Advances in gene therapy using OCU410 for geographic atrophy (common dry AMD)

Advances in gene therapy using OCU410 for geographic atrophy (common dry AMD)

Ocugen presented promising preliminary results from the ArMaDa phase 1 clinical trial evaluating gene therapy. OKU410 as a treatment geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD). This innovative therapy, administered as a single dose by subretinal injection, has the potential to revolutionize our approach to this eye disease that severely affects vision.

Main results after six months in the initial phase of the study:

1. Guaranteed safety: no serious adverse events associated with the treatment were recorded, indicating its tolerability.
2. Proven Effectiveness: Lesion growth was 21.4% slower in treated eyes than in untreated eyes.
3. Structural preservation: There was greater preservation of retinal tissue around the lesions, which could prolong the visual health of patients.
4. Functional improvements: Low-light visual acuity (LLVA), a key measure of visual functionality, stabilized in 100% of treated eyes.

Complete solution in one dose

Main advantage OKU410 is that it targets multiple disease pathways, including lipid metabolism, inflammation, oxidative stress and complement activation, all in one treatment. This distinguishes it from existing treatments, which require frequent injections and only target part of the disease process. Via nuclear receptor HP2E3OCU410 protects and can repair retinal tissue and retinal pigment epithelium (RPE).

Progress in clinical trials

The study is now moving into phase two, where the safety and effectiveness of higher doses will be assessed in a larger group of 45 patients divided into three cohorts: medium dose, high dose and control group. This study involves up to 13 retinal surgery centers in the United States, with dosing expected to be completed in 2025.

Ocugen leads the way in retinal disease research

In addition to OCU410, the company is developing other promising gene therapies. GARDian court investigates OKU410ST For Stargardt diseasewhile OCU400 focuses on Leber congenital amaurosis (DKA). These developments strengthen Ocugen’s mission to offer innovative solutions for retinal diseases for which there have been no effective therapeutic options to date.

With these advances, Ocugen is ushering in a new era of hope for patients and families suffering from disorders that severely impact vision health.

Advances in gene therapy using OCU410 for geographic atrophy (common dry AMD)

Translation: Retinal macula association

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