Coave Therapeutics has unveiled its new lead gene therapy program for the treatment of retinal vascular diseases such as wet age-related macular degeneration (AMD) and diabetic macular edema (DME).
First, a refresh on Coave.
Headquartered in Paris, France, the clinical-stage biotechnology company has developed a targeted gene therapy approach to “transform the treatment” of ocular diseases.
This approach is dependent on the proprietary ALIGATER platform, which conjugates ligands—small molecules, peptides, or antibody fragments—onto viral vectors (primarily adenovirus-associated viruses [AAVs]) in a one-step chemical process that results in precision vectors.
- Importantly: The platform is designed to address key limitations in genetic payload delivery to “extra-hepatic tissues” (any tissues outside the liver), including limited tissue specificity as well as delivery efficiency and safety.
- See a visual of this process.
Talk about these precision vectors.
Coave’s tissue-specific peptide-conjugated AAV (coAAV) vectors are reportedly agnostic to genetic payloads and capable of treating a range of indications previously untreatable via traditional gene therapy approaches.
- The ligand-conjugated vector of our interest: co-AAV-SCS.
And this lead candidate?
That would be CoTx-101, an AAV capsid powered by coAAV-SCS and designed for suprachoroidal delivery during an in-office procedure.
Per the company, its first precision suprachoroidal vector has the potential to treat wet AMD and DME, enabling:
- Durable efficacy, ideally resulting in ≥70% of patients injection-free at 1 year
- In contrast to repeated anti-vascular endothelial growth factor (VEGF) injections
- A low burden delivery targeting the back of the eye in an eyecare provider’s office
- Steroid-free treatment with continuous anti-angiogenic effects
- Treatment in the second eye
- Due to evading triggered immune responses following first administration
Interesting … and has it undergone any clinical research yet?
CoTx-101’s coAAV-SCS vector has.
In fact, just last month Coave presented a poster at the European Society of Gene and Cell Therapy (EGSCT) Congress 2025 detailing two clinical studies on the candidate.
Those studies: Compared coAAV-SCS against first and second-generation capsids (AAV2, AAV3B, AAV.7m8, AAV.v128, and AAV8) in primates.
The findings:
- The vector outperformed all other capsids by up to 26 times in targeting retinal pigment epithelium (RPE)-choroid and retinal cells
- Its superiority over AAV8 was confirmed by demonstrating improved transduction of both RPE-choroid and retinal photoreceptor cells
- coAAV-SCS also demonstrated an improved safety profile with de-targeting from the anterior segment and immune evasion properties
- Hence its potential for a safe second-eye treatment.
Sounds promising! Now back to this candidate—what’s next for CoTx-101?
Coave plans to complete target product profile (TPP)-validating studies on non-human primates sometime next year.
- Keep in mind: TTP studies are designed to test the profile of a new drug among clinicians to help define its intended use and target population, as well as safety, efficacy, and dosing.
The goal: For CoTx-101 to be ready for an investigational new drug (IND) submission by 2027.