ATX001

ATX001 is an engineered adeno-associated virus (AAV) capsid derived from the AAV2 parental serotype, developed by Avista Therapeutics. It is designed for efficient gene delivery to the central nervous system (CNS) via cerebrospinal fluid (CSF) administration, such as intracisternal magna (ICM) or intracerebroventricular (ICV) injection. ATX001 demonstrates significantly improved CNS transduction and manufacturability (higher empty/full ratios) compared to natural serotypes like AAV2 and AAV9. In preclinical studies involving mice and non-human primates (NHPs), ATX001 showed robust, largely neuronal transgene expression across various brain regions, including the cerebellum, midbrain, brainstem, hippocampus, and cortex, as well as significant transduction of ependymal cells. Its ability to achieve widespread distribution at lower doses compared to systemic administration suggests potential for treating diverse neurological disorders while minimizing the risk of liver toxicity.