Adeno-Associated Virus (AAV) Vectors in Gene Therapy Clinical, Companies, Therapeutic Assessment, Therapies, Treatment Algorithm, Pipeline Analysis | GenSight Biologics, Ultragenyx Pharmaceutical, MeiraGTx, Nanoscope Therapeutics, REGENXBIO, Astellas Gene

Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline constitutes 180+ key companies continuously working towards developing 250+ Adeno-Associated Virus (AAV) Vectors in Gene Therapy treatment therapies, analyzes DelveInsight.

Adeno-Associated Virus (AAV) Vectors in Gene Therapy Overview:

Adeno-Associated Virus (AAV) vectors are considered among the most efficient tools for gene therapy due to their precision in delivering therapeutic genes to targeted cells while eliciting a low immune response. These viruses occur naturally and are non-pathogenic in humans, with no known links to human diseases, which contributes to their favorable safety profile. This makes AAV vectors particularly suitable for in vivo gene delivery, especially when sustained or long-term gene expression is needed. AAVs can remain within cells for extended periods, supporting prolonged therapeutic effects.

Each AAV serotype exhibits distinct tissue-targeting capabilities, allowing gene therapies to be customized based on specific treatment goals. For example, AAV9 can penetrate the blood-brain barrier, making it ideal for neurological conditions, while AAV2 is effective in delivering genes to ocular tissues. This versatility enables the use of AAVs in treating a range of inherited disorders, such as hemophilia, spinal muscular atrophy (SMA), and genetic eye diseases. Approved AAV-based therapies like Luxturna and Zolgensma have represented major milestones in advancing gene therapy for rare genetic conditions.

However, challenges remain-particularly in the areas of production and scalability. Manufacturing AAV vectors for clinical use requires stringent processes to ensure high purity and stability, which can be both technically demanding and expensive. These difficulties contribute to the high cost of AAV-based treatments. To tackle this, novel production methods like the baculovirus-insect cell system have been explored to enhance scalability. Still, continued innovation in manufacturing is critical to improving the affordability and broader availability of AAV gene therapies.

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"Adeno-Associated Virus (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 Adeno-Associated Virus (AAV) Vectors in Gene Therapy Therapeutics Market.

Key Takeaways from the Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline Report

DelveInsight's Adeno-Associated Virus (AAV) Vectors in Gene Therapy pipeline report depicts a robust space with 180+ active players working to develop 250+ pipeline therapies for Adeno-Associated Virus (AAV) Vectors in Gene Therapy treatment.
In March 2025, Dr. Peter Marks resigned from the FDA's Center for Biologics Evaluation and Research. His successor, under a new FDA leadership style that emphasizes scrutiny and cost-control, may impose tighter standards on gene therapy reviews, suggesting slower approvals and deeper scrutiny of manufacturing processes.
Key Adeno-Associated Virus (AAV) Vectors in Gene Therapy companies such as GenSight Biologics, Ultragenyx Pharmaceutical, MeiraGTx, Nanoscope Therapeutics, REGENXBIO, Astellas Gene therapy, GeneCradle Therapeutics, Asklepios BioPharmaceutical, MeiraGTx, 4D molecular therapeutics, Adverum Biotechnologies, Rocket Pharmaceuticals, Innostellar Biotherapeutics, Passage Bio, Aspa therapeutics, Solid Biosciences, Lantu Biopharma, Ascidian Therapeutics, Decibel Therapeutics, Sio Gene Therapies, and others are evaluating new drugs for Adeno-Associated Virus (AAV) Vectors in Gene Therapy to improve the treatment landscape.
Promising Adeno-Associated Virus (AAV) Vectors in Gene Therapy pipeline therapies in various stages of development include DTX401, SRP-9003, GNT-0003, RP-A501, HG202, RTX 015, IVB102, ZM-01, and others.

Adeno-Associated Virus (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 Adeno-Associated Virus (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 Adeno-Associated Virus (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 Adeno-Associated Virus (AAV) Vectors in Gene Therapy market.

