DelveInsight’s, “Adeno-Associated Virus Vectors in Gene Therapy Pipeline Insight 2024” report provides comprehensive insights about 70+ companies and 235+ pipeline drugs in the Adeno-Associated Virus Vectors in Gene Therapy pipeline landscape. It covers the Adeno-Associated Virus Vectors in Gene Therapy pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Adeno-Associated Virus 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 Adeno-Associated Virus Vectors in Gene Therapy Pipeline Report
- DelveInsight’s Adeno-Associated Virus Vectors in Gene Therapy pipeline report depicts a robust space with 70+ active players working to develop 235+ pipeline therapies for Adeno-Associated Virus Vectors in Gene Therapy treatment.
- The leading companies working in the Adeno-Associated Virus Vectors in Gene Therapy Market include BioMarin Pharmaceutical, Gensight Biologics, PTC therapeutics, Ultragenyx Pharmaceutical, MeiraGTx, Pfizer, Biogen, uniQure, 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, uniQure, Taysha Gene Therapies, Asklepios BioPharmaceutical, Sarepta Therapeutics, Abeona Therapeutics, Forge Biologics, Sangamo therapeutics, and others.
- Promising Adeno-Associated Virus Vectors in Gene Therapy Pipeline Therapies in the various stages of development include Valoctocogene Roxaparvovec, AAV – CNGB3, SB-525 (PF-07055480), AAV2/5-RPGR, BMN 307, GC301, rAAV-Olig001-ASPA, and others.
- November 2023 Shanghai Belief-Delivery BioMed Co., Ltd announced a study of Phase 1 & 2 clinical trials for Single dose intravenous injection of BBM-H803. This is a multi-center, single-arm, and open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of BBM-H803 injection in heavy Hemophilia A subjects. BBM-H803 is an adeno-associated viral (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor VIII transgene and raises circulating levels of endogenous FVIII.
- October 2023: BioMarin Pharmaceutical announced a study of phase 3 clinical trials for Valoctocogene Roxaparvovec. This Phase III clinical study will assess the efficacy of BMN 270 defined as FVIII activity, during weeks 49-52 following intravenous infusion of BMN 270 and assess the impact of BMN 270 on usage of exogenous FVIII replacement therapy and the number of bleeding episodes from week 5 to week 52.
- October 2023: Adverum Biotechnologies Inc. announced a study of Phase 1 & 2 clincial trials for ADVM-043. The ADVANCE study is being conducted by Adverum Biotechnologies, Inc. as an open-label, multicenter, dose-escalation study in order to assess the safety and protein expression of ADVM-043 following a single intravenous or intrapleural administration.
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In the Adeno-Associated Virus Vectors in Gene Therapy Pipeline Report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, AAV vectors in gene therapy collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Adeno-Associated Virus Vectors in Gene Therapy Overview
Adeno-Associated Virus (AAV) vectors have emerged as a cornerstone in the field of gene therapy, revolutionizing the potential for treating a wide range of genetic and acquired diseases. These vectors, derived from naturally occurring non-pathogenic viruses, have gained prominence due to their safety profile, low immunogenicity, and ability to deliver therapeutic genes into target cells with remarkable precision. In gene therapy, AAV vectors act as vehicles for transporting corrected or therapeutic genes to replace or supplement malfunctioning ones.
Find out more about Adeno-Associated Virus Vectors in Gene Therapy Therapeutics Assessment @ Adeno-Associated Virus Vectors in Gene Therapy Preclinical and Discovery Stage Products
Adeno-Associated Virus 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
Adeno-Associated Virus Vectors in Gene Therapy Pipeline Therapeutics Assessment
There are approx. 70+ key companies developing Adeno-Associated Virus Vectors in Gene Therapy therapies. The Adeno-Associated Virus 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 Products have been categorized under various Molecule types such as
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
- Product Type
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Scope of the Adeno-Associated Virus Vectors in Gene Therapy Pipeline Report
- Coverage- Global
- Adeno-Associated Virus Vectors in Gene Therapy Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
- Adeno-Associated Virus Vectors in Gene Therapy Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
- Adeno-Associated Virus Vectors in Gene Therapy 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, and others.
- Adeno-Associated Virus Vectors in Gene Therapy Pipeline Therapies- Valoctocogene Roxaparvovec, AAV – CNGB3, SB-525 (PF-07055480), AAV2/5-RPGR, BMN 307, GC301, rAAV-Olig001-ASPA, 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 Adeno-Associated Virus Vectors in Gene Therapy pipeline therapeutics, reach out to Adeno-Associated Virus Vectors in Gene Therapy Unmet Needs and Analyst Views
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