Coave Therapeutics showcases its Aligater platform for generating conjugated AAV vectors to deliver ocular gene therapy through suprachoroidal route


The data will be presented in oral session at the 2024 American Society of Gene & Cell Therapy meeting May 7-11 in Baltimore, Maryland.

Image credit: AdobeStock/adragan

(Image credit: AdobeStock/adragan)

Coave Therapeutics announced that preclinical studies on its novel Conjugated AAV (coAAV) gene therapy vectors demonstrate promising results for ocular gene therapy.

According to a news release, engineered coAAV vectors, generated using Coave’s Aligater platform, demonstrate the ability to increase both the distribution and expression patterns of transgenes delivered via suprachoroidal administration, effectively improving the targeting of key tissues at the back of the eye.

“Delivering gene therapies to the posterior segment of the eye, notably the retina, poses a key challenge: achieving broader distribution through a less invasive procedure compared to the subretinal injection route, while still ensuring sustained transgene expression in target cells,” said Lolita Petit, chief scientific officer at Coave. “This is an exciting time as our new data emphasize, for the first time, the potential of improving the delivery of gene therapy through the suprachoroidal space using Coave’s chemically engineered coAAVs. This holds the potential to enable broader applications in terms of scope of ocular diseases.”

The company noted that the development suggests a new, potentially impactful approach for treating acquired retinal diseases. These data, generated in collaboration with REGENXBIO, will be presented at the 2024 American Society of Gene & Cell Therapy (ASGCT) meeting by Gaelle Lefevre, vice president of Alliance & Portfolio Strategy at Coave Therapeutics. The meeting will be held virtually and in person May 7-11 in Baltimore, Maryland.1

According to its news release, through its Aligater platform, Coave has developed a range of new coAAV vectors, each specifically engineered with a distinct set of small molecule ligands at its surface through bioconjugation. These coAAVs have been screened for administration to the suprachoroidal space (SCS) of the eye, an attractive alternative to invasive subretinal administration to deliver AAV-based gene therapies to the back of the eye.

A library of coAAV candidates was injected into the SCS of non-human primates (NHPs) and evaluated in comparison to an unconjugated, benchmark AAV capsid. Three weeks post-injection, the distribution of vector genome copies and the levels of transgene-specific expression were analyzed in ocular and peripheral tissues.1

The company noted that its coAAVs showed more widespread and enhanced expression in key target tissues of the eye, notably the retina, versus the control vector, and lower off-target systemic distribution to tissues such as the liver and kidney.

According to the news release, Coave’s coAAVs represent some of the first engineered AAV vectors optimized for SCS delivery. These vectors open new avenues for next-generation gene therapy vectors for ocular administration, potentially eliminating the need for invasive subretinal injections. The superior properties of coAAVs for ocular gene transfer via SCS administration have the potential to enable the development of more efficient, targeted, and well-tolerated gene therapies for the treatment of common retinal disorders.1

  1. Therapeutics C. Coave Therapeutics Showcases its ALIGATERTM Platform for Generating Conjugated AAV (coAAV) Vectors Exhibiting Superior Performance in Delivering Ocular Gene Therapy through the Suprachoroidal Route. GlobeNewswire News Room. Published April 29, 2024. Accessed April 29, 2024.
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