Adeno-Associated Virus (AAV) Vectors in Gene Therapy: Clinical Insights, Key Companies, Therapeutic Evaluation, Treatment Approaches, and Pipeline Analysis | GenSight Biologics, Ultragenyx Pharmaceuti

Adeno-Associated Virus (AAV) Vectors in Gene Therapy companies are MeiraGTx, Nanoscope Therapeutics, REGENXBIO, Astellas Gene Therapy, GeneCradle Therapeutics, Asklepios BioPharmaceutical, 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.
According to DelveInsight, the Adeno-Associated Virus (AAV) Vectors in Gene Therapy pipeline features over 180 active companies engaged in the ongoing development of more than 250 therapeutic candidates targeting a wide range of genetic disorders.

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

Adeno-Associated Virus (AAV) vectors are widely recognized as one of the most effective delivery systems in gene therapy, valued for their ability to accurately transfer therapeutic genes into target cells while provoking minimal immune reactions. Naturally occurring and non-pathogenic to humans, AAVs have an excellent safety record, as they are not associated with any known diseases. This makes them ideal candidates for in vivo applications where stable and long-term gene expression is desired. Once inside target cells, AAVs can persist for extended durations, sustaining therapeutic benefits over time.

Different AAV serotypes possess unique tissue-targeting abilities, enabling precise customization of gene therapies for specific disorders. For instance, AAV9 can cross the blood-brain barrier, making it suitable for neurological diseases, whereas AAV2 is commonly used in ocular gene delivery. Such adaptability has made AAV vectors pivotal in treating various genetic disorders, including hemophilia, spinal muscular atrophy (SMA), and inherited retinal diseases. The approvals of AAV-based therapies such as Luxturna and Zolgensma have marked transformative milestones in modern gene therapy.

Despite these successes, significant manufacturing and scalability challenges persist. Producing high-quality AAV vectors at clinical scale demands rigorous purification and stability processes, which are complex and costly-contributing to the high price of AAV-based treatments. To overcome these barriers, innovative production platforms like the baculovirus-insect cell system are being developed to enhance scalability and reduce costs. Continued advancements in vector engineering and large-scale manufacturing remain crucial to expanding access to AAV gene therapies globally.

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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

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DelveInsight's report on Adeno-Associated Virus (AAV) Vectors in Gene Therapy highlights a dynamic and competitive landscape, with over 180 active companies developing 250+ gene therapy candidates utilizing AAV vector technology.

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In March 2025, Dr. Peter Marks stepped down from his position at the FDA's Center for Biologics Evaluation and Research (CBER). His successor, under a new FDA leadership approach emphasizing heightened regulatory scrutiny and cost-efficiency, is expected to implement stricter oversight of gene therapy evaluations-potentially resulting in slower approval timelines and more rigorous review of manufacturing quality.

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Leading companies driving progress in the AAV gene therapy field include GenSight Biologics, Ultragenyx Pharmaceutical, MeiraGTx, Nanoscope Therapeutics, REGENXBIO, Astellas Gene Therapy, GeneCradle Therapeutics, Asklepios BioPharmaceutical, 4D Molecular Therapeutics, Adverum Biotechnologies, Rocket Pharmaceuticals, Innostellar Biotherapeutics, Passage Bio, Aspa Therapeutics, Solid Biosciences, Lantu Biopharma, Ascidian Therapeutics, Decibel Therapeutics, and Sio Gene Therapies, among others.

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Promising AAV-based gene therapy candidates currently in various stages of development include DTX401, SRP-9003, GNT-0003, RP-A501, HG202, RTX-015, IVB102, ZM-01, and several others, reflecting continuous innovation and advancement in the field.

Adeno-Associated Virus (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 Adeno-Associated Virus (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 Adeno-Associated Virus (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 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 investigational gene therapy utilizing an AAV8 vector designed to achieve long-term expression and function of the G6Pase- enzyme under the control of its native promoter. Administered as a single intravenous infusion, the therapy has shown in preclinical studies to increase G6Pase- activity and reduce liver glycogen accumulation, a key indicator of disease severity.

In a Phase I/II clinical trial, all nine treated patients demonstrated favorable outcomes, including a decrease in cornstarch dependence, improved blood glucose stability, and enhanced metabolic control. DTX401 is currently being evaluated in a Phase III clinical trial for Glycogen Storage Disease Type I (GSD-I).

SRP-9003 - Sarepta Therapeutics

SRP-9003 is an investigational gene therapy developed for Limb-Girdle Muscular Dystrophy Type 2E (LGMD2E), also known as beta-sarcoglycanopathy (LGMDR4)-a rare, inherited neuromuscular disorder resulting from the lack of beta-sarcoglycan (-SG) protein. The therapy is engineered to deliver a functional, full-length -SG gene directly to skeletal and cardiac muscle tissues, aiming to restore normal protein expression, halt progressive muscle degeneration, and address the underlying cause of the disease's severe symptoms and reduced life expectancy.

GNT-0003 - Genethon

GNT-0003 is an investigational gene therapy designed to deliver functional copies of the UGT1A1 gene, which encodes the enzyme crucial for bilirubin metabolism. Developed by Genethon's liver-targeted gene therapy division, it utilizes an AAV8 viral vector administered intravenously to introduce the therapeutic gene. The treatment aims to achieve sustained expression of the functional UGT1A1 gene to correct the underlying genetic deficiency. GNT-0003 is currently being evaluated in Phase II clinical trials for Crigler-Najjar syndrome.

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

More than 180 major companies are actively working on developing AAV vector-based gene therapies. Among these, Ultragenyx Pharmaceutical leads with a drug candidate currently progressing through Phase III clinical trials, representing the most advanced stage of development in this space.

DelveInsight's report covers around 75+ 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

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

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Intravenous

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Subcutaneous

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Oral

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Intramuscular

Adeno-Associated Virus (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

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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

Download Adeno-Associated Virus (AAV) Vectors in Gene Therapy Sample report to know in detail about the Adeno-Associated Virus (AAV) Vectors in Gene Therapy treatment market @ Adeno-Associated Virus (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=abnewswire&utm_medium=market&utm_campaign=kpr]

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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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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