Retinitis pigmentosa Market: Epidemiology, Therapies, Companies, DelveInsight | Johnson & Johnson Innovative Medicine, MeiraGTx, Beacon Therapeutics, Nanoscope Therapeutics, Gensight Biologics, 4D Molecular Therapeutics, Coave Therapeutics, Ocugen, Bionic

Retinitis pigmentosa therapies, such as LUXTURNA, Botaretigene sparoparvovec, MCO-010, and others, are expected to boost the Retinitis pigmentosa Market in the upcoming years.

DelveInsight has launched a new report on "Retinitis pigmentosa - Market Insights, Epidemiology, and Market Forecast-2034" that delivers an in-depth understanding of the Retinitis pigmentosa, historical and forecasted epidemiology as well as the Retinitis pigmentosa market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

Discover about the Retinitis pigmentosa market report @ https://www.delveinsight.com/report-store/retinitis-pigmentosa-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Some of the key facts of the Retinitis pigmentosa Market Report:

In 2023, the U.S. market for retinitis pigmentosa treatments was valued at approximately USD 260 million, with projections indicating growth driven by the introduction of new and innovative therapies.
In 2023, the total market size for Retinitis Pigmentosa (RP) treatment across the seven major markets (7MM) was approximately USD 500 million, with substantial growth anticipated from 2024 to 2034. RP is the most prevalent form of inherited retinal disease (IRD), impacting over 1.5 million individuals globally. Despite its relatively widespread presence, it is still categorized as a rare disease.
According to DelveInsight, there were around 274,000 diagnosed RP cases in the 7MM in 2023, with the number expected to rise over the forecast period. Roughly 65% of RP cases are nonsyndromic. Among these, autosomal recessive forms account for about 10-15%, autosomal dominant for 20-25%, and X-linked RP (XLRP) is the rarest, marked by early onset and rapid vision deterioration.
Genetic studies reveal that in nonsyndromic RP and Usher syndrome, the RHO and RP1 genes are the most commonly mutated in autosomal dominant RP (ADRP). In XLRP cases, about 75% involve RPGR mutations, with RP2 and OFD1 mutations also contributing.
The introduction of Next-Generation Sequencing (NGS) has revolutionized RP diagnostics by enabling the identification of genetic mutations that were previously undetectable.
Currently, patients with RP mostly depend on off-label treatments that do not address the underlying genetic cause-especially those without the RPE65 mutation. These patients are often managed with supportive therapies like vitamin supplementation, light protection, and visual aids. At present, LUXTURNA remains the only approved treatment specifically targeting RP, limited to patients with confirmed RPE65 mutations.
Optogenetics is emerging as a groundbreaking gene therapy solution that bypasses gene-specific limitations and shows promise even in advanced disease stages characterized by significant photoreceptor loss.
Several companies are advancing optogenetic gene therapies into clinical trials for RP. These include GenSight Biologics (GS030), Bionic Sight (BS01), and Nanoscope Therapeutics (MCO-010), among others.
In 2023, the United States recorded approximately 113,000 prevalent cases of Retinitis Pigmentosa. Among these, nonsyndromic retinitis pigmentosa accounted for the majority, representing around 65% of all cases, making it more common than the syndromic form.
The RP treatment pipeline is diverse, with a range of drug candidates addressing various disease stages. One novel candidate, Ultevursen, is an RNA-based oligonucleotide that targets exon 13 of the USH2A gene, representing a unique approach in RP therapy development.
In January 2025, Beacon Therapeutics announced that the FDA had granted Regenerative Medicine Advanced Therapy (RMAT) designation to laru-zova (laruparetigene zovaparvovec) for the treatment of X-linked retinitis pigmentosa (XLRP), marking a significant step forward in its efforts to restore vision in patients with inherited retinal disorders.
In January 2025, PYC Therapeutics revealed that its VP-001 program received Rare Pediatric Disease designation from the U.S. FDA. This program targets vision loss associated with PRPF31 mutations, reinforcing PYC's position in the market and offering renewed hope to patients affected by Retinitis Pigmentosa type 11.
In January 2025, ViGeneron GmbH announced two key developments for VG901, its gene therapy targeting CNGA1-related retinitis pigmentosa. The FDA awarded it Rare Pediatric Disease designation, and the Data Safety Monitoring Board approved advancing to dose escalation in the ongoing Phase 1b clinical trial.
Key Retinitis pigmentosa companies such as Johnson & Johnson Innovative Medicine, MeiraGTx, Beacon Therapeutics, Nanoscope Therapeutics, Gensight Biologics, 4D Molecular Therapeutics, Coave Therapeutics, Ocugen, Bionic Sight, jCyte, Endogena Therapeutics, ProQR Therapeutics, and Aldeyra Therapeutics, and others are evaluating new drugs for Retinitis pigmentosa to improve the treatment landscape.
Promising Retinitis pigmentosa therapies include LUXTURNA, Botaretigene sparoparvovec, MCO-010, and others.

Retinitis pigmentosa Overview

Retinitis pigmentosa (RP) encompasses a group of inherited retinal conditions that result in progressive retinal degeneration and eventual blindness. It is marked by the gradual deterioration of both rod and cone photoreceptors in both eyes. This typically begins with night blindness and narrowing of the visual field, eventually leading to a decline in central vision under daylight conditions due to cone photoreceptor loss. The condition is primarily genetic, with mutations in over 44 genes identified as contributing factors.

