Coave Therapeutics unveils lead AAV gene therapy program for retinal vascular diseases

PARIS, France – 14 November 2025

Coave Therapeutics (“Coave”), a company redefining targeted gene therapy with a best-in-class pipeline powered by ligand-conjugated AAV vectors (AAV), today announced the nomination of its lead gene therapy program, CoTx-101, for the treatment of retinal vascular diseases, such as wet age-related macular degeneration (wAMD) and diabetic macular edema (DME).

AAV Vector Innovation: CoTx-101 Poised for Breakthrough

CoTx-101 represents a potential breakthrough for patients with retinal vascular diseases, offering the efficacy and safety of biologics with the unique durability of gene therapy (AAV). It is powered by Coave’s first-in-class proprietary AAV suprachoroidal vector (coAAV-SCS), which is a unique AAV vector designed to target the back of the eye.

The unique AAV vector in CoTx-101 (coAAV-SCS) has demonstrated unprecedented targeting of cells at the locus of the disease, including RPE and photoreceptors, according to non-human primate studies. This deep retinal penetration and broad tissue coverage are designed to enable sustained expression of two clinically validated biologics targeting the angiogenesis pathway.

AAV-Powered Delivery: In-Office Suprachoroidal Procedure

The program utilizes AAV for suprachoroidal delivery, which enables an in-office procedure targeting the back of the eye—a significant advantage over current invasive surgical gene therapy procedures. By combining a best-in-class in-office delivery procedure with a first-in-class AAV vector, CoTx-101 aims to deliver durable vision gains, offering the highest clinical benefit with the lowest treatment burden.

“The launch of our lead therapeutic suprachoroidal program validates our delivery-first approach to redefining targeted gene therapy,” commented Rodolphe Clerval, CEO of Coave Therapeutics. “With our first-in-class ligand-conjugated AAVs, we are creating precision AAV vectors that are highly tissue-specific, precisely delivered, and safer, powering targeted gene therapies.”

He added that CoTx-101 aims to bring patients the ultimate treatment for retinal vascular diseases, offering the efficacy and safety of biologics with the unique durability of gene therapy (AAV).

Addressing High Treatment Burden with AAV Durability

Retinal vascular diseases, such as wAMD (affecting an estimated 10-20 million adults globally) and DME (affecting 37 million people), are currently treated with Anti-VEGF biologics. However, this current standard of care requires repeated injections every 4-12 weeks, often for life. This results in a significant treatment burden, with over 40% of patients discontinuing treatment after the first year, which leads to uncontrolled disease progression and further vision loss.

Coave recently presented data at the European Society of Gene and Cell Therapy (EGSCT) Congress 2025 where its novel AAV vector demonstrated unprecedented transduction efficacy in non-human primate studies, as well as evasion from the immune system. This suggests a safer, more effective AAV treatment, potentially even enabling second-eye treatment.

The Company plans to complete TPP (Target Product Profile) validating non-human primate studies of CoTx-101 in 2026, with the aim to be IND (Investigational New Drug)-ready in 2027.