The retinitis pigmentosa market, estimated to be valued at USD 542.8 Million in 2025, is projected to exhibit a CAGR of 6.4%, reaching USD 838.0 Million by 2032. This growth is primarily driven by advancements in gene and cell-based therapies, as well as increased investments in ophthalmic research. Rising awareness about inherited retinal diseases and improved access to genetic testing are enhancing early diagnosis and patient eligibility for emerging treatments. Additionally, the expansion of clinical trials, regulatory support for orphan drug development, and strategic collaborations among biotech firms are accelerating innovation in this space. The growing burden of visual impairment and strong demand for targeted, disease-modifying treatments are expected to further support the market growth over the forecast period.

Market Takeaways

• By Inheritance Type, the Autosomal Dominant segment is projected to dominate the market with a 48.7% share in 2025, owing to its higher prevalence rate and more frequent diagnosis due to distinct inheritance patterns and family history tracking.
• By Disease Type, the Syndromic Retinitis Pigmentosa segment is expected to account for the largest share at 37.2% in 2025, driven by its association with systemic conditions such as Usher and Bardet-Biedl syndromes, prompting early and more frequent clinical attention.
• By Age of Onset, Early-Onset Retinitis Pigmentosa is anticipated to lead the market, as earlier diagnosis correlates with greater engagement in long-term disease monitoring, therapeutic intervention, and clinical trial participation.
• By Region, North America is expected to dominate the global market in 2025, supported by advanced healthcare infrastructure, a strong presence of leading biotechnology companies, and increased funding for rare retinal disease research and gene therapy development.

Retinitis Pigmentosa Market Report Coverage

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Report Coverage	Details
Market Revenue in 2025	USD 542.8 Million
Estimated Value by 2032	USD 838.0 Million
Growth Rate	Poised to exhibit a CAGR of 6.4%
Historical Data	2020-2024
Forecast Period	2025–2032
Forecast Units	Value (USD Million)
Report Coverage	Revenue Forecast, Competitive Landscape, Growth Factors, and Trends
Segments Covered	By Inheritance Type, Disease Type, and Age of Onset
Geographies Covered	North America (U.S. and Canada), Latin America (Brazil, Argentina, Mexico, and Rest of Latin America), Europe (Italy, Spain, U.K., Germany, France, Russia, and Rest of Europe), Asia Pacific (China, India, Japan, Australia, South Korea, ASEAN, and Rest of Asia Pacific), Middle East (GCC Countries, Israel, and Rest of Middle East), and Africa (South Africa, North Africa, and Central Africa)
Growth Drivers	Increasing prevalence of retinitis pigmentosa globally Advances in gene therapy and optogenetics
Trends	Gene-targeted therapies reshaping the inherited retinal disease landscape Growing integration of artificial intelligence in early diagnosis and disease monitoring
Opportunities	Development of novel gene therapies for a broader patient population Expansion of diagnostic techniques like Next-Generation Sequencing (NGS)
Restraints & Challenges	Limited approved therapies, with most patients relying on off-label treatments High costs associated with gene therapy treatments

Market Dynamics

The Retinitis Pigmentosa market is witnessing progressive growth, propelled by increasing disease prevalence, greater diagnostic awareness, and a rising number of clinical trials targeting inherited retinal disorders. Retinitis Pigmentosa, a group of rare genetic disorders causing retinal degeneration, is attracting attention due to its significant impact on vision and quality of life, especially among younger populations affected by early-onset forms.

The market is further driven by advancements in genetic testing and precision medicine, enabling earlier diagnosis and more personalized treatment strategies. The growing adoption of next-generation sequencing (NGS) and whole exome/genome sequencing has made it possible to identify causative mutations, opening doors to gene-targeted therapies and improving clinical decision-making.

In January 2022, 4D Molecular Therapeutics (NASDAQ: FDMT), a clinical-stage gene therapy company harnessing the power of directed evolution for targeted gene therapies, announced that the U.S. Food and Drug Administration (FDA) had granted Fast Track Designation for 4D-125 for the treatment of patients with inherited retinal dystrophies due to defects in the RPGR gene, including X-linked Retinitis Pigmentosa (XLRP). 4D-125 is a targeted and evolved R100-based product candidate, which was invented at 4DMT for efficient intravitreal delivery, and is designed to deliver a functional copy of the RPGR gene to photoreceptors in the retina.