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Adeno-Associated Virus (AAV) Vectors in Gene Therapy Emerging Drugs

DTX401 - Ultragenyx Pharmaceutical

DTX401 is an experimental gene therapy using an AAV8 vector, developed to enable sustained expression and function of the G6Pase-α enzyme, regulated by its native promoter. Delivered via a single intravenous infusion, the therapy has demonstrated in preclinical studies its ability to enhance G6Pase-α activity and lower liver glycogen accumulation-a key marker of disease progression. In a Phase I/II trial, all nine participants exhibited positive clinical outcomes, including reduced dependence on cornstarch and improved glucose stability and metabolic markers. DTX401 is currently in a Phase III clinical trial for the treatment of Glycogen Storage Disease Type I.

SRP-9003 - Sarepta Therapeutics

SRP-9003 is being developed to treat Limb-Girdle Muscular Dystrophy Type 2E (LGMD2E), also known as beta-sarcoglycanopathy or LGMDR4-a severe genetic neuromuscular disorder caused by the absence of beta-sarcoglycan (β-SG) protein. This gene therapy is designed to deliver the full-length β-SG gene directly to skeletal and heart muscle, aiming to restore the missing protein and address the root cause of muscle degeneration and the shortened lifespan associated with the disease.

GNT-0003 - Genethon

GNT-0003 is a gene therapy candidate that introduces functional copies of the UGT1A1 gene, which encodes the enzyme responsible for metabolizing bilirubin. Developed by Genethon's team specializing in liver-targeted gene therapies, the treatment uses an AAV8 viral vector to deliver the gene via intravenous administration. The goal is to achieve long-lasting expression of the healthy gene to correct the genetic deficiency. GNT-0003 is currently in Phase II clinical trials for the treatment of Crigler-Najjar syndrome.

Adeno-Associated Virus (AAV) Vectors in Gene Therapy Companies

Over 180 key companies are actively engaged in developing therapies utilizing AAV vectors in gene therapy. Among them, Ultragenyx Pharmaceutical has a drug candidate that is currently in the most advanced stage of development-Phase III clinical trials.

DelveInsight's report covers around 75+ 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

Adeno-Associated Virus (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

Adeno-Associated Virus (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 Adeno-Associated Virus (AAV) Vectors in Gene Therapy Therapies and Key Companies: Adeno-Associated Virus (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

Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline Therapeutic Assessment
• Adeno-Associated Virus (AAV) Vectors in Gene Therapy Assessment by Product Type
• Adeno-Associated Virus (AAV) Vectors in Gene Therapy By Stage
• Adeno-Associated Virus (AAV) Vectors in Gene Therapy Assessment by Route of Administration
• Adeno-Associated Virus (AAV) Vectors in Gene Therapy Assessment by Molecule Type

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Table of Content

1. Report Introduction
2. Executive Summary
3. Adeno-Associated Virus (AAV) Vectors in Gene Therapy Current Treatment Patterns
4. Adeno-Associated Virus (AAV) Vectors in Gene Therapy - DelveInsight's Analytical Perspective
5. Therapeutic Assessment
6. Adeno-Associated Virus (AAV) Vectors in Gene Therapy Late-Stage Products (Phase-III)
7. Adeno-Associated Virus (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. Adeno-Associated Virus (AAV) Vectors in Gene Therapy Discontinued Products
13. Adeno-Associated Virus (AAV) Vectors in Gene Therapy Product Profiles
14. Adeno-Associated Virus (AAV) Vectors in Gene Therapy Key Companies
15. Adeno-Associated Virus (AAV) Vectors in Gene Therapy Key Products
16. Dormant and Discontinued Products
17. Adeno-Associated Virus (AAV) Vectors in Gene Therapy Unmet Needs
18. Adeno-Associated Virus (AAV) Vectors in Gene Therapy Future Perspectives
19. Adeno-Associated Virus (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.

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