Ocular gene therapy introduces functional genes to correct mutations underlying inherited retinal diseases (IRDs) and other vision disorders. Leveraging advanced viral vectors such as adeno-associated virus (AAV), these therapies can restore sight and slow disease progression, offering hope for conditions once considered incurable. The eye’s immune-privileged status, small tissue volume, and accessibility make it an attractive site for gene therapy.

The global Ocular Gene Therapy market was valued at $1300 million in 2024 and is estimated to reach $7,360 million by 2033, registering a CAGR of 22% from 2025 to 2033.

Following Factors Driving the Market Growth 📈:

(1) Clinical & Regulatory Advancements 🔬

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) Investment Resilience 💸

Despite the broader gene therapy sector experiencing an 83% decline in venture funding between 2021 and 2024, ocular gene therapy continued to attract capital in 2024 and 2025. Atsena Therapeutics secured $150 million in a Series C round in April 2025, while Beacon Therapeutics and Ryze Therapeutics closed significant rounds of $170 million (Series B) and $86 million (Series A) in 2024. Big Pharma is also fueling momentum. AbbVie, for instance, entered a $1.3 billion agreement with REGENXBIO for RGX-314, which included a $370 million upfront payment. 

Despite challenges such as high treatment costs, complex manufacturing, and limited treatment facilities, the market outlook remains highly promising. The rising prevalence of inherited retinal diseases (IRDs) continues to create strong demand for novel therapies. At the same time, sustained investment from venture capital and major pharmaceutical companies underscores growing confidence in this field. Advances in CRISPR-based editing, optogenetics, and AI-driven genetic engineering are expected to accelerate innovation further and expand therapeutic options.

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:

• Market Growth: $1.3B (2024) → $7.36B (2033), CAGR 22%.

• Vector Trends: Viral vectors dominate (~80%), AAV leads; non-viral approaches growing fastest (37.9% CAGR).

• Indications: LCA & retinitis pigmentosa lead; AMD & glaucoma showing rapid expansion.

• Regional Outlook: North America leads, Europe follows, Asia-Pacific rising fastest.

• Clinical Pipeline: Strong presence in Phase 2 & 2/3; key assets: RGX-314, AGTC-501, ADVM-022, EDIT-101. Phase 3 trials are approaching regulatory submissions across the FDA, EMA, and PMDA.

• Funding & Deals: Over $7B in M&A/partnerships (2020–2025), including J&J–MeiraGTx ($415M) and AbbVie–REGENXBIO ($1.3B).

The report covers the following topics:

• Market drivers, restraints & opportunities

• Porter’s Five Forces Analysis

• Covid-19 Impact

• Segmentation by vector type, disease indication, and region

• Profiles of 25 companies with pipeline, financial analysis, and key strategic moves

• Comprehensive profile analysis of 5 leading therapies (including 2 approved)

• Clinical trial landscape analysis

• Market size & forecast (2023–2033)

• Investment trends, M&A activity & strategic recommendations

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