DelveInsight’s, “AAV Vectors in Gene Therapy Pipeline Insight 2023” report provides comprehensive insights about 70+ companies and 235+ pipeline drugs in AAV vectors in the gene therapy pipeline landscape. It covers the AAV Vectors in Gene Therapy pipeline drug profiles, including clinical and nonclinical stage products. It also covers the AAV Vectors in Gene Therapy pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Key Takeaways from the AAV Vectors in Gene Therapy Pipeline Report
- DelveInsight’s AAV Vectors in Gene Therapy pipeline report depicts a robust space with 70+ active players working to develop 235+ pipeline therapies for AAV Vectors in Gene Therapy treatment.
- The leading companies working in the AAV Vectors in Gene Therapy market include BioMarin Pharmaceutical, Gensight Biologics, PTC therapeutics, Ultragenyx Pharmaceutical, MeiraGTx, Pfizer, Biogen, uniQure, Pfizer, Ultragenyx Pharmaceutical, REGENXBIO, Biogen, Spark therapeuics (Roche), Sarepta therapeutics, Neurophth Therapeutics, LYSOGENE, Gyroscope Therapeutics, Nanoscope Therapeutics, Homology medicines, Ultragenyx Pharmaceutical, Passage Bio, Freeline therapeutics, Astellas Pharma, Aspa therapeutics, Adrenas Therapeutics, ESTEVE, Sio Gene Therapies, Amicus therapeutics, 4D Molecular therapeutics, Taysha Gene Therapies, Atsena Therapeutics, BioMarin Pharmaceutical, Abeona Therapeutics, REGENXBIO, Taysha Gene Therapies, Asklepios BioPharmaceutical, Sarepta Therapeutics, Abeona Therapeutics, Forge Biologics, Sangamo therapeutics, Bayer, LogicBio therapeutics, Solid Biosciences, Applied Genetic Technologies, Spark therapeuics (Roche), Eli lilly and company, Lexeo Therapeutics, Spark therapeutics, Gensight Biologics, Alcyone Lifesciences, MeiraGTx, Audentes therapeutics, Vivet Therapeutics, Takeda, Astellas Gene Therapies, Homology Medicines, Adverum Biotechnologies, Libella Gene Therapeutics, Rocket Pharmaceuticals, Brain Neurotherapy Bio, Inc., Ocugen, Asklepios BioPharmaceutical, Neurogene, Jaguar Gene Therapy, LLC, Axovia Therapeutics, and others.
- Promising AAV Vectors in Gene Therapy Pipeline Therapies in the various stages of development include SB-525 (PF-07055480), Valoctocogene Roxaparvovec, AAV – CNGB3, AAV2/5-RPGR, BMN 307, GC301, AAV2hAQP1, and others.
- November 2023: The National Human Genome Research Institute announced a study of Phase 1 & 2 clinical trials for AAV9-GLB1 and Sirolimus. A Phase 1/2 Study of Intravenous Gene Transfer with an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis.
- November 2023: BioMarin Pharmaceuticals announced a study of Phase 3 clinical trials for Valoctocogene Roxaparvovec. This Phase III clinical study will assess the impact of BMN 270 (compared to FVIII prophylaxis) on the number of bleeding episodes irrespective of exogenous FVIII replacement treatment in the efficacy evaluation period (EEP) (from Week 5 post-BMN 270 infusion (Study Day 33) or the end of FVIII prophylaxis plus the washout period (3 days for products of standard half-life or plasma-derived and 5 days for products of extended half-life), whichever is later, to last visit by the data cut-off for the 2-year analysis, hereafter referred to as “Post FVIII Prophylaxis to Last Visit”).
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The AAV Vectors in Gene Therapy pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage AAV Vectors in Gene Therapy drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the AAV Vectors in Gene Therapy clinical trial landscape.
AAV Vectors in Gene Therapy Overview
Out of the several viral vectors that have been used to date for delivering the genes of interest, the Adeno-associated viral (AAV) vector appears to be the safest and most effective vehicle and can maintain long-term gene and protein expression following a single injection of the vector.
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AAV Vectors in Gene Therapy Emerging Drugs Profile
- GS010: Gensight Biologics
- Valoctocogene roxaparvovec: BioMarin Pharmaceutical
- DTX401: Ultragenyx Pharmaceutical
- AAV5-RPGR: MeiraGTx
- Timrepigene emparvovec: Biogen
- AMT 061: UniQure
- RGX-314: REGENXBIO
- SPK-8011: Spark Therapeutics
- NFS-01: Neurophth
- GT 005: Gyroscope Therapeutics
AAV Vectors in Gene Therapy Pipeline Therapeutics Assessment
There are approx. 70+ key companies developing therapies for AAV vectors in gene therapy. The AAV Vectors in Gene Therapy companies that have their AAV vectors in gene therapy drug candidates in the most advanced stage, i.e. Preregistration include BioMarin Pharmaceutical.