Additionally, the increasing engagement of biotechnology firms and academic institutions in rare ophthalmic disease research is resulting in robust R&D pipelines. Additionally, orphan drug designations and regulatory incentives from agencies such as the FDA and EMA are encouraging innovation in this underserved domain.

Market Trends

• Gene-Targeted Therapies Reshaping the Inherited Retinal Disease Landscape

The retinitis pigmentosa market is undergoing a paradigm shift driven by the rise of gene-targeted therapeutics, which aim to address the underlying genetic causes rather than just managing symptoms. Companies are actively developing mutation-specific therapies targeting genes such as RHO, RPGR, CNGA1, and CEP290.

For instance, in April 2023, Ocugen, Inc., a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, announced positive preliminary data among retinitis pigmentosa (RP) participants treated in the first two cohorts of the Phase 1/2 trial to assess the safety and efficacy of OCU400 for RP associated with NR2E3 and Rhodopsin (RHO) mutations and Leber Congenital Amaurosis (LCA) with mutation(s) in the CEP290 gene. These preliminary results provide support that OCU400, Ocugen’s first-in-class therapeutic approach utilizing a proprietary modifier gene therapy platform, has the potential to be a gene-agnostic therapeutic for RP and LCA patients with inherited retinal degeneration.

• Growing Integration of Artificial Intelligence in Early Diagnosis and Disease Monitoring

Artificial intelligence (AI) tools are increasingly being adopted in ophthalmology to aid in the early detection and progression monitoring of retinitis pigmentosa. AI-powered imaging analytics, such as deep learning models analyzing optical coherence tomography (OCT) and fundus autofluorescence images, are improving diagnostic accuracy and enabling longitudinal tracking of retinal changes.

In February 2023, a multicenter retrospective study led by Nagasato et al., published in JAMA Ophthalmology and covered by Ophthalmology Times, showed that a deep-learning model using ultra-widefield fundus autofluorescence (UW‑FAF) images can accurately estimate RP patients’ visual function—including mean visual field deviation, central sensitivity, and visual acuity—based solely on imaging data.

Market Opportunities

• Optogenetics and Vision Restoration Devices Gaining Momentum

The convergence of gene therapy and device-based interventions is giving rise to optogenetic therapies aimed at restoring visual function in late-stage retinitis pigmentosa patients. These approaches utilize light-sensitive proteins and wearable visual aids to reestablish light perception in patients with degenerated photoreceptors.

In February 2023, in the PIONEER Phase I/II trial, GS030 showed a favorable safety profile at 1 year in 9 late-stage RP patients, with up to 4‑year follow-up for some. Patients transitioned from near no light perception to the ability to locate and count objects using the wearable light‑stimulating goggles, particularly at the highest dose (5×10¹¹ vg). The DSMB recommended expansion at the highest dose.

• Patient Advocacy and Genomic Testing Programs Driving Diagnosis Rates

Global patient advocacy organizations are playing a central role in accelerating RP diagnosis through free or subsidized genetic testing initiatives and clinical trial awareness campaigns. Programs such as My Retina Tracker (Foundation Fighting Blindness) in the U.S. and Target 5000 in Ireland have significantly increased access to genetic diagnosis.

In May 2024, the Foundation announced a renewed partnership with PreventionGenetics (Exact Sciences) as its genetic testing lab partner and re‑engaged InformedDNA for genetic testing. nrolmentg. The program continues to offer no-cost genetic testing and nrolmentg to eligible IRD/RP patients across the U.S., aiming to remove financial barriers and enhance trial nrolment and research participation.

