Coave Therapeutics Unveils coAAV-CSF-01, a New CNS Gene Therapy Vector

Coave Therapeutics, a leader in genetic medicine innovation, has unveiled promising new findings related to its groundbreaking gene therapy vector, coAAV-CSF-01. This vector, part of Coave’s ALIGATER™-engineered AAV2-based capsid series (S01coAAV2), is demonstrating significant advancements in gene delivery to brain tissues. These findings are set to be presented as a Late-Breaking Abstract at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting in 2025, highlighting its potential to revolutionize central nervous system (CNS) gene therapy.

The recent study involving non-human primates (NHPs) illustrates that coAAV-CSF-01, administered through intra-cerebrospinal fluid routes such as intracisternal magna and intracerebroventricular injections, leads to notably enhanced transgene expression in targeted brain areas. This improved delivery method also shows a superior safety profile compared to the widely used AAV9.

Lolita Petit, Chief Scientific Officer of Coave Therapeutics, emphasized the significance of these developments. According to Petit, the robust brain transduction achieved through the intra-CSF route with coAAV-CSF-01 marks a pivotal advancement. Historically, this method was seen as promising due to its potential to reach the CNS effectively, but it faced challenges related to limited efficacy and unintended off-target effects. The new data on enhanced biodistribution and improved safety demonstrate the potential of coAAV-CSF-01 in advancing treatments for complex neurodegenerative and neurodevelopmental disorders of the CNS.

Developed using Coave's proprietary ALIGATER™ platform, coAAV-CSF-01 represents a modular innovation in capsid engineering. This platform allows for precise chemical modification of AAV2 and other capsids, aiming to optimize tissue targeting and therapeutic efficacy, potentially extending the scope of gene therapies.

Coave Therapeutics is building a robust portfolio of proprietary AAV capsids to enhance gene delivery in challenging therapeutic domains. The ALIGATER™ platform facilitates chemical conjugation-based adjustments of AAV vectors (coAAVs), presenting a scalable and versatile method to augment tissue targeting and enhance transduction efficiency. These features position coAAV-CSF-01 as a potential transformative solution in gene therapy delivery systems.

The full details of Coave’s findings are accessible through the ASGCT meeting platform, where more insights into this innovative therapy will be shared.

Coave Therapeutics, headquartered in Paris, France, is committed to advancing genetic medicine by addressing critical challenges in gene therapy delivery, particularly to tissues beyond the liver. The company’s ALIGATER™ platform is pivotal in overcoming previous limitations such as tissue specificity and safety. This technology supports the development of best-in-class gene therapies, tailored for specific medical conditions, leveraging targeted ligand conjugation to improve delivery precision and efficiency.

As a pioneer in genetic medicine, Coave's efforts are backed by prominent life sciences investors, underscoring the growing interest and potential impact of its pioneering research and development in genetic treatments.