DelveInsight’s, “Retinitis Pigmentosa Pipeline Insight 2025” report provides comprehensive insights about 40+ companies and 40+ pipeline drugs in Retinitis Pigmentosa pipeline landscape. It covers the Retinitis Pigmentosa pipeline drugs assessment profiles, including clinical and nonclinical stage products. It also covers the Retinitis Pigmentosa 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 Retinitis Pigmentosa Pipeline Drugs Assessment Report

• In May 2025, Neurotech Pharmaceuticals announced a study is to look at the safety and effectiveness of CNTF implants on vision in persons with retinitis pigmentosa, Usher type II & III, and Choroideremia. This research is being done because there are no effective therapies for people with these retinal degenerations. They are genetic disorders that affect one’s ability to see at night, and later cause tunnel vision and loss of central vision. Retinal degenerations affect the retina, a light sensitive layer of cells in the back of the eye. Slowly over time, these cells die and cause permanent loss of vision.
• In May 2025, Beacon Therapeutics conducted a study will evaluate and compare the safety, efficacy, and tolerability of 2 doses of a recombinant adeno-associated virus vector (AGTC-501/laruparetigene zovaparvovec )) to an untreated control group in male participants with X-linked retinitis pigmentosa caused by RPGR mutations.
• In May 2025, AAVantgarde Bio Srl organized 081-101 study is to evaluate the safety and tolerability of a single subretinal injection of AAVB-081 in USH1B patients with retinitis pigmentosa due to a mutation in the MYO7A gene. The study will also assess the initial efficacy following AAVB-081 administration.
• DelveInsight’s Retinitis Pigmentosa Pipeline report depicts a robust space with 40+ active players working to develop 40+ pipeline therapies for Retinitis Pigmentosa treatment.
• The leading Retinitis Pigmentosa Companies such as MeiraGTx, Aldeyra Therapeutics, Inc., SparingVision, Frontera Therapeutics, ProQR Therapeutics, Bionic Sight, RetroSense Therapeutics, Dompe Farmaceutici, Endogena Therapeutics, Nacuity Pharmaceuticals, Kiora Pharmaceuticals, jCyte, Ocugen, Neurotech USA, Nanoscope Therapeutics and others.
• Promising Retinitis Pigmentosa Pipeline Therapies such as Setmelanotide, PBI-4050, OpCT-001, QR-421a, NPI-001, QR-1123, AAVB-081 and others.

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Retinitis Pigmentosa Emerging Drugs Profile

• Botaretigene sparoparvovec : MeiraGTx

Botaretigene sparoparvovec (bota-vec) is designed to treat the most common form of X-linked retinitis pigmentosa (XLRP) caused by mutations in the eye-specific form of the RPGR gene called RPGR open reading frame 15 (RPGR ORF15). Both rods and cones photoreceptors require RPGR ORF15 to function. AAV-RPGR has received Fast Track and Orphan Drug designations from the FDA, as well as PRIME, ATMP and Orphan Medicinal Product designations from the EMA. Currently, the drug is in Phase III stage of its development for the treatment of renitis pigmentosa.

• ADX-2191: Aldeyra Therapeutics, Inc.

ADX-2191 is a novel drug candidate being developed by Aldeyra Therapeutics, Inc. The drug candidate is tetrahydrofolate dehydrogenase inhibitors and thymidylate synthase inhibitors. ADX-2191 received orphan drug designation in 2018. Currently, the drug is in Phase II stage of its development for the treatment of renitis pigmentosa.

