X-linked retinitis pigmentosa Pipeline Analysis and Clinical Trials Assessment, 2025 by DelveInsight | Biogen, 4 D Molecular therapeutics, Applied Genetics Technology Corporation

X-linked retinitis pigmentosa Pipeline constitutes 5+ key companies continuously working towards developing 5+ X-linked retinitis pigmentosa treatment therapies, analyzes DelveInsight.

X-linked retinitis pigmentosa Overview:

X-linked Retinitis Pigmentosa is a rare hereditary genetic disorder caused by a mutation in the X chromosome. It leads to the gradual degeneration of photoreceptor cells in the retina, resulting in progressive loss of peripheral vision. The condition affects approximately 1 in 4,000 people globally. Because it is a recessive disorder, males are more commonly affected, while females, with two X chromosomes, typically act as carriers. Early symptoms include night blindness and a progressive reduction in the visual field.

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"X-linked retinitis pigmentosa Pipeline Insight 2025" report by DelveInsight provides a comprehensive analysis of the ongoing clinical development activities and growth prospects across the X-linked retinitis pigmentosa Therapeutics Market.

Key Takeaways from the X-linked retinitis pigmentosa Pipeline Report

DelveInsight's X-linked retinitis pigmentosa pipeline report depicts a robust space with 5+ active players working to develop 5+ pipeline therapies for X-linked retinitis pigmentosa treatment.
In January 2025, Beacon Therapeutics received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for Laru-Zova, a gene therapy targeting XLRP. This designation facilitates expedited development and review processes, recognizing the therapy's potential to address unmet medical needs. Laru-Zova also holds Fast Track status in the U.S., PRIME designation in the EU, and ILAP designation in the UK .​
In April 2025, Atsena Therapeutics announced that the FDA granted RMAT designation to ATSN-201, a gene therapy for X-linked retinoschisis, a related retinal disorder. This designation aims to expedite the development and review of therapies addressing serious or life-threatening conditions .​
Bionic Sight's BS01 gene therapy received RMAT designation from the FDA in February 2025. Preliminary results from Phase 1/2 clinical trials have shown promise in patients with advanced retinitis pigmentosa, indicating potential benefits in vision restoration .​
Key X-linked retinitis pigmentosa companies such as Biogen, 4 D Molecular therapeutics, Applied Genetics Technology Corporation, and others are evaluating new drugs for X-linked retinitis pigmentosa to improve the treatment landscape.
Promising X-linked retinitis pigmentosa therapies include BIIB 112 (NSR-RPGR), 4D-125, and others.

X-linked retinitis pigmentosa Pipeline Analysis
The X-linked retinitis pigmentosa pipeline insights report 2025, provides insights into:
Provides comprehensive insights into key companies developing therapies in the X-linked retinitis pigmentosa Market.

Categorizes X-linked retinitis pigmentosa 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 X-linked retinitis pigmentosa drugs under development based on:

Stage of development

X-linked retinitis pigmentosa Route of administration

Target receptor

Monotherapy vs. combination therapy

X-linked retinitis pigmentosa Mechanism of action

Molecular type

Offers detailed analysis of:

Company-to-company and company-academia collaborations

X-linked retinitis pigmentosa Licensing agreements

Funding and investment activities supporting future X-linked retinitis pigmentosa market advancement.

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X-linked retinitis pigmentosa Emerging Drugs

BIIB 112 (NSR-RPGR): Biogen

Biogen's primary drug candidate, BIIB 112 (NSR-RPGR), is a gene therapy that uses a standard AAV8 vector to deliver a codon-optimized RPGR gene. Currently in Phase II/III trials, it aims to treat rare X-linked Retinitis Pigmentosa and has received orphan drug designation. The ongoing trial is designed to assess the drug's tolerability and its ability to stabilize and improve central vision.

4D-125: 4 D Molecular Therapeutics

4D Molecular Therapeutics' leading drug, 4D-125, is currently under evaluation for safety and maximum tolerated dose in the treatment of X-linked Retinitis Pigmentosa. 4D-125 is an AAV-based gene therapy with an optimized vector designed to deliver a functional RPGR gene to retinal photoreceptors.

X-linked retinitis pigmentosa Companies

Around five or more key companies are working on therapies for X-linked Retinitis Pigmentosa. Among them, Biogen and others have drug candidates in the mid to advanced stages, specifically Phase II/III.

DelveInsight's report covers around 5+ products under different phases of X-linked retinitis pigmentosa clinical trials like

X-linked retinitis pigmentosa Late stage Therapies (Phase III)
X-linked retinitis pigmentosa Mid-stage Therapies (Phase II)
X-linked retinitis pigmentosa Early-stage Therapies (Phase I)
X-linked retinitis pigmentosa Pre-clinical and X-linked retinitis pigmentosa Discovery stage Therapies
X-linked retinitis pigmentosa Discontinued & Inactive Therapies

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

Intravenous
Subcutaneous
Oral
Intramuscular

X-linked retinitis pigmentosa Products have been categorized under various Molecule types such as

Monoclonal antibody
Small molecule
Peptide

Download Sample Pages to Get an in-depth Assessment of the Emerging X-linked retinitis pigmentosa Therapies and Key X-linked retinitis pigmentosa Companies: X-linked retinitis pigmentosa Clinical Trials and recent advancements https://www.delveinsight.com/report-store/x-linked-retinitis-pigmentosa-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

X-linked retinitis pigmentosa Pipeline Therapeutic Assessment
• X-linked retinitis pigmentosa Assessment by Product Type
• X-linked retinitis pigmentosa By Stage
• X-linked retinitis pigmentosa Assessment by Route of Administration
• X-linked retinitis pigmentosa Assessment by Molecule Type

Download X-linked retinitis pigmentosa Sample report to know in detail about the X-linked retinitis pigmentosa treatment market @ X-linked retinitis pigmentosa Therapeutic Assessment https://www.delveinsight.com/sample-request/x-linked-retinitis-pigmentosa-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Table of Content

1. Report Introduction
2. Executive Summary
3. X-linked retinitis pigmentosa Current Treatment Patterns
4. X-linked retinitis pigmentosa - DelveInsight's Analytical Perspective
5. Therapeutic Assessment
6. X-linked retinitis pigmentosa Late-Stage Products (Phase-III)
7. X-linked retinitis pigmentosa 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. X-linked retinitis pigmentosa Discontinued Products
13. X-linked retinitis pigmentosa Product Profiles
14. X-linked retinitis pigmentosa Key Companies
15. X-linked retinitis pigmentosa Key Products
16. Dormant and Discontinued Products
17. X-linked retinitis pigmentosa Unmet Needs
18. X-linked retinitis pigmentosa Future Perspectives
19. X-linked retinitis pigmentosa Analyst Review
20. Appendix
21. Report Methodology

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Contact Info:
Kritika Rehani
krehani@delveinsight.com
info@delveinsight.com
+14699457679

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.

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