Metagenomi Unveils Compact SMART Editing Platform at AIChE 7th International Conference on CRISPR Technologies By Investing.com

Poster presentation highlights SMART adenine base editors (ABEs)
small enough to be encapsulated in a single adeno-associated virus (AAV)

The data shows the configuration of the integrated SMART system that enables it
genome editing related to therapy

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

We are very excited to share the progress we have made with our joint SMART initiatives, said Chris Brown, PhD and Head of Discovery (NASDAQ: ) at Metagenomi. Our review highlights the improved efficiency of our SMART platform across multiple targets and cell types. We have shown that our SMART nucleases can be programmed into efficient base editors within the packaging constraints of standard AAV. We believe that effective planning of these SMART programs will allow the pursuit of neuromuscular goals, either individually or collaboratively.

The SMART Metagenomi platform may address a key challenge of gene editing in vivo: the efficient delivery of gene editing components to tissues beyond the liver. Currently, AAV is an established method for extrahepatic tissue delivery, which is necessary for the treatment of 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 material capacity of standard AAV vectors, which may create the need for dual AAV systems, which may reduce overall editing efficiency, increase required doses, and complicate the manufacturing process. Metagenomi's SMART genome editing systems are small enough to be packaged into standard AAV vectors, even if additional effector domains are included for editing, potentially enabling a different therapeutic approach.

Metagenomi has taken a multifaceted approach to developing the SMART platform since its initial discovery (Aliaga Goltsman, Daniela S., et al. Compact… Nature Communications, vol. 13, no. 1), including structure-directed engineering provided energy through collaboration with Professor David Taylor and his lab at UT Austin (Ocampo, Rodrigo Fregoso, et al. “DNA…” bioRxiv). In addition, Metagenomi used advanced Artificial Intelligence (AI) tools trained on SMART natural enzymes, presented at the Cold Spring Harbor Laboratory (CSHL) CRISPR Frontiers conference earlier this year. The AI-driven approach emphasizes the importance of using multiple cutting-edge methods to develop next-generation programming systems.

About the Metagenome
Metagenomi is a precision genetics medicine company dedicated to improving medical treatments for patients using its related, comprehensive toolbox based on metagenomics. Metagenomics uses the power of metagenomics, the study of genes found in the natural environment, to unlock four billion years of bacterial evolution to discover and develop a series of novel editing tools capable of correcting any type of genetic mutation found anywhere in the genome. . A comprehensive genome editing toolbox includes programmable nucleases, base editors, and RNA and DNA integration programs (including core editing programs and short tandem repeat palindromic repeats). Metagenomi believes that its diverse and modular toolbox positions the company to access the entire genome and select the right tool to unlock the full potential of gene editing for patients. For more information, please visit

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Investor Contact:
Simon Harnest – CIO, SVP Investor Relations
simon@‹metagenomi.‹co

Media Contact:
Ashlye Hodge – Communications Manager
ashye@‹metagenomi.‹co

Source: Metagenomi, Inc.