• SPVN-06 : SparingVision

SPVN06, is a novel product candidate that acts independently of the causative gene mutation. SPVN06 counteracts the degeneration of cone photoreceptors by restoring RdCVF, a neurotrophic factor naturally produced by functioning rods in the retina; and by promoting RdCVFL, a potent antioxidant which protects cones against oxidative stress. The DNA of the two distinct isoforms (RdCVF and RdCVFL) of the NXNL1 gene are supplied via an Adeno-associated virus (AAV), a viral vector classically used in gene therapy. SPVN06 is delivered via the subretinal route to the cones and retinal pigmented epithelium (RPE). Upon delivery of SPVN06, the transgenes are mainly expressed in the RPE cells for RdCVF and in the cones for RdCVFL. SPVN06 is expected to provide a long-lasting neuroprotective effect to prevent the progression of RP in patients with moderate to severe pathology. Currently, the drug is in Phase I/II stage of its development for the treatment of renitis pigmentosa.

• FT-002: Frontera Therapeutics

FT-002 is a recombinant adeno-associated virus gene therapy drug intended to treat patients with X-linked retinitis pigmentosa (XLRP) caused by RPGR gene mutation. There is no treatment for this disease globally. FT-002 is an intraocular injection of recombinant AAV virus carrying the gene that expresses active functional proteins and repairs damaged retinal cell structure and function. One injection can effectively delay disease progression or restore the patient’s visual function. FT-002 is the first AAV gene therapy drug to be tested in XLRP patients in China and is also a potential first-in-class drug. Currently, the drug is in Phase I/II stage of its development for the treatment of Renitis Pigmentosa.

The Retinitis Pigmentosa Pipeline Report Provides Insights into

• The report provides detailed insights about companies that are developing therapies for the treatment of Retinitis Pigmentosa 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 Retinitis Pigmentosa Treatment.
• Retinitis Pigmentosa Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Retinitis Pigmentosa 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 Retinitis Pigmentosa market

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Retinitis Pigmentosa Companies

MeiraGTx, Aldeyra Therapeutics, Inc., SparingVision, Frontera Therapeutics, ProQR Therapeutics, Bionic Sight, RetroSense Therapeutics, Dompe Farmaceutici, Endogena Therapeutics, Nacuity Pharmaceuticals, Kiora Pharmaceuticals, jCyte, Ocugen, Neurotech USA, Nanoscope Therapeutics and others.

Retinitis pigmentosa pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Retinitis Pigmentosa Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

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Scope of the Retinitis Pigmentosa Pipeline Report

• Coverage- Global
• Retinitis Pigmentosa Companies- MeiraGTx, Aldeyra Therapeutics, Inc., SparingVision, Frontera Therapeutics, ProQR Therapeutics, Bionic Sight, RetroSense Therapeutics, Dompe Farmaceutici, Endogena Therapeutics, Nacuity Pharmaceuticals, Kiora Pharmaceuticals, jCyte, Ocugen, Neurotech USA, Nanoscope Therapeutics and others.
• Retinitis Pigmentosa Pipeline Therapies- Setmelanotide, PBI-4050, OpCT-001, QR-421a, NPI-001, QR-1123, AAVB-081 and others.
• Retinitis Pigmentosa Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Retinitis Pigmentosa Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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Table of Content

1. Introduction
2. Executive Summary
3. Retinitis Pigmentosa: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Retinitis Pigmentosa– DelveInsight’s Analytical Perspective
7. Late Stage Products (Phase III)
8. Botaretigene sparoparvovec : MeiraGTx
9. Drug profiles in the detailed report…..
10. Mid Stage Products (Phase II)
11. ADX-2191: Aldeyra Therapeutics, Inc.
12. Drug profiles in the detailed report…..
13. Early Stage Products (Phase I/II)
14. SPVN-06 : SparingVision
15. Drug profiles in the detailed report…..
16. Preclinical and Discovery Stage Products
17. Drug Name: Company Name
18. Drug profiles in the detailed report…..
19. Inactive Products
20. Retinitis Pigmentosa Key Companies
21. Retinitis Pigmentosa Key Products
22. Retinitis Pigmentosa- Unmet Needs
23. Retinitis Pigmentosa- Market Drivers and Barriers
24. Retinitis Pigmentosa- Future Perspectives and Conclusion
25. Retinitis Pigmentosa Analyst Views
26. Retinitis Pigmentosa Key Companies
27. Appendix

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