Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline 2025: Innovations by 180+ Global Leaders - DelveInsight | Featuring GenSight Biologics, Ultragenyx Pharmaceutical, MeiraGTx

Adeno-Associated Virus (AAV) Vectors in Gene Therapy companies are 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.
As the use of Adeno-Associated Virus (AAV) vectors in gene therapy expands globally, there is increasing demand for safer and more effective treatment options, addressing conditions such as diabetes, cardiovascular disease, and certain cancers. DelveInsight reports that over 180 pharmaceutical and biotech companies are actively developing more than 250 therapeutic candidates targeting AAV-based gene therapies. These programs span multiple stages of clinical and preclinical development, reflecting significant innovation and dedication to tackling key public health challenges.

The "Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline Insight 2025" report from DelveInsight offers a comprehensive analysis of the R&D landscape, including clinical trial progress, emerging therapies, mechanisms of action, competitive positioning, and major company initiatives. It serves as a vital resource for researchers, healthcare investors, and decision-makers seeking insights into the evolving AAV gene therapy market and the breakthroughs shaping its future.

Explore the Cutting-Edge Landscape of Adeno-Associated Virus (AAV) Vectors in Gene Therapy Drug Development @ https://www.delveinsight.com/report-store/adeno-associated-virus-aav-vectors-in-gene-therapy-pipeline-insight?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr

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

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DelveInsight's report on the Adeno-Associated Virus (AAV) Vectors in Gene Therapy pipeline highlights a dynamic landscape with over 180 active companies developing more than 250 therapies targeting AAV-based gene therapies.

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In November 2024, the FDA approved Kebilidi, the first gene therapy delivered directly into the brain to treat aromatic L-amino acid decarboxylase (AADC) deficiency. This AAV2-based therapy provides the DDC gene, essential for dopamine and serotonin production. Earlier, in April 2024, Beqvez, an AAV-based therapy from Pfizer for adults with moderate to severe hemophilia B, was approved, delivering a functional FIX gene to substantially reduce bleeding episodes.

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Leading companies in the AAV gene therapy space 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, Sio Gene Therapies, and others, all working to advance treatments in this field.

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Notable pipeline therapies under development include DTX401, SRP-9003, GNT-0003, RP-A501, HG202, RTX 015, IVB102, ZM-01, and several others.

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

Adeno-Associated Virus (AAV) vectors are regarded as highly effective tools for gene therapy due to their ability to deliver therapeutic genes precisely to target cells while triggering minimal immune responses. Being naturally occurring and non-pathogenic in humans, AAVs are considered safe, with no known association with human disease. This favorable safety profile makes them ideal for in vivo gene delivery, enabling sustained or long-term gene expression and prolonged therapeutic effects.

Different AAV serotypes possess unique tissue-targeting properties, allowing therapies to be tailored to specific medical needs. For instance, AAV9 can cross the blood-brain barrier, making it suitable for neurological disorders, whereas AAV2 effectively targets ocular tissues. This adaptability allows AAVs to treat a wide array of genetic conditions, including hemophilia, spinal muscular atrophy (SMA), and inherited retinal diseases. Approved AAV-based therapies like Luxturna and Zolgensma highlight significant milestones in the advancement of gene therapy.

Download the 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 @ 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

Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline Analysis

The Adeno-Associated Virus (AAV) Vectors in Gene Therapy pipeline insights report 2025, provides insights into:

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Provides comprehensive insights into key companies developing therapies in the Adeno-Associated Virus (AAV) Vectors in Gene Therapy Market.

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Categorizes Adeno-Associated Virus (AAV) Vectors in Gene Therapy therapeutic companies by development stage: early, mid, and late-stage.

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Highlights major companies involved in targeted therapy development, including both active and inactive (paused/discontinued) projects.

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Reviews emerging Adeno-Associated Virus (AAV) Vectors in Gene Therapy drugs under development based on:

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Stage of development

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Adeno-Associated Virus (AAV) Vectors in Gene Therapy Route of administration

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Target receptor

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Monotherapy vs. combination therapy

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Adeno-Associated Virus (AAV) Vectors in Gene Therapy Mechanism of action

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Molecular type

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Offers detailed analysis of:

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Company-to-company and company-academia collaborations

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Adeno-Associated Virus (AAV) Vectors in Gene Therapy Licensing agreements

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Funding and investment activities supporting future Adeno-Associated Virus (AAV) Vectors in Gene Therapy market advancement.