DelveInsight’s Adeno-Associated Vector Pipeline Report covers around 235+ products under different phases of clinical development like
- Late stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage product (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
Adeno-Associated Vector pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
- Intracerebral
- Intracerebroventricular
- Intracisternal
- Intramuscular
- Intraocular
- Intrapancreatic
- Intrastriatal
- Intrathecal
- Intravenous
- Intravitreous
- Introvitinreous
- Oral
- Parenteral
- Retinal
- Intra-arterial
- Molecule Type
Adeno-Associated Vector Pipeline Products have been categorized under various Molecule types such as
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
- Product Type
Learn more about the emerging AAV Vectors in Gene Therapy Pipeline Therapies @ AAV Vectors in Gene Therapy Clinical Trials Assessment
Scope of the AAV Vectors in Gene Therapy Pipeline Report
- Coverage- Global
- AAV Vectors in Gene Therapy Pipeline Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
- AAV Vectors in Gene Therapy Pipeline Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
- Companies- BioMarin Pharmaceutical, Gensight Biologics, PTC therapeutics, Ultragenyx Pharmaceutical, MeiraGTx, Pfizer, Biogen, uniQure, Pfizer, Ultragenyx Pharmaceutical, REGENXBIO, Biogen, Spark therapeuics (Roche), Sarepta therapeutics, Neurophth Therapeutics, LYSOGENE, Gyroscope Therapeutics, Nanoscope Therapeutics, Homology medicines, Ultragenyx Pharmaceutical, Passage Bio, Freeline therapeutics, Astellas Pharma, Aspa therapeutics, Adrenas Therapeutics, ESTEVE, Sio Gene Therapies, Amicus therapeutics, 4D Molecular therapeutics, Taysha Gene Therapies, Atsena Therapeutics, BioMarin Pharmaceutical, Abeona Therapeutics, REGENXBIO, Taysha Gene Therapies, Asklepios BioPharmaceutical, Sarepta Therapeutics, Abeona Therapeutics, Forge Biologics, Sangamo therapeutics, Bayer, LogicBio therapeutics, Solid Biosciences, Applied Genetic Technologies, Spark therapeuics (Roche), Eli lilly and company, Lexeo Therapeutics, Spark therapeutics, Gensight Biologics, Alcyone Lifesciences, MeiraGTx, Audentes therapeutics, Vivet Therapeutics, Takeda, Astellas Gene Therapies, Homology Medicines, Adverum Biotechnologies, Libella Gene Therapeutics, Rocket Pharmaceuticals, Brain Neurotherapy Bio, Inc., Ocugen, Asklepios BioPharmaceutical, Neurogene, Jaguar Gene Therapy, LLC, Axovia Therapeutics, and others.
- Pipeline Therapies- SB-525 (PF-07055480), Valoctocogene Roxaparvovec, AAV – CNGB3, AAV2/5-RPGR, BMN 307, GC301, AAV2hAQP1, and others.
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Table of Content
- Introduction
- Executive Summary
- AAV vectors in gene therapy: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- Late Stage Products (Preregistration)
- Valoctocogene roxaparvovec: BioMarin Pharmaceutical
- Drug profiles in the detailed report…..
- Late Stage Products (Phase III)
- DTX401: Ultragenyx Pharmaceutical
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- GT 005: Gyroscope Therapeutics
- Drug profiles in the detailed report…..
- Early stage products (Phase I/II)
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- HMI-203: Homology Medicine
- Drug profiles in the detailed report…..
- Preclinical stage products
- AXV101: Axovia Therapeutics
- Drug profiles in the detailed report…..
- Discovery stage products
- DINA-002: DiNAQOR
- Drug profiles in the detailed report…..
- Inactive Products
- AAV vectors in gene therapy Key Companies
- AAV vectors in gene therapy Key Products
- AAV vectors in gene therapy- Unmet Needs
- AAV vectors in gene therapy- Market Drivers and Barriers
- AAV vectors in gene therapy- Future Perspectives and Conclusion
- AAV vectors in gene therapy Analyst Views
- AAV vectors in gene therapy Key Companies
- Appendix
For further information on the AAV Vectors in Gene Therapy Pipeline therapeutics, reach out to AAV Vectors in Gene Therapy Unmet Needs and Analyst Views
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