NanoCas

NanoCas is an ultracompact CRISPR-associated (Cas) nuclease engineered for efficient in vivo genome editing. Developed by Mammoth Biosciences, it is approximately one-third the size of traditional Cas9 nucleases and can be delivered systemically using a single adeno-associated viral (AAV) vector. This compact size overcomes major delivery limitations of first-generation CRISPR systems, enabling gene editing in tissues beyond the liver—such as skeletal muscle—in both mice and non-human primates. In preclinical studies, NanoCas achieved high editing efficiencies (up to 60% in mouse liver and 30%+ in primate muscle), matching or exceeding larger Cas enzymes like SaCas9. Its small genetic footprint allows room for additional elements such as guide RNAs or base/epigenetic editors within a single AAV payload. The primary mechanism involves programmable DNA cleavage at target sites specified by guide RNAs, facilitating gene disruption or correction for therapeutic applications including Duchenne muscular dystrophy and hypercholesterolemia[2][3][4][5][7].

Brand names

NanoCas

Other names

NanoCasultracompact CRISPR nucleaseultracompact Cas enzyme