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.

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About PackGene

PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.

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