Choroideremia Pipeline Insight 2025: Advancing Gene Therapies for an Inherited Retinal Degeneration | DelveInsight

DelveInsight's "Choroideremia - Pipeline Insight, 2025" offers an in-depth analysis of the emerging therapeutic landscape for Choroideremia, a rare X-linked retinal degenerative disorder caused by mutations in the CHM gene. This progressive condition predominantly affects males and leads to the gradual loss of vision due to degeneration of the choroid, retinal pigment epithelium (RPE), and photoreceptors, often resulting in blindness by middle age.

Given the monogenic nature of the disease and its confined ocular pathology, Choroideremia has become a promising target for gene therapy, with several candidates advancing through clinical development. These therapies aim to deliver a functional copy of the CHM gene via adeno-associated virus (AAV) vectors directly into the retina to preserve or restore vision.

One of the most advanced approaches includes subretinal delivery of AAV2-based vectors, which have shown encouraging results in early-phase trials in terms of safety, durability of gene expression, and potential functional benefit. Long-term follow-up studies are underway to assess sustained efficacy, slowing of disease progression, and optimal dosing strategies.

In addition to gene replacement, pipeline research is also exploring gene editing, RNA-based therapies, and optogenetics as potential future avenues for therapy, particularly for patients in advanced disease stages where traditional gene supplementation may have limited benefit.

With orphan drug designations, fast-track status, and regulatory support from agencies like the FDA and EMA, the Choroideremia pipeline is receiving significant investment and collaborative attention. Advances in retinal imaging and functional assessment tools are also enhancing trial design and endpoint evaluation, accelerating progress in this ultra-rare indication.

As we move through 2025, the momentum in Choroideremia drug development continues to grow, positioning it as a potential model for gene therapy in inherited retinal diseases and paving the way for transformative, sight-preserving treatments.

Interested in learning more about the current treatment landscape and the key drivers shaping the choroideremia pipeline? Click here: https://www.delveinsight.com/report-store/choroideremia-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Key Takeaways from the Choroideremia Pipeline Report
• DelveInsight's choroideremia pipeline analysis depicts a strong space with 5+ active players working to develop 5+ pipeline drugs for choroideremia treatment.
• The leading choroideremia companies include Spark Therapeutics, Molecular Therapeutics, Curative Biotechnology, Ray Therapeutics, and others are evaluating their lead assets to improve the choroideremia treatment landscape.
• Key choroideremia pipeline therapies in various stages of development include AAV2-hCHM, 4D-110, Metformin, RAY-001, and others.
• In June 2025, the Choroideremia Research Foundation (CRF) awarded six global grants supporting projects in gene editing (CRISPR), AI-based molecular glue discovery, natural history registries, and long-term follow-up of gene therapy recipients-underlining expanded research momentum in 2025.
• In August 2024, the ENVISION (RTx-015-CP-101) trial led by Ray Therapeutics commenced as a Phase 1, open-label, dose-escalation study assessing intravitreal RTx‐015 (optogenetic gene therapy) for patients with choroideremia or retinitis pigmentosa. It is expected to be completed by October 1, 2030.

Request a sample and discover the recent breakthroughs happening in the choroideremia pipeline landscape at https://www.delveinsight.com/report-store/choroideremia-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Choroideremia Overview
Choroideremia is a rare, inherited retinal degenerative disorder that primarily affects males. It is caused by mutations in the CHM gene, leading to progressive loss of the retinal pigment epithelium, photoreceptors, and choroid. Symptoms typically begin with night blindness in childhood, followed by peripheral vision loss and eventual central vision deterioration, potentially leading to blindness in adulthood.
Currently, there is no approved cure for choroideremia. However, gene therapies-such as AAV-based treatments aiming to replace the faulty CHM gene- are under investigation and show promise in slowing disease progression and preserving vision.

Find out more about choroideremia medication at https://www.delveinsight.com/report-store/choroideremia-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Choroideremia Treatment Analysis: Drug Profile
AAV2-hCHM: Spark Therapeutics
Spark Therapeutics is conducting an open-label, dose-escalation Phase I/II trial to evaluate the safety and early efficacy of SPK-7001, an investigational gene therapy delivered via subretinal injection. The therapy uses an adeno-associated viral (AAV) vector developed in collaboration with Spark's research team.

Learn more about the novel and emerging choroideremia pipeline therapies at https://www.delveinsight.com/report-store/choroideremia-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Choroideremia Therapeutics Assessment
By Product Type
• Mono
• Combination
• Mono/Combination.

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

By Route of Administration
• Oral
• Intravenous
• Subcutaneous

By Molecule Type
• Small molecule
• Cell Therapy
• Peptides
• Polymer
• Small molecule
• Gene therapy

Scope of the Choroideremia Pipeline Report
• Coverage: Global
• Key Choroideremia Companies: Spark Therapeutics, Molecular Therapeutics, Curative Biotechnology, Ray Therapeutics, and others.
• Key Choroideremia Pipeline Therapies: AAV2-hCHM, 4D-110, Metformin, RAY-001, and others.

To dive deep into rich insights for drugs used for choroideremia treatment, visit: https://www.delveinsight.com/report-store/choroideremia-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Table of Contents
1. Introduction
2. Executive Summary
3. Choroideremia Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Choroideremia Pipeline Therapeutics
6. Choroideremia Pipeline: Late-Stage Products (Phase III)
7. Choroideremia Pipeline: Mid-Stage Products (Phase II)
8. Choroideremia Pipeline: Early Stage Products (Phase I)
9. Therapeutic Assessment
10. Inactive Products
11. Company-University Collaborations (Licensing/Partnering) Analysis
12. Key Companies
13. Key Products
14. Unmet Needs
15. Market Drivers and Barriers
16. Future Perspectives and Conclusion
17. Analyst Views
18. Appendix

Contact Us:
Jatin Vimal
jvimal@delveinsight.com
+14699457679
Healthcare Consulting
https://www.delveinsight.com/consulting-services

About DelveInsight
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This release was published on openPR.