Shape Therapeutics RNAfix® Editing Platform Achieves >90% Editing in Mouse and Non-Human Primate Brain
May 08 2024 - 9:31AM
Shape Therapeutics Inc. (ShapeTX), a leader at the convergence of
AI and RNA editing to revolutionize genomic medicine, today
presented data at the 27th American Society for Gene and Cell
Therapy (ASGCT) Annual Meeting in Baltimore, MD showcasing the high
therapeutic potential of its RNAfix platform in for the treatment
of central nervous system (CNS) disorders.
The Company will be releasing the data in an oral presentation
titled “>95% targeted RNA editing in total brain with high
durability and specificity enabled by systemic injection of
AAV-packaged ADAR guide RNAs.” The presentation will highlight
best-in-class RNA editing efficiency in vivo in mouse, non-human
primate (NHP) and in human neuronal model systems. Potential
therapeutic applications of RNAfix will be highlighted in Rett
syndrome, Parkinson’s disease, and Alzheimer’s disease, showcasing
a diversity of therapeutic mechanisms enabled by nucleotide-level
precision RNA editing. A superior safety profile will also be
presented, with no significant off-target editing across the entire
transcriptome. These findings demonstrate a breakthrough in the
potential for translating RNAfix into human clinical studies and
represent significant advancements in the field of genomic
medicine.
David Huss, PhD, Chief Scientific Officer at ShapeTX commented,
“RNAfix is efficient, specific, durable, and tunable. The data
presented at ASGCT highlight levels of editing in the CNS
unparalleled by any other editing technology, unlocking vast
therapeutic opportunities. Importantly, the RNAfix platform leaves
genomic DNA intact and does not require the delivery of foreign
proteins with immunogenicity risks. These significant scientific
advancements provide a clear path forward and give us confidence as
we build transformative therapies for patients in need.”
About ShapeTXShape Therapeutics operates at the
convergence of AI and RNA editing to revolutionize genomic
medicine. Our proprietary RNA editing technology, RNAfix®,
represents a significant leap forward over CRISPR-based tools,
achieving 95% editing efficiency in vivo, and a path to one-time
curative treatments for thousands of diseases. With over 90 million
laboratory-validated guide RNA and 12 billion NHP-derived AAV data
points, ShapeTX is building the largest therapeutic RNA foundation
model in the industry. Having secured strategic collaborations
worth over $4 billion with industry-leading pharmaceutical firms,
ShapeTX is poised for substantial growth and to cement our position
as a leader in AI for genomic medicine. You can find us
at ShapeTX.com and on LinkedIn and X.
Contact:Lisa Taylor Ashlisa@shapetx.com