AAV Vectors in Gene Therapy Clinical and Non-Clinical Studies, Key Companies, Therapeutic Assessment, Emerging Therapies, Treatment Algorithm, and Pipeline Analysis | Belief BioMed, Gensight Biologics, Johnson & Johnson Innovative Medicine, MeiraGTx, Pass

AAV Vectors in Gene Therapy Pipeline constitutes 180+ key companies continuously working towards developing 200+ AAV Vectors in Gene Therapy treatment therapies, analyzes DelveInsight.

AAV Vectors in Gene Therapy Overview:

Adeno-Associated Virus (AAV) vectors are among the most effective gene therapy delivery systems, known for their ability to precisely introduce therapeutic genes into target cells while triggering minimal immune response. Naturally occurring in humans and non-pathogenic, AAVs are considered safe, as they have not been associated with any known diseases. This makes them particularly useful for in vivo gene delivery, especially for therapies requiring sustained or long-term gene expression, since AAV vectors can integrate into cells and maintain their function over extended periods.

Different AAV serotypes have unique tissue-targeting properties, allowing treatments to be tailored to specific conditions. For instance, AAV9 can cross the blood-brain barrier, making it ideal for treating central nervous system (CNS) disorders, while AAV2 has demonstrated effectiveness in eye-related therapies. This adaptability enables AAV vectors to address a variety of genetic disorders, such as hemophilia, spinal muscular atrophy (SMA), and inherited retinal diseases. Notably, AAV-based treatments like Luxturna and Zolgensma have received regulatory approval for rare genetic conditions, marking significant progress in the field of gene therapy.

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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

DelveInsight's AAV Vectors in Gene Therapy pipeline report depicts a robust space with 25+ active players working to develop 30+ pipeline therapies for AAV Vectors in Gene Therapy treatment.
Key AAV Vectors in Gene Therapy companies such as Belief BioMed, Gensight Biologics, Johnson & Johnson Innovative Medicine, MeiraGTx, Passage Bio, InnoVec Biotherapeutics, Ultragenyx Pharmaceutical, MeiraGTx, Biogen, REGENXBIO, Beacon Therapeutics, Genethon, 4D molecular therapeutics, Adverum Biotechnologies, Rocket Pharmaceuticals, Innostellar Biotherapeutics, Aspa therapeutics, iECURE, AviadoBio, Sarepta therapeutics, Ray Therapeutics, Genascence Corporation, Exegenesis Bio, DiNAQOR, Tenaya Therapeutics, InnoVec Biotherapeutics, Jaguar Gene Therapy, LLC, Neurophth, and others are evaluating new drugs for AAV Vectors in Gene Therapy to improve the treatment landscape.
Promising AAV Vectors in Gene Therapy pipeline therapies in various stages of development include BBM-H901, GS010, AAV-CNGA3, PBGM01, IVB102, and others.

Recent breakthroughs in the AAV Vectors in Gene Therapy Pipeline Segment:

In June 2023, the FDA approved Roctavian, an AAV-based gene therapy developed by BioMarin Pharmaceutical Inc., for adults with severe hemophilia A. Roctavian delivers a functional gene to produce clotting Factor VIII, addressing the underlying cause of the bleeding disorder. Clinical trials demonstrated a reduction in annual bleeding rates among treated patients.
Later in June 2023, the FDA granted accelerated approval to Elevidys, an AAV-mediated gene therapy by Sarepta Therapeutics, for pediatric patients aged 4 to 5 with DMD. Elevidys introduces a gene encoding a shortened form of dystrophin, a protein lacking in DMD patients, aiming to improve muscle function.
In December 2025, Pfizer terminated its collaboration with Sangamo Therapeutics on a hemophilia A gene therapy, giroctocogene fitelparvovec, despite positive Phase 3 trial results. Pfizer cited clinical trial outcomes, expert opinions, and limited patient interest in additional gene therapies for hemophilia A as reasons for the decision. This termination significantly impacted Sangamo's stock value and delayed potential market entry.
In March 2025, a patient receiving Elevidys for DMD experienced acute liver failure leading to death. The patient had also contracted cytomegalovirus, known to affect the liver. This incident raised concerns about the safety profile of AAV-based therapies, particularly regarding liver complications, and affected Sarepta's market performance.

AAV Vectors in Gene Therapy Pipeline Analysis
The report provides insights into:
The report provides detailed insights into the key companies that are developing therapies in the AAV Vectors in Gene Therapy Market.
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.
It analyzes the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
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.
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.

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AAV Vectors in Gene Therapy Emerging Drugs

BBM-H901: Belief BioMed
GS010: Gensight Biologics
AAV-CNGA3: Johnson & Johnson Innovative Medicine/ MeiraGTx
PBGM01: Passage Bio
IVB102: InnoVec Biotherapeutics

AAV Vectors in Gene Therapy Companies

More than 180 leading companies are actively developing therapies using Adeno-Associated Virus (AAV) vectors for gene therapy. Among them, Belief BioMed has drug candidates in the most advanced stage of development, the registration phase.

DelveInsight's report covers around 30+ 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

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

Intravenous
Subcutaneous
Oral
Intramuscular

AAV Vectors in Gene Therapy Products have been categorized under various Molecule types such as

Monoclonal antibody
Small molecule
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 @ https://www.delveinsight.com/report-store/adeno-associated-virus-aav-vectors-in-gene-therapy-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

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 @ https://www.delveinsight.com/sample-request/adeno-associated-virus-aav-vectors-in-gene-therapy-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

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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Contact Info:
Kritika Rehani
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info@delveinsight.com
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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.

This release was published on openPR.