“AAV for the Hereditary Retinal Diseases Competitive landscape, 2025" report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the AAV for the Hereditary Retinal Diseases Market.
The AAV for the Hereditary Retinal Diseases Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.
Some of the key takeaways from the AAV for the Hereditary Retinal Diseases Pipeline Report:
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Companies across the globe are diligently working toward developing novel AAV for the Hereditary Retinal Diseases treatment therapies with a considerable amount of success over the years.
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AAV for the Hereditary Retinal Diseases companies working in the treatment market are Novartis, Johnson & Johnson, Beacon Therapeutics, 4D Molecular Therapeutics, Coave Therapeutics, MeiraGTx Limited, and others, are developing therapies for the AAV for the Hereditary Retinal Diseases treatment
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Emerging AAV for the Hereditary Retinal Diseases therapies in the different phases of clinical trials are- LUXTURNA, Botaretigene sparoparvovec, AGTC-501, 4D 125, CTx PDE6B, A007, and others are expected to have a significant impact on the AAV for the Hereditary Retinal Diseases market in the coming years.
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In May 2025, Ocugen has been granted rare pediatric disease designation by the FDA for OCU410ST, an investigational gene therapy using an adeno-associated virus (AAV) vector, aimed at treating ABCA4-associated retinopathies, including Stargardt disease, retinitis pigmentosa, and cone-rod dystrophy.
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In April 2025, Atsena Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ATSN-201 for the treatment of X-linked retinoschisis (XLRS).
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In March 2025, SpliceBio announced that the first patient has been dosed in the Phase I/II ASTRA clinical trial, which is assessing SB-007, a dual adeno-associated virus (AAV) vector-based gene therapy for the treatment of Stargardt disease.
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In March 2025, Avirmax Biopharma announced the initiation of Investigational New Drug (IND)-enabling studies for ABI-201, a potential AAV vector designed to deliver three genes to correct complement activation dysregulation, provide anti-inflammatory effects, and protect retinal pigment epithelium and photoreceptors. The investigational therapy also aims to inhibit retinal neovascularization.
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In December 2024, ViGeneron GmbH announced that the U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application for the Phase I/II trial of VG801, a promising gene therapy aimed at treating Stargardt disease and other retinal dystrophies linked to ABCA4 gene mutations.
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In August 2024, Opus Genetics has been granted Rare Pediatric Disease (RPD) designation by the FDA for OPGx-LCA5, its investigational ocular gene therapy, intended to treat patients with Leber congenital amaurosis (LCA) caused by biallelic mutations in the LCA5 gene.
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In July 2024, Charles River Laboratories International, Inc. and AAVantgarde announced a contract development and manufacturing organization (CDMO) partnership to produce Good Manufacturing Practice (GMP) plasmid DNA. AAVantgarde, a clinical-stage biotech company with two proprietary adeno-associated virus (AAV) vector platforms for large gene delivery and developing therapies for inherited retinal diseases, will utilize Charles River’s expertise in GMP plasmid DNA production.
AAV for the Hereditary Retinal Diseases Overview
Adeno-associated virus (AAV) vectors are used in gene therapy to treat hereditary retinal diseases, which are genetic disorders causing progressive vision loss. AAV acts as a delivery system to transport functional copies of defective genes directly into retinal cells, aiming to restore or preserve vision. These therapies are being developed for conditions like retinitis pigmentosa, Leber congenital amaurosis, and choroideremia. AAV vectors are preferred due to their ability to target retinal cells efficiently, induce long-lasting gene expression, and provoke minimal immune response, making them a key platform in ocular gene therapy research and development.
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Emerging AAV for the Hereditary Retinal Diseases Drugs Under Different Phases of Clinical Development Include:
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LUXTURNA: Novartis
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Botaretigene sparoparvovec: Johnson & Johnson/MeiraGTx
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AGTC-501: Beacon Therapeutics
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4D 125: 4D Molecular Therapeutics
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CTx PDE6B: Coave Therapeutics
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A007: MeiraGTx Limited
AAV for the Hereditary Retinal Diseases Route of Administration
AAV for the Hereditary Retinal Diseases 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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Oral
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Parenteral
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Intravenous
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Subcutaneous
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Topical
AAV for the Hereditary Retinal Diseases Molecule Type
AAV for the Hereditary Retinal Diseases Products have been categorized under various Molecule types, such as
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Monoclonal Antibody
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Peptides
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Polymer
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Small molecule
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Gene therapy
AAV for the Hereditary Retinal Diseases Pipeline Therapeutics Assessment
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AAV for the Hereditary Retinal Diseases Assessment by Product Type
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AAV for the Hereditary Retinal Diseases By Stage and Product Type
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AAV for the Hereditary Retinal Diseases Assessment by Route of Administration
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AAV for the Hereditary Retinal Diseases By Stage and Route of Administration
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AAV for the Hereditary Retinal Diseases Assessment by Molecule Type
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AAV for the Hereditary Retinal Diseases by Stage and Molecule Type
DelveInsight's AAV for the Hereditary Retinal Diseases Report covers around 80+ 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)
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Pre-clinical and Discovery stage candidates
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Discontinued & Inactive candidates
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Route of Administration
Further AAV for the Hereditary Retinal Diseases product details are provided in the report. Download the AAV for the Hereditary Retinal Diseases pipeline report to learn more about the emerging AAV for the Hereditary Retinal Diseases therapies
Some of the key companies in the AAV for the Hereditary Retinal Diseases Therapeutics Market include:
Key companies developing therapies for AAV for the Hereditary Retinal Diseases are - Coave Therapeutics, Neurophth Therapeutics, Ocugen, Atsena Therapeutics, Novartis, MeiraGTx, 4D Molecular Therapeutics, Beacon Therapeutics, SpliceBio, and others.
