Retinitis Pigmentosa Treatment Market - 2025-2033

Retinitis Pigmentosa Treatment Market Size

Retinitis Pigmentosa Treatment Market size reached US$ 5.72 Billion in 2024 and is expected to reach US$ 11.13 Billion by 2033, growing at a CAGR of 7.7% during the forecast period 2025-2033.

Retinitis Pigmentosa Treatment Market The retinitis pigmentosa treatment market is poised for robust expansion, underpinned by groundbreaking gene and cell therapies, growing diagnostic capabilities, and increasing patient engagement. Stakeholders ranging from large pharma to specialized biotech must navigate high development costs, regulatory complexities, and access barriers to capitalize on this high-growth opportunity. A strategic focus on personalized approaches, digital health integration, and emerging market access will be critical for sustainable leadership in this evolving therapeutic landscape.

Retinitis Pigmentosa Treatment Market Dynamics: Drivers & Restraints

Rising demand for innovative therapeutics is significantly driving the retinitis pigmentosa treatment market growth

Retinitis Pigmentosa is a rare genetic disorder with very limited treatment options. The lack of effective therapies for the condition creates a substantial unmet medical need, expected to increase the demand for innovative solutions that can address the causes of the disease and provide meaningful benefits to patients. The major market players are performing clinical trials to develop innovative therapeutics.

Innovative therapeutics, such as gene therapies and advanced treatments, have the potential to offer improved patient outcomes compared to other traditional or symptomatic approaches. Patients and healthcare providers often seek treatments that can not only manage symptoms but also slow down or halt the progression of the disease. So, there is an increasing demand for innovative gene therapies. Thus, market players are focusing on developing gene therapies, boosting the market growth.

For instance, in February 2025, Ocugen, Inc., a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, announced that the European Commission has provided a positive opinion from the European Medicines Agency’s (EMA) Committee for Advanced Therapies (CAT) for OCU400 Advanced Therapy Medicinal Product (ATMP) classification. OCU400 is the first gene therapy to enter Phase 3 with a broad retinitis pigmentosa (RP) indication.

Additionally, in May 2025, Skyline Therapeutics, an innovation-driven clinical-stage gene therapy company focused on developing unique and novel solutions for rare and severe diseases, announced a late-breaking abstract presentation on SKG1108, its investigational optogenetic gene therapy for Retinitis Pigmentosa (RP), at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting.

Availability of limited approved treatment options is hampering the retinitis pigmentosa treatment market's growth

Luxturna (voretigene neparvovec), the only FDA-approved gene therapy for retinitis pigmentosa, is indicated solely for biallelic RPE65 mutations, present in fewer than 5% of all retinitis pigmentosa patients. This leaves the vast majority of retinitis pigmentosa sufferers without any approved gene-based option. With over 60 genes implicated in retinitis pigmentosa, each requiring separate clinical development and regulatory review, few companies have the resources to pursue more than a handful of indications. The time and expense of running dozens of small, mutation-specific trials create a de-risking challenge for investors.

The lack of multiple approved therapies reflects the technical and regulatory challenges in developing RP treatments, such as difficulties in designing clinical trials for rare mutations and proving long-term efficacy. Biotech firms and investors may hesitate to fund RP drug development due to uncertain regulatory pathways and limited precedent for approval, thereby slowing innovation.

The limited number of approved RP treatments, exemplified by Luxturna’s narrow mutation target and the absence of other widely accepted therapies, restricts market growth by limiting patient access, increasing development risk, complicating reimbursement, and dampening demand. Expanding approved treatment options across diverse RP genotypes is critical for unlocking the full market potential.

Retinitis Pigmentosa Treatment Market, Segment Analysis

The global retinitis pigmentosa treatment market is segmented based on disease type, treatment type, and region.

The gene therapy from the treatment type segment is expected to hold 59.75% of the market share in 2024 in the retinitis pigmentosa treatment market

The gene therapy segment is expected to hold the largest market share over the forecast period. Gene therapy for retinitis pigmentosa involves introducing functional copies of a defective gene into the patient's cells to compensate for the genetic mutation causing the condition. Voretigene neparvovec-ryzl (Luxturna) is the only gene therapy product approved by the FDA to treat a specific type of retinitis pigmentosa with mutations in both copies of the RP65 gene. Luxturna delivers a functional copy of the RPE65 gene to the retinal cells, aiming to restore vision.

Besides Luxturna, various other gene therapies for retinitis pigmentosa are under investigation in clinical trials. These trials are exploring different gene therapy approaches, including the use of various viral vectors to deliver therapeutic genes, to address various genetic mutations associated with the condition. The major market players are focusing on the development of gene therapies for the management of retinitis pigmentosa, which is driving the segment’s growth.

For instance, in May 2025, Beacon Therapeutics Holdings Limited 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). Laru-zova is a potential best-in-class gene therapy designed to restore the natural function of both rods and cones in XLRP by delivering a functional copy of the RPGRORF15 gene using a well-established vector with a proprietary capsid designed for high transduction of photoreceptors, and a codon-optimized gene to produce the full-length protein.

Additionally, in January 2025, ViGeneron GmbH, a next-generation clinical-stage gene therapy company, announced two important milestones for its novel gene therapy candidate VG901, to treat patients with retinitis pigmentosa (RP) caused by mutations in the CNGA1 gene. The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to VG901, and the independent Data Safety Monitoring Board (DSMB) has unanimously approved dose escalation in the ongoing Phase 1b clinical trial.

Retinitis Pigmentosa Treatment Market, Geographical Analysis

North America is expected to dominate the global retinitis pigmentosa treatment market with a 46.98% share in 2024

North America region is expected to hold the largest market share over the forecast period, owing to the strong presence of major players and increasing research activities for developing advanced therapies. North America, especially the United States and Canada, is a known hub for its strong presence of major players such as pharmaceutical companies, which actively focus on the development of novel therapies for better treatment.

For instance, in August 2024, Ocugen, Inc., a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that Health Canada provided a “No Objection Letter” to initiate the OCU400 Phase 3 liMeliGhT clinical trial in Canada. OCU400 is a modifier gene therapy product candidate being developed for retinitis pigmentosa (RP). These focus on developing novel therapeutics, boosting market growth in the region.

In addition, there are increasing research activities and clinical trials in the region for developing more advanced therapeutics for the better management of retinitis pigmentosa, which also helps to drive the market in the region. As the research activities and clinical trials increase, therapeutics are developed more precisely by reducing the complications associated with the different types of therapies, resulting in better patient outcomes.

Retinitis Pigmentosa Treatment Market Top Companies

Top companies in the retinitis pigmentosa treatment market include Spark Therapeutics, Inc., jCyte, Inc., Coave Therapeutics, Kiora Pharmaceuticals, Inc., Aldeyra Therapeutics, Inc., Beacon Therapeutics, ViGeneron GmbH, Nanoscope Therapeutics Inc., SparingVision, Inc., and Endogena Therapeutics Inc., among others.

The global Retinitis Pigmentosa Treatment market report delivers a detailed analysis with 54 key tables, more than 45 visually impactful figures, and 157 pages of expert insights, providing a complete view of the market landscape.