DelveInsight’s, “Adeno-Associated Virus (AAV) Vectors in Gene Therapy - Pipeline Insight, 2025” report provides comprehensive insights about 180+ companies and 250+ pipeline drugs in AAV Vectors in Gene Therapy pipeline landscape. It covers the AAV Vectors in Gene Therapy pipeline drug profiles, including clinical and nonclinical stage products. It also covers the AAV Vectors in Gene Therapy pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
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Key Takeaways from the AAV Vectors in Gene Therapy Pipeline Report
- In July 2025, Adverum Biotechnologies Inc. announced a Phase 3 study in a broad participant population (treatment-naïve and treatment-experienced) with neovascular (wet) age-related macular degeneration (nAMD). The study will evaluate a single intravitreal (IVT) injection of Ixo-vec compared to an active comparator. The primary endpoint of this study is the mean change in best corrected visual acuity (BCVA) of Ixo-vec compared to an active comparator measured at an average of Weeks 52 and 56.
- DelveInsight’s AAV Vectors in Gene Therapy pipeline report depicts a robust space with 180+ active players working to develop 250+ pipeline therapies for AAV Vectors in Gene Therapy treatment.
- The leading 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.
- Promising AAV Vectors in Gene Therapy Pipeline Therapies such as Valoctocogene Roxaparvovec, AAV - CNGB3, SB-525 (PF-07055480), AAV2/5-RPGR, BMN 307, GC301, rAAV-Olig001-ASPA, and others.
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AAV Vectors in Gene Therapy Emerging Drugs Profile
- DTX401: Ultragenyx Pharmaceutical
DTX401 is an investigational AAV8 gene therapy designed to deliver stable expression and activity of G6Pase-α under control of the native promoter. DTX401 is administered as a single intravenous infusion and has been shown in preclinical studies to improve G6Pase-α activity and reduce hepatic glycogen levels, a well-described biomarker of disease progression. In a Phase I/II clinical study, all nine patients showed a clinical response, with significant reductions in the need for cornstarch and improvements in glucose control and other metabolic parameters compared to baseline. The drug is currently being evaluated in Phase III clinical trial to treat patients with Glycogen storage disease type I.
- SRP-9003: Sarepta Therapeutics
SRP-9003 is in development for the treatment of LGMD2E (also known as beta-sarcoglycanopathy and LGMDR4), a devastating monogenic neuromuscular disease caused by a lack of beta-sarcoglycan (beta-SG) proteins. SRP-9003 is a gene therapy construct that transduces skeletal and cardiac muscle, delivering a gene that codes for the full-length beta-SG protein, the absence of which is the sole cause of the progressive degeneration and a shortened lifespan characterized by the disease.
- GNT-0003: Genethon
GNT-0003 combines normal copies of the UGT1A1 gene coding for the bilirubin metabolizing enzyme with an AAV vector. The gene therapy, administered intravenously, was designed by Genethon’s Immunology and Gene Therapy of Liver Diseases team, led by Dr. Giuseppe Ronzitti. GNT0003 is a genetically modified recombinant (r) viral vector composed of the AAV8 viral capsid carrying the UGT1A1 transgene which aims to correct the dysfunction of the mutated gene by achieving durable expression of a functional copy of the affected gene. The drug is currently in Phase II stage of development for the treatment of Crigler-Najjar syndrome.
- RP-A501: Rocket Pharmaceuticals
RP-A501 is Rocket’s investigational gene therapy product for the treatment of Danon disease and the first gene therapy for a cardiovascular condition to demonstrate safety and efficacy in clinical studies. Danon disease is caused by mutations in the LAMP2 gene. RP-A501 consists of a recombinant adeno-associated serotype 9 (AAV9) capsid containing a full-length, wild-type version of the human LAMP2B transgene (AAV9.LAMP2B) which, when inserted into heart cells harboring mutations in the endogenous LAMP2 gene, has the potential to fully restore cardiac function at its root. RP-A501 represents a single dose treatment and is administered as an intravenous infusion. In preclinical and clinical studies, AAV9.LAMP2B has been shown to target cardiac cells (cardiomyocytes) and deliver the functional LAMP2B gene to heart tissue, which ultimately leads to improved cardiac structure and function in patients.
