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Beam Therapeutics Presents First Data Highlighting Base Editing Program for Glycogen Storage Disease Type Ia at AASLD
Novel Base Editors for Two Most Common GSDIa Mutations Demonstrate Significantly Higher Levels of In Vivo Mutation Correction than Required to Restore Glucose
About this update from Beam Therapeutics Inc.
[{"type":"text","content":"Novel Base Editors for Two Most Common GSDIa Mutations Demonstrate Significantly Higher Levels of In Vivo Mutation Correction than Required to Restore Glucose Homeostasis\n Previously Presented Data from Alpha-1 Program to be Reviewed in Encore Presentation CAMBRIDGE, Mass., Nov. 13, 2020 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the company will present preclinical data from its liver-focused programs, including the first data highlighting its novel base-editing strategy for correcting disease-causing mutations underlying Glycogen Storage Disease Type Ia (GSDIa), during the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting Digital Experience being held virtually November 13-16, 2020. GSDIa, also known as Von Gierke disease, is an inborn disorder of glucose metabolism caused by mutations in the G6PC gene that disrupt a key enzyme, Glucose-6- Phosphatase (G6Pase), which is involved in glucose homeostasis. This disruption results in low blood glucose levels that can be fatal if patients do not adhere to a strict regimen of slow-release forms of glucose, administered every one to four hours (including overnight). There are currently no pharmacological therapies approved for patients with GSDIa. Beam has engineered novel adenine base editors (ABE) that, in preclinical models, have achieved high levels of precise correction of the two most prevalent GSD1a mutations, R83C and Q347X, in both in vitro and in vivo settings. “Base editing represents an exciting new opportunity for the treatment of serious diseases, including GSDIa, where patients are in need of a disease-modifying, life-long treatment,” said Giuseppe Ciaramella, Ph.D., president and chief scientific officer of Beam. “These first preclinical data from our GSDIa program demonstrate significant levels of precise correction of the disease-causing R83C and Q347X point mutations, well above the level that we believe is needed to have a clinically relevant treatment effect for patients with GSD1a. We look forward to presenting these data, as well as our encore presentation of our Alpha-1 data from ASGCT, as we continue to advance our liver base editing programs toward the clinic.” Details of the GSDIa presentation are as follows:...