Coave Therapeutics has announced the launch of coAAV-CSF-01, a novel CNS-targeted gene therapy vector developed using its proprietary ALIGATER™ platform. The company revealed breakthrough data from non-human primate studies demonstrating the vector's superior performance in brain tissue transduction and safety following intra-cerebrospinal fluid administration compared to conventional AAV9 vectors.
The groundbreaking results have been selected for presentation as a Late-Breaking Abstract at the 2025 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, taking place May 13-17 in New Orleans.
Remarkable Efficacy and Safety Profile
In non-human primate studies, coAAV-CSF-01 (research code S01coAAV2) was administered via intra-cerebrospinal fluid routes, including intracisternal magna and intracerebroventricular delivery methods. The results revealed dramatic improvements over existing gene therapy vectors:
- 100-fold higher transgene expression in the cortex compared to AAV9
- 10,000-fold increased expression in the hippocampus versus AAV9 at equivalent doses
- Comparable CNS biodistribution and expression at just one-fifth the dose of AAV9
- Significantly reduced peripheral transduction, including strong liver de-targeting
- Enhanced peripheral nerve safety, addressing key concerns in CNS gene therapy
"These exciting new data from coAAV-CSF-01 represent a major step forward in CNS gene therapy," said Lolita Petit, CSO of Coave Therapeutics. "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."
Addressing Critical Challenges in CNS Gene Therapy
The development of effective gene therapies for central nervous system disorders has faced persistent challenges, particularly regarding delivery efficiency, tissue specificity, and safety. Traditional AAV vectors often require high doses to achieve therapeutic effects in the brain, increasing the risk of adverse events and immunogenicity.
coAAV-CSF-01 appears to overcome these limitations through targeted engineering of the AAV2 capsid using Coave's ALIGATER™ platform. This proprietary technology enables chemical conjugation-based modification of AAV vectors, offering a modular approach to enhance tissue targeting and transduction efficiency.
The enhanced biodistribution and improved safety profile demonstrated in these studies support the vector's potential for developing new genetic medicines targeting neurodegenerative and neurodevelopmental CNS disorders.
ALIGATER™: A Platform Technology for Next-Generation Gene Therapies
Coave's ALIGATER™ (Advanced Vectors-Ligand Conjugates) platform represents a significant technological advancement in the gene therapy field. The platform enables conjugation of targeting ligands—such as small molecules, peptides, or antibody fragments—onto AAV or non-viral vectors.
This approach offers several advantages:
- Superior delivery efficiency to target tissues
- Enhanced tissue specificity for precision medicine applications
- Improved safety profiles through reduced off-target effects
- Streamlined manufacturing without requiring prior AAV capsid modifications
"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 explained. "Together, the data position coAAV-CSF-01 as a potentially transformative advance in gene therapy delivery."
Upcoming Scientific Presentation
The detailed findings will be presented at the ASGCT Annual Meeting in a Late-Breaking Poster Presentation titled "Enhanced CNS transduction and safety of S01coAAV2 (coAAV-CSF-01) following intra-cerebrospinal fluid (CSF) administration in cynomolgus macaques." The presentation will be delivered by Julien Spatazza, Senior Director of Discovery & Preclinical Research at Coave Therapeutics, on Thursday, May 15, 2025.
coAAV-CSF-01 is part of Coave's expanding portfolio of proprietary AAV capsids engineered to optimize gene delivery in challenging therapeutic areas. The Paris-based company, backed by leading international life sciences investors, continues to advance its pipeline targeting CNS, neuromuscular, and eye diseases.
