others.
- Key Gene Therapies in Pipeline: 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, and others.
Dive deep into rich insights for approved gene therapies; visit @ FDA-approved Gene Therapies
Table of Contents
| 1. | Introduction |
| 2. | Executive Summary |
| 3. | Gene Therapy Pipeline: Overview |
| 4. | Analytical Perspective In-depth Commercial Assessment |
| 5. | Gene Therapy Pipeline Therapeutics |
| 6. | Gene Therapy Pipeline: Late Stage Products (Phase III) |
| 7. | Gene Therapy Pipeline: Late Stage Products (Phase III) |
| 8. | Gene Therapy Pipeline: Mid Stage Products (Phase II) |
| 9. | Gene Therapy Pipeline: Early Stage Products (Phase I) |
| 10. | Therapeutic Assessment |
| 11. | Inactive Products |
| 12. | Company-University Collaborations (Licensing/Partnering) Analysis |
| 13. | Key Companies |
| 14. | Key Products |
| 15. | Unmet Needs |
| 16. | Market Drivers and Barriers |
| 17. | Future Perspectives and Conclusion |
| 18. | Analyst Views |
| 19. | Appendix |
For further information on gene therapy uses, reach out @ Viral Vectors for Gene Therapy
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