- HG202: HuidaGene Therapeutics
HG202 is a novel CRISPR/Cas13 RNA-targeting therapy packaged in a single adeno-associated viral (AAV) vector independently-developed by HuidaGene's proprietary HG-PRECISE platform to target and knockdown the expression of VEGF-A mRNA within the local retina. This HG202 therapy is designed to treat nAMD patients who have developed resistance to anti-VEGF treatment as well as patients who are responsive to current standard anti-VEGF therapies. HG202 efficiently inhibits choroidal neovascularization better than existing therapies such as Aflibercept antibodies in head-to-head preclinical comparison studies. HG202 is currently being evaluated in a Phase I trial for the treatment of neovascular Age-related Macular Degeneration (nAMD).
- RTX 015: Ray Therapeutics
RTX 015, also known as RAY-001, is a gene therapy that utilizes an adeno-associated virus (AAV) vector. It is being developed by Ray Therapeutics specifically for the treatment of retinitis pigmentosa, a degenerative eye disease that leads to vision loss. The therapy employs optogenetics, which involves the introduction of a gene that encodes a light-sensitive protein into retinal cells, thereby enabling these cells to respond to light and potentially restore vision in individuals with damaged photoreceptors.
- IVB102: InnoVec Biotherapeutics
IVB102 is a drug for the treatment of X-linked retinoschisis (XLRS) developed based on a new vector independently developed by Innovecon. Preclinical data showed that the visual electrophysiological signals of model animals treated with IVB102 can be restored to a level comparable to that of wild-type animals, with the potential of being 'best in class'. The positive feedback received from the FDA on IVB102 proves the FDA's recognition of IVB102 and also reflects the urgent need for drugs from regulators and patients. Innovecon is rapidly advancing the clinical research of IVB102. The drug is currently being evaluated in a Phase I trial for the treatment of XLRS.
- ZM-01: Zhongmou Therapeutics
ZM-01 is a novel gene therapy developed by Zhongmou Therapeutics, specifically designed for treating retinitis pigmentosa (RP), a hereditary retinal disease that leads to progressive vision loss and can result in legal blindness. This therapy utilizes an adeno-associated virus (AAV) vector to deliver genetic material aimed at addressing the underlying causes of RP, regardless of the specific mutations involved.
The AAV Vectors in Gene Therapy Pipeline Report provides insights into
- The report provides detailed insights about companies that are developing therapies for the treatment of AAV Vectors in Gene Therapy with aggregate therapies developed by each company for the same.
- It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for AAV Vectors in Gene Therapy Treatment.
- AAV Vectors in Gene Therapy Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
- AAV Vectors in Gene Therapy 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 Companies
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.
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
- Intra-articular
- Intraocular
- Intrathecal
- Intravenous
- Oral
- Parenteral
- Subcutaneous
- Topical
- Transdermal
AAV Vectors in Gene Therapy Products have been categorized under various Molecule types such as
- Oligonucleotide
- Peptide
- Small molecule
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Scope of the AAV Vectors in Gene Therapy Pipeline Report
- Coverage- Global
- AAV Vectors in Gene Therapy Companies- 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.
- AAV Vectors in Gene Therapy Pipeline Therapies- Valoctocogene Roxaparvovec, AAV-CNGB3, SB-525 (PF-07055480), AAV2/5-RPGR, BMN 307, GC301, rAAV-Olig001-ASPA, and others.
- AAV Vectors in Gene Therapy Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
- AAV Vectors in Gene Therapy Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
For a detailed overview of our latest research findings and future plans, read the full details of AAV Vectors in Gene Therapy Pipeline on our website @ AAV Vectors in Gene Therapy Emerging Drugs and Companies
Table of Contents
- Introduction
- Executive Summary
- AAV Vectors in Gene Therapy: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- AAV Vectors in Gene Therapy – DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- DTX401: Ultragenyx Pharmaceutical
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- GNT-0003: Genethon
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- HG202: HuidaGene Therapeutics
- Preclinical Stage Products
- Drug Name: Company Name
- Drug profiles in the detailed report…..
- Inactive Products
- AAV Vectors in Gene Therapy - Collaborations Assessment- Licensing / Partnering / Funding
- AAV Vectors in Gene Therapy - Unmet Needs
- AAV Vectors in Gene Therapy - Market Drivers and Barriers
- Appendix
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