Metagenomi presents in vivo CNS gene editing data at ASGCT; CAST integration efficiency improved >50x

Metagenomi Therapeutics, Inc.

Type II nuclease MG21-1 in single AAV achieved 69% protein knockdown in mouse motor cortex and 82% in brain via systemic delivery.
Ultra-compact Type V nuclease MG119-28 (488 aa) achieved 64% target protein knockdown and up to 52% editing efficiency in mouse CNS.
Type V-K CAST system engineered for >50x improvement in large gene integration efficiency vs. natural system in mammalian cells.
In vitro, MG119-28 restored dystrophin levels >50% in DMD myocytes and achieved complete gene disruption in K562 cells.
Platform aims to enable extrahepatic in vivo gene editing for neurological and neuromuscular disorders.

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