X-Linked Retinitis Pigmentosa Pipeline Analysis & Clinical Trials, 2025, Delveinsight Biogen, 4 D Molecular Therapeutics, Applied Genetics Technology Corporation

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"X-linked retinitis pigmentosa Pipeline Analysis"DelveInsight's,“X-linked Retinitis Pigmentosa – Pipeline Insight, 2025,” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in X-linked Retinitis Pigmentosa pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

DelveInsight's X-linked retinitis pigmentosa Clinical Trials Analysis Report provides deep insights to support strategic decision-making for X-linked retinitis pigmentosa pipeline expansion, competitive positioning, and partnership identification in the rare neurodegenerative disease space.

X-linked retinitis pigmentosa Overview:

X-linked Retinitis Pigmentosa (XLRP) is a rare inherited genetic disorder caused by mutations on the X chromosome. It results in the gradual degeneration of retinal photoreceptor cells, leading to progressive loss of peripheral vision. The condition affects roughly 1 in 4,000 people worldwide. Being an X-linked recessive disorder, it primarily affects males, while females usually serve as carriers. Early signs include night blindness and a steadily narrowing 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 (XLRP) Pipeline Report highlights a dynamic space with over 5 companies developing more than 5 therapeutic candidates for XLRP.

• In January 2025, Beacon Therapeutics received FDA RMAT designation for Laru-Zova, a gene therapy targeting XLRP. This status accelerates development and review processes, reflecting the therapy's potential to address unmet medical needs. Laru-Zova also holds Fast Track designation 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, aiming to speed the development of treatments for serious conditions.

• Bionic Sight's BS01 gene therapy also received RMAT designation in February 2025. Early Phase 1/2 trial results suggest potential benefits in restoring vision for patients with advanced retinitis pigmentosa.

• Key players in the XLRP space include Biogen, 4D Molecular Therapeutics, Applied Genetics Technology Corporation, and others, all working to advance innovative treatments. Promising therapies in development include BIIB 112 (NSR-RPGR), 4D-125, and additional pipeline candidates aiming to transform the treatment landscape.

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 lead candidate, BIIB 112 (NSR-RPGR), is a gene therapy utilizing a standard AAV8 vector to deliver a codon-optimized RPGR gene. Currently in Phase II/III trials, it is being developed to treat rare X-linked Retinitis Pigmentosa and has received orphan drug designation. The ongoing study is evaluating the therapy's safety, tolerability, and its potential to stabilize and improve central vision.

• 4D-125: 4 D Molecular Therapeutics

4D Molecular Therapeutics' lead candidate, 4D-125, is being evaluated for safety and maximum tolerated dose in treating X-linked Retinitis Pigmentosa. This AAV-based gene therapy employs an optimized vector to deliver a functional RPGR gene directly to retinal photoreceptors.

X-linked retinitis pigmentosa Companies

More than five key companies are developing therapies for X-linked Retinitis Pigmentosa, with Biogen and others advancing candidates into mid-to-late-stage clinical trials (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

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

X-linked retinitis pigmentosa Report Section Includes:

• X-linked retinitis pigmentosa competitive landscape clinical trials

• X-linked retinitis pigmentosa emerging drugs analysis

• X-linked retinitis pigmentosa investigational drugs report

• X-linked retinitis pigmentosa clinical trial trends analysis

• X-linked retinitis pigmentosa pipeline benchmarking report

• X-linked retinitis pigmentosa drug pipeline competitive intelligence

• X-linked retinitis pigmentosa competitive landscape

Major trends shaping the clinical trial and competitive landscape for X-linked retinitis pigmentosa:

• Focus on Gene Therapies: Rapid growth in AAV-based and other viral vector gene therapies targeting RPGR mutations, aiming to halt or reverse retinal degeneration.

• Expansion of Clinical Trials: Increasing number of early- and mid-stage trials, often globally conducted to ensure diverse patient recruitment and robust data.

• Regulatory Support: Orphan Drug, Fast Track, and RMAT designations accelerate development timelines and regulatory review for rare retinal disorders.

• Emerging Biomarkers and Imaging Techniques: Use of optical coherence tomography (OCT), electroretinography, and genetic biomarkers for patient stratification and precise monitoring of disease progression.

• Strategic Collaborations: Partnerships between biotech firms, pharmaceutical companies, and academic institutions to pool expertise, technology, and resources.

• Innovative Delivery Approaches: Focus on subretinal, intravitreal, and optimized vector delivery systems to enhance gene transfer efficiency and safety.

• Patient-Centric Outcomes: Incorporation of visual function metrics, quality-of-life assessments, and patient-reported outcomes in clinical trial endpoints.

• Competitive Differentiation: Companies differentiating pipelines based on gene therapy platform, delivery route, stage of development, and mechanism of action.

• Integration of Real-World Evidence: Leveraging real-world patient data to support regulatory approval, market access, and long-term safety evaluation.

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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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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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