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Beam Therapeutics Announces Preclinical Data Highlighting Base Editing Approach to Correct a Glycogen Storage Disease Type Ia Disease-Causing Mutation
Findings Show Base Editing Leads to Elimination of Disease Phenotype in an In Vivo Model CAMBRIDGE, Mass., Oct. 19, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics
About this update from Beam Therapeutics Inc.
[{"type":"text","content":"Findings Show Base Editing Leads to Elimination of Disease Phenotype in an In Vivo Model\nCAMBRIDGE, Mass., Oct. 19, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported new preclinical data demonstrating the ability of its liver-targeted base editing approach to directly correct R83C, one of the primary disease-causing mutations of glycogen storage disease type Ia (GSDIa). The data will be presented at the European Society of Gene and Cell Therapy (ESGCT) 2021 Virtual Congress in an oral presentation at 9:45AM (EST) / 3:45PM (CEST) on Wednesday, October 20, 2021, during the Liver & Metabolic Disease I Session 3a. “The data we will present at ESGCT highlight the continued innovation of our in vivo, liver-targeted base editing approach for GSDIa, which aims to directly correct R83C, a highly prevalent disease-causing mutation in this patient population,” said Giuseppe Ciaramella, Ph.D., president and chief scientific officer of Beam. “These findings showcase the exceptional work of our team of scientists, who created a novel, humanized GSDIa R83C mouse model. When treated with our base editor, we directly corrected the human gene by editing a single base in the gene, without creating double-stranded breaks in DNA, and restored normal glucose metabolism in these animals. These data demonstrate robust preclinical proof of concept for the treatment of GSDIa and add to the significant collection of research findings we have generated supporting the promise of base editing to address a wide range of diseases.” GSDIa is an autosomal recessive disorder caused by mutations in the G6PC gene that disrupt a key enzyme, glucose-6- phosphatase (G6Pase), which is involved in maintaining glucose homeostasis. Inhibition of G6Pase activity results in low fasting blood glucose levels that can be fatal. To mitigate fasting hypoglycemia, GSDIa patients must adhere to a strict regimen of slow-release forms of glucose, administered every one to four hours, including overnight. Beam’s approach to treating GSDIa is to deliver an adenine base editor (ABE) via lipid nanoparticle (LNP) to the liver to repair the G6PC-p.R83C mutation. To evaluate its approach, Beam created a novel, humanized R83C knockout mouse model (huR83C), mimicking the abnorma...