Gene therapy in ophthalmology involves the use of viral and non-viral vectors to deliver genetic material directly into ocular tissues, aiming to treat inherited retinal diseases, age-related macular degeneration (AMD), and other degenerative disorders of the eye. Key products include adeno-associated virus (AAV) vectors, lentiviral systems, and emerging CRISPR/Cas-based modalities, each offering precise targeting and long-term gene expression with minimized systemic exposure. Gene Therapy in Ophthalmology Market Advantages of these therapies include the potential for one-time administration, restoration of lost vision, and reduced reliance on chronic injections or implants. The rising incidence of hereditary retinal dystrophies and the unmet need for durable, disease-modifying solutions have fueled intense market research and investment. Strategic collaborations between biotech firms and academic centers have accelerated early-stage pipeline development, while ongoing clinical trials demonstrate encouraging safety and efficacy profiles. Growing awareness of market opportunities among investors and regulatory agencies has further strengthened confidence in gene therapy as a mainstream ophthalmic treatment.

The gene therapy in ophthalmology market is estimated to be valued at USD 1.51 Bn in 2025 and is expected to reach USD 7.36 Bn by 2032, growing at a compound annual growth rate (CAGR) of 25.4% from 2025 to 2032.

Key Takeaways

Key players operating in the Gene Therapy in Ophthalmology Market are

Johnson & Johnson Innovative Medicine,

·         MeiraGTx,

·         Beacon Therapeutics,

·         Nanoscope Therapeutics,

·         GenSight Biologics.

These market players have established robust pipelines encompassing both inherited and acquired retinal conditions and are leveraging partnerships to expand R&D capabilities. Johnson & Johnson Innovative Medicine has invested heavily in vector engineering and has secured several orphan drug designations, strengthening its market share. MeiraGTx and GenSight Biologics focus on Leber congenital amaurosis (LCA) and retinitis pigmentosa, respectively, while Beacon Therapeutics and Nanoscope Therapeutics are advancing next-generation AAV and nanoparticle platforms. Through licensing agreements and joint ventures, these companies are executing targeted market growth strategies to broaden their geographic footprint, optimize manufacturing scale-up, and secure reimbursement pathways. Comprehensive market research reports highlight their competitive positioning, revealing an industry structure with a mix of established pharma giants and agile biotech startups driving market dynamics.

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