RP can follow autosomal recessive, autosomal dominant, or X-linked inheritance patterns. Notably, around half of all cases involve genetic mutations that remain unidentified. Clinically, RP is classified into syndromic, non-syndromic, and systemic forms, while its onset is categorized as either early-onset or late-onset. The disease presents a range of clinical features, such as characteristic changes in the retina's appearance, dust-like particles in the vitreous body, white deposits deep in the retina, exudative vasculopathy similar to Coats disease, and localized forms like sectoral RP.

Retinitis pigmentosa Market Outlook

As research uncovers more targetable genetic mutations and new therapies are developed, both patients and ophthalmologists will gain access to an expanding range of treatment options for retinitis pigmentosa. However, with the increasing pace of drug approvals, it is essential to ensure that each therapy is backed by robust clinical data and is used appropriately across various treatment phases, such as adjuvant, consolidation, first-line, or later-line therapy.

Currently, LUXTURNA (voretigene neparvovec) remains the only approved treatment for retinitis pigmentosa, and its use is limited to a small subset of patients carrying the RPE65 mutation. This gene therapy is approved for both adult and pediatric patients experiencing vision loss due to inherited retinal disease (IRD). Companies targeting therapies for both age groups stand to access a broader patient base. Notable players in this space include Beacon Therapeutics (AGTC-501), MeiraGTx in collaboration with Janssen Research & Development (Botaretigene sparoparvovec), 4D Molecular Therapeutics (4D-125), ProQR Therapeutics in partnership with Laboratoires Théa (Ultevursen), Coave Therapeutics (CTx-PDE6b), and Ocugen (OCU400).

For patients without the RPE65 mutation, no standard treatment currently exists. These individuals largely depend on supportive care measures such as vitamin supplementation, light protection, and visual aids. This gap highlights a potential market opportunity for enhanced supportive therapies tailored to retinitis pigmentosa management.

Discover how the Retinitis pigmentosa market is rising in the coming years @ https://www.delveinsight.com/sample-request/retinitis-pigmentosa-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Retinitis Pigmentosa Marketed Drugs

LUXTURNA: Sparks Therapeutics (Roche)/Novartis

Retinitis pigmentosa Emerging Drugs
Botaretigene sparoparvovec: Johnson & Johnson Innovative Medicine /MeiraGTx
MCO-010: Nanoscope Therapeutics

Scope of the Retinitis pigmentosa Market Report
Study Period: 2020-2034
Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
Key Retinitis pigmentosa Companies: Johnson & Johnson Innovative Medicine, MeiraGTx, Beacon Therapeutics, Nanoscope Therapeutics, Gensight Biologics, 4D Molecular Therapeutics, Coave Therapeutics, Ocugen, Bionic Sight, jCyte, Endogena Therapeutics, ProQR Therapeutics, and Aldeyra Therapeutics, and others
Key Retinitis pigmentosa Therapies: LUXTURNA, Botaretigene sparoparvovec, MCO-010, and others
Retinitis pigmentosa Therapeutic Assessment: Retinitis pigmentosa current marketed and Retinitis pigmentosa emerging therapies
Retinitis pigmentosa Market Dynamics: Retinitis pigmentosa market drivers and Retinitis pigmentosa market barriers
Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies
Retinitis pigmentosa Unmet Needs, KOL's views, Analyst's views, Retinitis pigmentosa Market Access and Reimbursement

To know what's more in our Retinitis pigmentosa report, visit https://www.delveinsight.com/report-store/retinitis-pigmentosa-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Key benefits of the Retinitis pigmentosa Market Report:
Retinitis pigmentosa market report covers a descriptive overview and comprehensive insight of the Retinitis pigmentosa Epidemiology and Retinitis pigmentosa market in the 7MM (the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan).
The Retinitis pigmentosa market report provides insights into the current and emerging therapies.
The Retinitis pigmentosa market report provides a global historical and forecasted market covering drug outreach in 7MM.
The Retinitis pigmentosa market report offers an edge that will help in developing business strategies by understanding trends shaping and driving the Retinitis pigmentosa market.

Got queries? Click here to know more about the Retinitis pigmentosa market Landscape https://www.delveinsight.com/sample-request/retinitis-pigmentosa-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Table of Contents

1. Report Introduction
2. Executive Summary
3. SWOT analysis
4. Retinitis pigmentosa Patient Share (%) Overview at a Glance
5. Retinitis pigmentosa Market Overview at a Glance
6. Retinitis pigmentosa Disease Background and Overview
7. Retinitis pigmentosa Epidemiology and Patient Population
8. Country-Specific Patient Population of Retinitis pigmentosa
9. Retinitis pigmentosa Current Treatment and Medical Practices
10. Unmet Needs
11. Retinitis pigmentosa Emerging Therapies
12. Retinitis pigmentosa Market Outlook
13. Country-Wise Retinitis pigmentosa Market Analysis (2020-2034)
14. Market Access and Reimbursement of Therapies
15. Market drivers
16. Market barriers
17. Appendix
18. Retinitis pigmentosa Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight

Click here to read more about Retinitis pigmentosa Market Outlook 2034 https://www.delveinsight.com/report-store/retinitis-pigmentosa-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Related Reports:

Retinitis pigmentosa Pipeline Insights, DelveInsight

"Retinitis pigmentosa Pipeline Insight, 2024" report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the Retinitis pigmentosa market. A detailed picture of the Retinitis pigmentosa pipeline landscape is provided, which includes the disease overview and Retinitis pigmentosa treatment guidelines.

Contact Info:

Kritika Rehani, Assistant Manager, Marketing & Branding
krehani@delveinsight.com
info@delveinsight.com
www.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.

This release was published on openPR.