Coave Therapeutics Unveils coAAV-CSF-01: A Novel CNS-Targeted Gene Therapy Vector Poised to Transform Treatment of Neurodegenerative and Other CNS Diseases

Paris, France – May 09, 2025 – Coave Therapeutics, a pioneering gene therapy company headquartered in Paris, today announced the launch of coAAV-CSF-01, a groundbreaking central nervous system (CNS)-targeted gene therapy vector developed using its proprietary ALIGATER™ platform. Concurrent with this announcement, compelling new preclinical data highlighting the vector’s exceptional potential are being presented as a Late-Breaking Abstract at the 2025 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting in New Orleans, LA.

The newly unveiled coAAV-CSF-01 (research code S01coAAV2) demonstrates a significant leap forward in the delivery of gene therapies to the complex environment of the CNS. Studies in non-human primates (NHPs) have shown that administration via intra-cerebrospinal fluid (intra-CSF) routes, including intracisternal magna (ICM) and intracerebroventricular (ICV) delivery, results in dramatically enhanced transgene expression in key brain regions with a superior safety profile compared to the widely utilized AAV9 vector.

Key breakthrough findings associated with coAAV-CSF-01 include:

• Remarkably Elevated Gene Expression: Compared to AAV9 at the same dose, coAAV-CSF-01 achieved a 100-fold increase in transgene expression in the cortex and an extraordinary 10,000-fold increase in the hippocampus, critical areas affected in numerous neurodegenerative and neurodevelopmental conditions.
• Efficient CNS Targeting at Lower Doses: The novel vector demonstrated comparable CNS biodistribution and gene expression at a mere one-fifth of the dosage required for AAV9, potentially mitigating safety risks and reducing treatment costs.
• Enhanced Safety Profile through Reduced Off-Target Effects: A significant advantage of coAAV-CSF-01 is its substantially reduced transduction in peripheral tissues, including notable de-targeting of the liver, and an improved profile concerning peripheral nerve safety – crucial factors for the safe and effective treatment of CNS disorders.

“These exciting new data from coAAV-CSF-01 represent a major step forward in our quest to develop transformative genetic medicines for devastating CNS diseases,” stated Lolita Petit, CSO of Coave Therapeutics. “The robust brain transduction achieved via intra-CSF delivery in non-human primates, coupled with the improved safety profile, overcomes historical limitations associated with this promising delivery route. We believe coAAV-CSF-01 holds immense potential for addressing a wide spectrum of neurodegenerative and neurodevelopmental conditions.”

The development of coAAV-CSF-01 is a testament to the power of Coave’s innovative ALIGATER™ platform. This proprietary technology enables the modular modification of AAV2 and other capsid serotypes through chemical conjugation, allowing for precise optimization of tissue targeting and therapeutic performance. This platform holds the key to unlocking broader therapeutic applications for gene therapy beyond the CNS.

As part of Coave’s growing portfolio of engineered AAV capsids, coAAV-CSF-01 signifies a major advancement in overcoming the challenges of gene delivery to the CNS. The promising data underscore its potential to revolutionize the treatment landscape for neurodegenerative diseases and other debilitating conditions affecting the central nervous system. Coave Therapeutics is committed to advancing this groundbreaking technology towards clinical development, offering new hope for patients and their families.