AAV for the Hereditary Retinal Diseases Pipeline Analysis:
The AAV for the Hereditary Retinal Diseases pipeline report provides insights into
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The report provides detailed insights about companies that are developing therapies for the treatment of AAV for the Hereditary Retinal Diseases with aggregate therapies developed by each company for the same.
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It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for AAV for the Hereditary Retinal Diseases Treatment.
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AAV for the Hereditary Retinal Diseases key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
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AAV for the Hereditary Retinal Diseases 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 AAV for the Hereditary Retinal Diseases market.
The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.
Download Sample PDF Report to know more about AAV for the Hereditary Retinal Diseases drugs and therapies
AAV for the Hereditary Retinal Diseases Pipeline Market Drivers
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Rising prevalence of hereditary retinal disorders such as retinitis pigmentosa, Leber congenital amaurosis, and choroideremia
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Advancements in gene therapy and AAV vector technology enabling targeted and effective treatment
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Growing focus on personalized medicine and precision therapies for rare genetic ocular diseases
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Increasing investments, partnerships, and collaborations among biopharmaceutical companies to accelerate pipeline development
AAV for the Hereditary Retinal Diseases Pipeline Market Barriers
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High costs associated with gene therapy research, development, and commercialization
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Challenges in ensuring long-term safety, efficacy, and stable gene expression in retinal cells
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Potential immune responses against AAV vectors, which may limit treatment effectiveness
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Limited patient populations for rare hereditary retinal diseases, impacting market size and clinical trial recruitment
Scope of AAV for the Hereditary Retinal Diseases Pipeline Drug Insight
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Coverage: Global
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Key AAV for the Hereditary Retinal Diseases Companies: Novartis, Johnson & Johnson, Beacon Therapeutics, 4D Molecular Therapeutics, Coave Therapeutics, MeiraGTx Limited, and others
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Key AAV for the Hereditary Retinal Diseases Therapies: LUXTURNA, Botaretigene sparoparvovec, AGTC-501, 4D 125, CTx PDE6B, A007, and others
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AAV for the Hereditary Retinal Diseases Therapeutic Assessment: AAV for the Hereditary Retinal Diseases current marketed and AAV for the Hereditary Retinal Diseases emerging therapies
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AAV for the Hereditary Retinal Diseases Market Dynamics: AAV for the Hereditary Retinal Diseases market drivers and AAV for the Hereditary Retinal Diseases market barriers
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Table of Contents
1. AAV for the Hereditary Retinal Diseases Report Introduction
2. AAV for the Hereditary Retinal Diseases Executive Summary
3. AAV for the Hereditary Retinal Diseases Overview
4. AAV for the Hereditary Retinal Diseases- Analytical Perspective In-depth Commercial Assessment
5. AAV for the Hereditary Retinal Diseases Pipeline Therapeutics
6. AAV for the Hereditary Retinal Diseases Late Stage Products (Phase II/III)
7. AAV for the Hereditary Retinal Diseases Mid Stage Products (Phase II)
8. AAV for the Hereditary Retinal Diseases Early Stage Products (Phase I)
9. AAV for the Hereditary Retinal Diseases Preclinical Stage Products
10. AAV for the Hereditary Retinal Diseases Therapeutics Assessment
11. AAV for the Hereditary Retinal Diseases Inactive Products
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. AAV for the Hereditary Retinal Diseases Key Companies
14. AAV for the Hereditary Retinal Diseases Key Products
15. AAV for the Hereditary Retinal Diseases Unmet Needs
16 . AAV for the Hereditary Retinal Diseases Market Drivers and Barriers
17. AAV for the Hereditary Retinal Diseases Future Perspectives and Conclusion
18. AAV for the Hereditary Retinal Diseases Analyst Views
19. Appendix
20. About DelveInsight
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. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.
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