Unlock key insights into emerging Adeno-Associated Virus (AAV) Vectors in Gene Therapy therapies and market strategies here: https://www.delveinsight.com/report-store/adeno-associated-virus-aav-vectors-in-gene-therapy-pipeline-insight?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr

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

DTX401 - Ultragenyx PharmaceuticalDTX401 is an investigational gene therapy using the AAV8 vector, designed to provide sustained expression of the G6Pase- enzyme under its native promoter. Administered via a single intravenous infusion, it has demonstrated encouraging preclinical results, boosting G6Pase- activity and reducing liver glycogen levels-an important measure of disease severity. In a Phase I/II study, all nine participants showed positive clinical outcomes, including reduced dependence on cornstarch and better glucose and metabolic control. DTX401 is now progressing through Phase III trials for the treatment of Glycogen Storage Disease Type I.

SRP-9003 - Sarepta TherapeuticsSRP-9003 is an investigational therapy aimed at treating limb-girdle muscular dystrophy type 2E (LGMD2E), also referred to as beta-sarcoglycanopathy or LGMDR4. This inherited neuromuscular disorder is caused by a lack of the beta-sarcoglycan (-SG) protein. SRP-9003 works by delivering a full-length -SG gene directly to skeletal and cardiac muscles, addressing the underlying cause of progressive muscle deterioration and reduced life expectancy in affected patients.

GNT-0003 - Genethon

GNT-0003 is an experimental gene therapy developed by Genethon for the treatment of Crigler-Najjar syndrome. Utilizing an AAV8 vector, it delivers functional UGT1A1 genes, which produce an enzyme essential for bilirubin metabolism. Given via intravenous infusion, the therapy seeks to address the root genetic defect by enabling sustained expression of the functional gene. It is currently being evaluated in Phase II clinical trials.

RP-A501 - Rocket Pharmaceuticals

RP-A501 is Rocket's primary gene therapy candidate for Danon disease, a rare inherited cardiac disorder resulting from LAMP2 gene mutations. The therapy employs an AAV9 vector to deliver a functional human LAMP2B gene directly to heart muscle cells through intravenous infusion. RP-A501 has shown promise in restoring cardiac function by addressing the underlying genetic defect, with preclinical and clinical studies reporting encouraging safety and efficacy outcomes.

HG202 - HuidaGene TherapeuticsHG202 is an advanced RNA-targeting therapy created with HuidaGene's HG-PRECISE CRISPR/Cas13 platform. Delivered via a single AAV vector, it aims to lower VEGF-A mRNA levels in the retina, positioning it as a potential treatment for neovascular age-related macular degeneration (nAMD), particularly in patients unresponsive to existing anti-VEGF therapies. In preclinical studies, HG202 demonstrated superior inhibition of abnormal blood vessel growth compared to standard treatments like Aflibercept. It is currently being evaluated in Phase I clinical trials.

RTX 015 - Ray Therapeutics

RTX 015 (also known as RAY-001) is a gene therapy designed to treat retinitis pigmentosa, a progressive eye disorder that causes vision loss. Using an AAV vector combined with optogenetic technology, the therapy delivers a light-sensitive protein to retinal cells, with the goal of restoring light perception and potentially improving vision in patients with damaged photoreceptors.

IVB102 - InnoVec Biotherapeutics

IVB102 is an innovative therapy for X-linked retinoschisis (XLRS), developed using Innovecon's proprietary vector. Preclinical studies showed that treated animals recovered visual function similar to healthy counterparts, highlighting its therapeutic potential. The FDA has provided positive feedback on the therapy, recognizing its promise and the significant unmet medical need. IVB102 is currently being evaluated in a Phase I clinical trial.

ZM-01 - Zhongmou Therapeutics

ZM-01 is a novel AAV-based gene therapy created for retinitis pigmentosa (RP), a hereditary condition that progressively impairs vision and may result in blindness. Engineered to address the underlying genetic causes across multiple RP mutations, ZM-01 seeks to slow or stop disease progression through precise gene-targeted therapy.

Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline Therapeutic Assessment

Adeno-Associated Virus (AAV) Vectors in Gene Therapy Assessment by Product Type

- Mono

- Combination

- Mono/Combination

Adeno-Associated Virus (AAV) Vectors in Gene Therapy By Stage

- 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 Assessment by Route of Administration

- Oral

- Parenteral

- Intravenous

- Subcutaneous

- Topical

Adeno-Associated Virus (AAV) Vectors in Gene Therapy Assessment by Molecule Type

- Recombinant fusion proteins

- Small molecule

- Monoclonal antibody

- Peptide

- Polymer

- Gene therapy

Download sample pages to get an in-depth assessment of the emerging Adeno-Associated Virus (AAV) Vectors in Gene Therapy therapies and key Adeno-Associated Virus (AAV) Vectors in Gene Therapy companies [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 Contents

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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