Scribe Therapeutics to present at the 43rd Annual J.P. Morgan Healthcare Conference.

ALAMEDA, Calif.--(BUSINESS WIRE)--Scribe Therapeutics Inc. (Scribe), a genetic medicines company unlocking the potential of CRISPR to transform human health, presented data on its X-Editor (XE) and Epigenetic Long-Term X-Repressor (ELXR) technologies at the 27th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting taking place in Baltimore, MD, and virtually.The data described in the company’s oral presentations and poster session showcase the dramatic potency, safety, and specificity of the novel XE genome editing and ELXR epigenetic editing platforms and the company's developments in successfully advancing these technologies into large animal models, including non-human primates.“ A major challenge in the field has been to achieve saturating levels of genome editing, at therapeutically relevant doses, with molecules that have better safety and delivery characteristics than CRISPR-Cas9—this has not been possible to date,” said Benjamin Oakes, Ph.D., co-founder and Chief Executive Officer of Scribe. “ For the first time, Scribe has demonstrated that the holistic engineering of a wholly unique and highly potent CRISPR enzyme can enable this goal. We have demonstrated that our XE platform can achieve editing rates of greater than 75% in the liver of NHPs, theoretically targeting all hepatocytes, while not displaying any detectable off-target effects in primary human cells.”“ Furthermore, we were thrilled to unveil a taste of the engineering behind Scribe’s ELXR epigenetic editing platform and present data elucidating the effectiveness of our comprehensive engineering approach compared to previous methodologies,” Dr. Oakes said. “ We believe this positions Scribe as the only company to have engineered, from the ground up, novel CRISPR genome editing and epigenetic editing platforms that can meet and often exceed the field’s expectations regarding therapeutically relevant potency and specificity.”Additional highlights on the developments of the XE platform and application in vivo include:Presentation: Engineering CasX to Create a Gene Editor with Potent Activity in Non-Human PrimatesUsing a variety of holistic engineering approaches, Scribe has created X-Editor molecules that are >100 steps in sequence space removed and >100-fold more potent than the naturally occurring CRISPR predecessorsXE achieves at or near-saturating levels of editing across a broad range of tissue types, including the eye, CNS, cardiac and skeletal muscle, and liver using either AAV or LNPXE is highly potent in non-human primate studies, achieving >75% editing in the liver and saturating editing at <1.5mg/kgWhen tested at different sites, XE is highly specific, demonstrating no detectable off-target editing at doses 10-fold higher than the effective dose in primary human hepatocytesScribe continues to expand the XE platform by engineering molecules with customizable genomic targetability by modifying the PAM recognition of the XE platformPresentation: AAV-Mediated Delivery of a Novel CasX-Editor Molecule Achieves Allele-Specific and Potent Editing of P23H Rhodopsin in a Mouse Model of Retinitis PigmentosaXE and AAV cargo engineering enables safe and selective XE delivery and editing in rod photoreceptors when administered subretinallyScribe demonstrates for the first time the use of XE to achieve allele-specific editing of the P23H.RHO locus, in an in vitro system and a phenotypically relevant in vivo model