Award
National Institute of Neurological Disorders and Stroke RFA-RM-22-015
Research Project I: AAV-CRISPR to Treat Huntington's Disease
Recipient
University of California Berkeley
Award Amount
$427,885.00
Ceiling
$427,885.00
Awarded
June 29, 2026
Identifier
RFA-RM-22-015
A research project led by University of California Berkeley to develop AAV-CRISPR treatment for Huntington's Disease, involving preclinical testing and aiming for FDA clinical trial application.
Description
The project involves employing CRISPR-Cas9 system delivered via engineered AAV capsid (AAV-LC.V1) for Huntington's Disease treatment. The study includes in vitro screening, efficacy testing in murine models, toxicity and biodistribution in rats, and efficacy and safety assessment in non-human primates. The project aims to support an IND application to the FDA for clinical testing.