Ocular Gene Therapy Market

Ocular gene therapy delivers functional genes to treat inherited retinal diseases (IRDs) and other eye disorders by correcting genetic mutations. Using advanced viral vectors such as adeno-associated virus (AAV), these therapies can restore vision and halt the progression of conditions that were previously considered untreatable. The eye’s unique characteristics, such as immune privilege, accessibility, and small tissue volume, make it an ideal target for gene therapy interventions.

According to Biotech Forecasts, the global Ocular Gene Therapy Market was valued at USD 1300 million in 2024 and is estimated to reach USD 7,360 million by 2033, registering a CAGR of 22% from 2025 to 2033.🚀

Following Factors Driving the Market Growth 📈:

(1) Pipeline Progress & Regulatory Momentum 🔬: As of June 2025, there are 73 active clinical trials underway in ocular gene therapy, with 75% focused on IRDs. A significant number of these candidates have progressed to late-stage development and may secure regulatory approval by 2026, including ADVM-022, GS010, and BIIB111. The FDA’s approval of LUXTURNA in 2017 marked a milestone in this space, with further momentum gained through the recent FDA approval of ENCELTO in March 2025.

(2) Sustained Investment Despite Sector Slowdown💸: While the broader gene therapy sector experienced an 83% decline in venture funding between 2021 and 2024, ocular gene therapy continued to attract capital in 2024 and 2025. Notable examples include Atsena Therapeutics, which secured $150 million (Series C) in April 2025, and Beacon Therapeutics and Ryze Therapeutics, which raised $170 million (Series B) and $86 million (Series A), respectively, in 2024. Major pharmaceutical firms are investing heavily in R&D and partnerships. For instance, AbbVie signed a $1.3 billion deal with REGENXBIO for RGX-314, including a $370 million upfront payment.

Additional factors driving market growth include the rising prevalence of inherited retinal diseases (IRDs) such as retinitis pigmentosa and Leber congenital amaurosis, along with advancements in non-viral gene editing technologies like CRISPR-Cas9 and Prime Editing, and the emergence of innovative techniques such as optogenetics, lipid nanoparticles, and AI-guided genetic modification.

However, challenges such as the high cost of treatment (e.g., Luxturna at $425,000 per eye), manufacturing bottlenecks, limited treatment centers, and immunogenicity risks may hinder market growth.

The report also presents a detailed quantitative analysis of the current market trends and future estimations from 2023 to 2033. The forecasts cover 2 Vector Types, 6 Disease Indication Types, 4 Regions, and 17 Countries.

The report includes a supplementary file containing all quantitative forecast data, along with a clinical trials dataset referenced throughout the report.

The report has the following key findings:

• The market is projected to grow from $1.3 billion in 2024 to $7.36 billion by 2033, with a CAGR of 22% (2025–2033).

• Viral vectors hold around 80% of the market, led by AAV use in ocular gene therapy, while non-viral vectors are growing fastest at a CAGR of 37.9%. 

• Leber Congenital Amaurosis (LCA) and Retinitis Pigmentosa dominate by disease indication. However, conditions like AMD and glaucoma are seeing fast growth.

• North America leads the market, with Europe following, while Asia-Pacific is emerging as the fastest-growing region.

• Phase 2 and Phase 2/3 trials make up a significant portion of ongoing clinical studies, with key candidates including RGX-314, AGTC-501, ADVM-022, and EDIT-101.

• Multiple Phase 3 trials are nearing completion, with regulatory filings anticipated across the FDA, EMA, and PMDA.

• Despite a general slowdown in funding, companies with strong platforms continue to attract investment, particularly those working on CRISPR and optogenetic technologies.

• From 2020 to 2025, the market has seen over $7 billion in M&A and collaboration deals, including: Johnson & Johnson (J&J) $415M agreement with MeiraGTx for bota-vec, AbbVie and REGENXBIO’s partnership for RGX-314, valued at $1.3 billion.

The report covers the following topics: 

• Market drivers, restraints, and growth opportunities

• Porter’s Five Forces Analysis

• Covid-19 Impact

• Detailed segmentation by vector type, disease indication, and region

• 25 company profiles, including pipelines, financials, and key strategies

• Product profile analysis of 5 leading therapies, including 2 approved products

• Clinical trial landscape analysis

• Market value and forecast from 2023 to 2033

• Investment trends, M&A activities, and strategic recommendations