• Poster presentation highlights SMART adenine base editors (ABEs) small enough to be packaged into a single adeno-associated virus (AAV)
• Data demonstrate compact SMART system optimizations allowing for therapeutically-relevant genome editing

EMERYVILLE, Calif., Oct. 14, 2024 (GLOBE NEWSWIRE) -- Metagenomi, Inc. (Nasdaq: MGX), a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary gene editing toolbox, today presented a poster titled “Engineering of compact and efficient adenine base editors from metagenomic derived systems” at the American Institute of Chemical Engineers (AIChE) 7th International Conference on CRISPR Technologies in San Diego, CA.

“We are thrilled to share the progress we’ve made with our compact SMART systems,” said Chris Brown, PhD and Head of Discovery at Metagenomi. “Our update highlights the improved efficiency of our SMART platform across multiple targets and cell types. We demonstrated that our SMART nucleases can be engineered into functional base editors within the packaging limits of standard AAV. We believe the improved editing efficiency of these SMART systems will enable pursuit of neuromuscular targets, either on our own or in partnerships.”

Metagenomi’s SMART platform potentially addresses a key challenge of in vivo gene editing: efficient delivery of gene editing components to tissues beyond the liver. Currently, AAV is an established approach for delivery to extrahepatic tissues, which is necessary for treating neuromuscular diseases such as Duchenne muscular dystrophy (DMD), familial amyotrophic lateral sclerosis (ALS), and Charcot-Marie-Tooth disease type 1A (CMT1A). Traditional gene editing tools such as SpCas9 exceed the cargo capacity of standard AAV vectors, potentially necessitating the need for dual AAV systems, which may reduce overall editing efficiency, increase required dosages, and complicate the manufacturing process. Metagenomi’s SMART genome editing systems are small enough to be packaged into standard AAV vectors, even when additional effector domains are included for base editing, potentially enabling a differentiated therapeutic approach.

Metagenomi has taken a multi-pronged approach to optimizing the SMART platform since its initial discovery (Aliaga Goltsman, Daniela S., et al. “Compact...” Nature Communications, vol. 13, no. 1), including structure-guided engineering enabled through collaboration with Professor David Taylor and his lab at UT Austin (Ocampo, Rodrigo Fregoso, et al. "DNA..." bioRxiv). In addition, Metagenomi has applied advanced artificial intelligence (AI) tools trained on natural SMART enzymes, which was presented at the Cold Spring Harbor Laboratory (CSHL) CRISPR Frontiers conference earlier this year. The AI-driven approach underscores the importance of leveraging multiple cutting-edge techniques to advance next-generation gene editing systems.

About Metagenomi

Metagenomi is a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary, comprehensive metagenomics-derived toolbox. Metagenomi is harnessing the power of metagenomics, the study of genetic material recovered from the natural environment, to unlock four billion years of microbial evolution to discover and develop a suite of novel editing tools capable of correcting any type of genetic mutation found anywhere in the genome. Its comprehensive genome editing toolbox includes programmable nucleases, base editors, and RNA and DNA-mediated integration systems (including prime editing systems and clustered regularly interspaced short palindromic repeat associated transposases). Metagenomi believes its diverse and modular toolbox positions the company to access the entire genome and select the optimal tool to unlock the full potential of genome editing for patients. For more information, please visit https://​metageno​mi​.co.

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