Coave Therapeutics Launches CNS-Targeted Gene Therapy Capsid Developed with Proprietary Platform

In a poster presentation at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, held May 13–17, 2025 in New Orleans, Paris-based Coave Therapeutics presented breakthrough data from studies of a novel, central nervous system (CNS)-targeted gene therapy capsid, research code S01coAAV2, that was developed using the company’s proprietary Advanced Vectors—Ligand Conjugates (ALIGATER) platform (1).

Coave Therapeutics took the occasion of ASGCT 2025 to officially launch the vector as coAAV-CSF-01, to go along with its late-breaking abstract presented in poster form on Thursday, May 15, “Enhanced CNS Transduction and Safety of S01coAAV2 (coAAV-CSF-01) Following Intra-Cerebrospinal Fluid (CSF) Administration in Cynomolgus Macaques,” presented by Julien Spatazza, PhD, Coave Therapeutics senior director of Discovery & Preclinical Research (1). Cynomolgus macaques are non-human primates also known as crab-eating or long-tailed macaques (2).

Improved brain tissue transduction

In the studies of coAAV-CSF-01, the vector was administered by multiple intra-cerebrospinal fluid (CSF) routes, among them intracisternal magna and intracerebroventricular, and its performance was compared to that of AAV9 (adeno-associated virus serotype 9) therapy (1). Results demonstrated, in general, higher transgene expression in targeted brain regions and a better safety profile.

More specifically, Coave Therapeutics said that coAAV-CSF-01’s transgene expression was 100-fold higher in the cortex, with a 10,000-fold increase in the hippocampus when compared with AAV9 at the same dosage (1). At just one-fifth the dose of AAV9, comparable CNS biodistribution and expression were observed. And peripheral transduction was significantly reduced, according to the study data, especially in terms of liver de-targeting, while peripheral nerve safety was preserved as well.

“These exciting new data from coAAV-CSF-01 represent a major step forward in CNS gene therapy,” said Lolita Petit, PhD, chief scientific officer of Coave Therapeutics, in a company press release (1). “In non-human primates, the vector achieved robust brain transduction via intra-CSF delivery—a route long viewed as promising but historically constrained by limited efficacy and off-target effects. The enhanced biodistribution and improved safety demonstrated in these studies support its potential in developing new genetic medicines for neurodegenerative and neurodevelopmental CNS disorders.”

ALIGATER’s function and financing

“Furthermore, the vector was developed using our proprietary ALIGATER platform, enabling modular modification of AAV2 and other capsids to optimize tissue targeting and therapeutic performance—potentially opening the door to broader therapeutic applications,” Petit continued (1). “Together, the data position coAAV-CSF-01 as a potentially transformative advance in gene therapy delivery.”

In January 2025, Coave Therapeutics announced that it had secured €32 million (US$33 million) in Series A financing that allowed the company to advance the ALIGATER platform (3). The financing was co-led by Novo Holdings and Bpifrance with participation from leading United States and European Union investors Invus and UI Investissement, respectively, plus existing investors Seroba Life Sciences, Fund+, Kurma Partners, Omnes Capital, and Turenne Capital.

Access all our ASGCT 2025 coverage!

References

1. Coave Therapeutics. Coave Therapeutics Launches coAAV-CSF-01, a Novel CNS-Targeted Gene Therapy Vector for Neurodegenerative and other CNS Diseases. Press Release. May 9, 2025.
2. Cynomolgus Macaque. The Macaque Website—National Centre for the Replacement, Refinement & Reduction of Animals in Research. Macaques.nc3rs.org.uk (accessed May 16, 2025).
3. Coave Therapeutics. Coave Therapeutics Secures €32 Million ($33 Million) in Series A Financing to Advance its Next-Generation Genetic Medicines. Press Release. Jan. 9, 2025.