Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline 2025: Groundbreaking Clinical Advancements by 180+ Global Leaders - DelveInsight | Featuring GenSight Biologics, Ultragenyx Pharmaceutical, MeiraGTx, Nanoscope Therapeutics, REGENXBIO, Astellas

With Adeno-Associated Virus (AAV) Vectors in Gene Therapy reaching epidemic proportions globally and contributing significantly to comorbid conditions such as diabetes, cardiovascular disease, and certain cancers, there is a growing demand for safer, more effective treatment options. According to DelveInsight, the Adeno-Associated Virus (AAV) Vectors in Gene Therapy pipeline comprises 180+ pharmaceutical and biotech companies actively developing 250+ therapeutic candidates targeting Adeno-Associated Virus (AAV) Vectors in Gene Therapy. These therapies span various stages of clinical and non-clinical development, underscoring the intense innovation and commitment to addressing one of the most pressing public health challenges of our time.
DelveInsight's "Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline Insight 2025" report provides a detailed and strategic evaluation of the ongoing R&D landscape. It covers clinical trial progression, emerging therapies, mechanisms of action, competitive positioning, and key company initiatives. The report serves as a crucial resource for stakeholders-including researchers, healthcare investors, and decision-makers, seeking insights into the evolving Adeno-Associated Virus (AAV) Vectors in Gene Therapy Therapeutics Market and the breakthroughs shaping its future trajectory.

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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 November 2024, the FDA approved Kebilidi, the first gene therapy administered directly into the brain for treating aromatic L-amino acid decarboxylase (AADC) deficiency. This AAV2-based therapy delivers the DDC gene, crucial for dopamine and serotonin production .
Approved in April 2024, Beqvez is an AAV-based gene therapy developed by Pfizer for adults with moderate to severe hemophilia B. It introduces a functional copy of the FIX gene, leading to a significant reduction in bleeding episodes .
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 Overview:

Adeno-Associated Virus (AAV) vectors are widely considered among the most effective tools for gene therapy, thanks to their precision in delivering therapeutic genes to specific cells while eliciting only a mild immune reaction. As naturally occurring viruses that are non-pathogenic to humans, AAVs are deemed safe, with no known links to human disease. This strong safety profile makes them particularly suitable for in vivo gene delivery, where consistent or long-lasting gene expression is desired. Once inside the cells, AAV vectors can remain active, promoting sustained therapeutic effects.

Various AAV serotypes have distinct tissue-targeting abilities, allowing for customized treatments based on specific medical needs. For example, AAV9 can cross the blood-brain barrier, making it ideal for addressing neurological disorders, while AAV2 is effective in targeting ocular tissues. This versatility enables AAVs to be used in treating a broad range of genetic disorders, such as hemophilia, spinal muscular atrophy (SMA), and inherited retinal diseases. Notable AAV-based therapies, including Luxturna and Zolgensma, have gained regulatory approval, representing major milestones in the progress of gene therapy.

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

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

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

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

Stage of development

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

Target receptor

Monotherapy vs. combination therapy

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

Molecular type

Offers detailed analysis of:

Company-to-company and company-academia collaborations

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

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=openpr&utm_medium=pressrelease&utm_campaign=kpr

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

DTX401 - Ultragenyx Pharmaceutical

DTX401 is an experimental gene therapy based on the AAV8 vector, designed to enable consistent expression and function of the G6Pase-α enzyme, regulated by its natural promoter. Delivered through a single intravenous dose, it has shown promising preclinical results, enhancing G6Pase-α activity and lowering liver glycogen levels-a key indicator of disease severity. In a Phase I/II trial, all nine participants exhibited positive clinical responses, including decreased reliance on cornstarch and improved glucose and metabolic control. DTX401 is currently undergoing Phase III evaluation 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. This genetic neuromuscular disorder stems from a deficiency in beta-sarcoglycan (β-SG) protein. SRP-9003 delivers the full-length β-SG gene to skeletal and cardiac muscles, targeting the root cause of the progressive muscle degeneration and shortened lifespan associated with the condition.
GNT-0003 - Genethon

GNT-0003 is an investigational gene therapy created by Genethon to treat Crigler-Najjar syndrome. It uses an AAV8 vector to deliver functional copies of the UGT1A1 gene, which encodes an enzyme crucial for bilirubin metabolism. Administered intravenously, the therapy aims to correct the underlying gene defect through durable expression of a working version of the gene. It is currently in Phase II clinical development.
RP-A501 - Rocket Pharmaceuticals

RP-A501 is Rocket's lead gene therapy candidate for Danon disease, a rare genetic heart disorder caused by mutations in the LAMP2 gene. The therapy uses an AAV9 vector carrying the functional human LAMP2B gene, which is delivered directly to heart muscle cells via intravenous infusion. RP-A501 has demonstrated the potential to restore cardiac function by correcting the genetic defect, with encouraging safety and efficacy results observed in both preclinical and clinical studies.
HG202 - HuidaGene Therapeutics

HG202 is a cutting-edge RNA-targeting therapy developed using HuidaGene's HG-PRECISE CRISPR/Cas13 platform. Encapsulated in a single AAV vector, it is designed to reduce VEGF-A mRNA levels in the retina, making it a candidate for treating neovascular age-related macular degeneration (nAMD), especially in patients resistant to current anti-VEGF treatments. In preclinical comparisons, HG202 outperformed standard therapies like Aflibercept in suppressing abnormal blood vessel growth. It is currently in Phase I trials.
RTX 015 - Ray Therapeutics

RTX 015 (also called RAY-001) is a gene therapy developed for retinitis pigmentosa, a degenerative eye condition leading to vision loss. Utilizing an AAV vector and optogenetic technology, the therapy introduces a light-sensitive protein into retinal cells, aiming to restore light responsiveness and potentially vision in individuals with damaged photoreceptors.
IVB102 - InnoVec Biotherapeutics

IVB102 is a novel therapy targeting X-linked retinoschisis (XLRS), based on a proprietary vector from Innovecon. Preclinical studies demonstrated that treated animals regained visual function comparable to healthy controls, indicating strong therapeutic potential. The therapy has received favorable feedback from the FDA, emphasizing both its promise and the unmet clinical need. It is now in Phase I clinical evaluation.
ZM-01 - Zhongmou Therapeutics

ZM-01 is an innovative AAV-based gene therapy developed for retinitis pigmentosa (RP), a genetic disorder that causes progressive vision deterioration and can lead to blindness. Designed to tackle the root causes of RP across various genetic mutations, ZM-01 aims to slow or halt disease progression through targeted genetic intervention.

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

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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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