DelveInsight reports that over 180 key companies are actively developing more than 200 therapies utilizing AAV vectors in gene therapy.
AAV Vectors in Gene Therapy Overview:
Adeno-associated virus (AAV) vectors are highly effective gene therapy delivery tools, valued for their precision in delivering therapeutic genes to target cells while eliciting minimal immune response. Naturally occurring in humans and non-pathogenic, AAVs are considered safe since they are not linked to any known diseases. Their ability to enable long-term or sustained gene expression makes them ideal for in vivo applications, as they can integrate into cells and maintain function over extended periods.
Various AAV serotypes have distinct tissue-targeting abilities, allowing therapies to be tailored for specific diseases. For example, AAV9 can cross the blood–brain barrier, making it suitable for central nervous system (CNS) disorders, while AAV2 is particularly effective for ocular treatments. This versatility enables AAV vectors to treat a range of genetic disorders, including hemophilia, spinal muscular atrophy (SMA), and inherited retinal diseases. Notably, AAV-based therapies such as Luxturna and Zolgensma have already gained regulatory approval for rare genetic conditions, representing major milestones in gene therapy advancement.
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"AAV Vectors in Gene Therapy Pipeline Insight 2025" report by DelveInsight provides a comprehensive analysis of the ongoing clinical development activities and growth prospects across the AAV Vectors in Gene Therapy Therapeutics Market.
Key Takeaways from the AAV Vectors in Gene Therapy Pipeline Report
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DelveInsight’s AAV Vectors in Gene Therapy pipeline report outlines a dynamic field with over 25 active companies developing more than 30 therapies.
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Leading players include Belief BioMed, Gensight Biologics, Johnson & Johnson Innovative Medicine, MeiraGTx, Passage Bio, InnoVec Biotherapeutics, Ultragenyx Pharmaceutical, Biogen, REGENXBIO, Beacon Therapeutics, Genethon, 4D Molecular Therapeutics, Adverum Biotechnologies, Rocket Pharmaceuticals, Aspa Therapeutics, Sarepta Therapeutics, Ray Therapeutics, Genascence Corporation, Exegenesis Bio, DiNAQOR, Tenaya Therapeutics, Jaguar Gene Therapy, Neurophth, and others, all aiming to advance the treatment landscape.
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Notable candidates in development include BBM-H901, GS010, AAV-CNGA3, PBGM01, and IVB102.
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In June 2023, the FDA approved Roctavian by BioMarin Pharmaceutical for adults with severe hemophilia A. This AAV-based therapy delivers a functional gene for clotting Factor VIII, significantly reducing annual bleeding rates in trials. That same month, the FDA granted accelerated approval to Elevidys by Sarepta Therapeutics for children aged 4–5 with Duchenne muscular dystrophy (DMD), designed to restore muscle function by introducing a shortened dystrophin gene.
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However, the field has also seen setbacks. In December 2025, Pfizer ended its partnership with Sangamo Therapeutics on the hemophilia A therapy giroctocogene fitelparvovec, despite positive Phase 3 results, citing clinical feedback and limited market demand. In March 2025, safety concerns arose when a patient treated with Elevidys developed acute liver failure and died, with concurrent cytomegalovirus infection potentially contributing. This incident heightened scrutiny of AAV-based therapies’ liver-related risks and affected Sarepta’s market position.
AAV Vectors in Gene Therapy Pipeline Analysis
The report provides insights into:
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The report provides detailed insights into the key companies that are developing therapies in the AAV Vectors in Gene Therapy Market.
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The report also evaluates different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for AAV Vectors in Gene Therapy treatment.
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It analyzes the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
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It navigates the emerging drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
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Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement, and financing details for future advancement of the AAV Vectors in Gene Therapy market.
Download our free sample page report on AAV Vectors in Gene Therapy pipeline insights
AAV Vectors in Gene Therapy Emerging Drugs
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BBM-H901: Belief BioMed
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GS010: Gensight Biologics
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AAV-CNGA3: Johnson & Johnson Innovative Medicine/ MeiraGTx
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PBGM01: Passage Bio
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IVB102: InnoVec Biotherapeutics
AAV Vectors in Gene Therapy Companies
Over 180 major companies are working on gene therapy treatments using Adeno-Associated Virus (AAV) vectors, with Belief BioMed advancing drug candidates to the most progressed stage—the registration phase.
DelveInsight’s report covers around 30+ products under different phases of clinical development like
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Late stage products (Phase III)
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Mid-stage products (Phase II)
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Early-stage product (Phase I) along with the details of
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Pre-clinical and Discovery stage candidates
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Discontinued & Inactive candidates
AAV Vectors in Gene Therapy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
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Intravenous
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Subcutaneous
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Oral
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Intramuscular
AAV Vectors in Gene Therapy Products have been categorized under various Molecule types such as
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Monoclonal antibody
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Small molecule
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Peptide
Download Sample Pages to Get an in-depth Assessment of the Emerging AAV Vectors in Gene Therapy Therapies and Key Companies: AAV Vectors in Gene Therapy Clinical Trials and advancements
AAV Vectors in Gene Therapy Pipeline Therapeutic Assessment
• AAV Vectors in Gene Therapy Assessment by Product Type
• AAV Vectors in Gene Therapy By Stage
• AAV Vectors in Gene Therapy Assessment by Route of Administration
• AAV Vectors in Gene Therapy Assessment by Molecule Type
Download AAV Vectors in Gene Therapy Sample report to know in detail about the AAV Vectors in Gene Therapy treatment market @ AAV Vectors in Gene Therapy Therapeutic Assessment
Table of Content
1. Report Introduction
2. Executive Summary
3. AAV Vectors in Gene Therapy Current Treatment Patterns
4. AAV Vectors in Gene Therapy - DelveInsight's Analytical Perspective
5. Therapeutic Assessment
6. AAV Vectors in Gene Therapy Late-Stage Products (Phase-III)
7. AAV Vectors in Gene Therapy Mid-Stage Products (Phase-II)
8. Early Stage Products (Phase-I)
9. Pre-clinical Products and Discovery Stage Products
10. Inactive Products
11. Dormant Products
12. AAV Vectors in Gene Therapy Discontinued Products
13. AAV Vectors in Gene Therapy Product Profiles
14. AAV Vectors in Gene Therapy Key Companies
15. AAV Vectors in Gene Therapy Key Products
16. Dormant and Discontinued Products
17. AAV Vectors in Gene Therapy Unmet Needs
18. AAV Vectors in Gene Therapy Future Perspectives
19. AAV Vectors in Gene Therapy Analyst Review
20. Appendix
21. Report Methodology
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About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.
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