NEW YORK, May 9, 2024 /PRNewswire/ -- Epitor Therapeutics,
a biotechnology pioneer in epigenetic editing, announces the
creation of CasNano, a groundbreaking ultracompact Cas protein.
CasNano, comprising less than 400 amino acids, offers an all-in-one
solution for AAV packaging, compatible with a diverse range of
protein effectors to control gene expression through epigenetic
modifications to activate or repress gene transcription, and enable
multiplexed editing. The company unveiled new data during the 27th
American Society of Gene & Cell Therapy (ASGCT) Annual Meeting,
showcasing over a 20-fold improvement in targeting efficiency via
directed evolution of CasNano and innovative re-engineering of the
tracrRNA component of the guide RNA.
"Our revolutionary Cas protein, CasNano, was meticulously
engineered utilizing protein language model-assisted analysis and
multiple rounds of saturated amino acid mutagenesis," stated lead
scientist, Dr. Woofung (Yi-Ting)
Tsai. "In addition to its nuclease activity, CasNano can
efficiently demethylate, methylate, and activate target genes when
catalytically deactivated, with protein effectors attached. Its low
tolerance to spacer mismatches supports greatly reduced off-target
effects."
"CasNano is 70% smaller than spCas9 and has been engineered to
optimize DNA targeting," added Epitor CEO, Dr. Megan McGill. "CasNano's compact size stands out
as the only Cas variant capable of being packaged alongside large
protein effectors, such as TET, within a single AAV for targeted
demethylation of gene promoters." We believe this size
advantage will help solve the longstanding delivery challenge that
has previously hampered many CRISPR/Cas based development
programs.
Epitor is advancing CasNano development for therapeutic
epigenetic modifications in disorders with high unmet medical
need.
About Epitor Therapeutics
Epitor Therapeutics is a leading epigenetic editing company
dedicated to curing diseases by altering pathologic gene expression
without direct editing or damage to the genome. This innovative
approach enables the therapeutic activation of necessary genes or
inhibition of toxic gene expression. Leveraging ultracompact tools,
Epitor unlocks delivery to diverse tissues to treat diseases
through precise control of gene expression. The company's
internal programs focus on X-reactivation and nucleotide repeat
disorders.
Contact:
Patricia Tolete, Director of
Operations
Email: patricia.tolete@epitortx.com
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SOURCE Epitor Therapeutics