Metagenomi Presents Compact SMART Editing Platform at AIChE 7th International Conference on CRISPR Technologies
15 10월 2024 - 9:58AM
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 MetagenomiMetagenomi 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://metagenomi.co.
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Investor Contact:Simon Harnest - CIO, SVP
Investor Relationssimon@metagenomi.co
Media Contact:Ashlye Hodge - Communications
Managerashlye@metagenomi.co
Metagenomi (NASDAQ:MGX)
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