Analyst View

• The retinitis pigmentosa market is entering a pivotal innovation phase driven by breakthroughs in gene and cell therapy, AI-enabled diagnostics, and optogenetics. These advancements are shifting RP management from symptomatic treatment to precision medicine aimed at preserving or restoring visual function.
• Biotech firms and research institutions are accelerating efforts to develop gene-targeted therapies for RP subtypes with high unmet needs, such as X-linked RP and syndromic forms like Usher syndrome. Platforms like 4DMT’s 4D-125 and Ocugen’s OCU400 are exemplifying next-generation gene therapy pipelines, signaling a strong outlook for disease-modifying therapies over the forecast period.
• The integration of artificial intelligence in ophthalmology is proving transformative, particularly in early disease detection and progression monitoring. Deep learning algorithms are enabling accurate analysis of retinal imaging (e.g., OCT, FAF), reducing reliance on subjective assessments, and enhancing enrollment for clinical trials.
• Optogenetics and vision restoration devices are gaining traction as late-stage therapeutic options for RP patients with advanced photoreceptor loss. Clinical progress in trials such as GenSight’s PIONEER study highlights the potential of combining genetic light-sensitization with wearable visual prosthetics to regain partial visual capabilities.
• Patient advocacy initiatives like My Retina Tracker (U.S.) and Target 5000 (Ireland) are democratizing access to genomic testing and promoting clinical research participation. These efforts are expected to drive earlier diagnoses and increase the eligible patient population for novel gene-based treatments.
• Regionally, North America is poised to remain the dominant market through 2032, backed by robust biotech pipelines, active regulatory pathways (e.g., FDA’s Orphan Drug and Fast Track designations), and a proactive advocacy ecosystem. Meanwhile, Asia Pacific presents a compelling growth frontier, given its large undiagnosed IRD population and increasing investments in ophthalmic healthcare infrastructure.
• Overall, the retinitis pigmentosa market is transitioning toward a precision medicine paradigm marked by gene modulation, AI-supported care pathways, and patient-centered diagnostics. Companies aligning with these innovation vectors—while addressing affordability and access—are best positioned to capitalize on emerging commercial and therapeutic opportunities.

Key Developments

• In May 2025, Beacon Therapeutics Holdings Limited (‘Beacon Therapeutics’ or ‘the Company’), a leading clinical-stage biotechnology company with a mission to save and restore the vision of patients with blinding retinal diseases, announced 6-month interim safety and efficacy results from the Phase 2 DAWN trial of the company’s lead program, laru-zova (laruparetigene zovaparvovec), in patients with X-linked retinitis pigmentosa (XLRP) at the Association for Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting being held May 4-8, 2025 in Salt Lake City. Laru-zova was generally well-tolerated by all DAWN participants evaluated at 6 months or beyond, and initial data showed promising improvements in visual function across several key measures.
• In October 2022, The Janssen Pharmaceutical Companies of Johnson & Johnson announced the primary results from the Phase 1/2 study evaluating the investigational gene therapy botaretigene sparoparvovec (formerly AAV-RPGR) in patients with the inherited retinal disease X-linked retinitis pigmentosa (XLRP) associated with the retinitis pigmentosa GTPase regulator (RPGR) gene. Treatment with botaretigene sparoparvovec was found to have an acceptable safety profile, and efficacy assessments in this proof-of-concept study demonstrated encouraging improvements in retinal sensitivity, visual function, and functional vision.

Competitive Landscape

• Johnson & Johnson Innovative Medicine
• MeiraGTx
• Beacon Therapeutics
• Nanoscope Therapeutics
• GenSight Biologics
• 4D Molecular Therapeutics
• Coave Therapeutics
• Ocugen
• Bionic Sight
• jCyte
• Endogena Therapeutics
• ProQR Therapeutics
• Aldeyra Therapeutics

Retinitis Pigmentosa Market Segmentation

• By Inheritance Type
• Autosomal Dominant
• Autosomal Recessive
• X-linked
• By Disease Type
• Syndromic Retinitis Pigmentosa
• Non-Syndromic Retinitis Pigmentosa
• Systemic Retinitis Pigmentosa
• By Age of Onset
• Early-Onset Retinitis Pigmentosa
• Late-Onset Retinitis Pigmentosa

Regional Insights

• North America
  • U.S.
  • Canada
• Latin America
  • Mexico
  • Brazil
  • Argentina
  • Rest of Latin America
• Europe
  • Italy
  • Germany
  • U.K.
  • Spain
  • France
  • Russia
  • Rest of Europe
• Asia Pacific
  • South Korea
  • India
  • Japan
  • China
  • Australia
  • ASEAN
  • Rest of Asia Pacific
• Middle East
  • GCC Countries
  • Israel
  • Rest of the Middle East
• Africa
  • South Africa
  • North Africa
  • Central Africa