Beam Therapeutics Presents Preclinical Data Highlighting Utility and Durability of BEAM-301 to Correct a Glycogen Storage Disease Type I Deficiency Disease-Causing Mutation at ESGCT
Retrieved on:
Wednesday, October 25, 2023
Gene editing, Survival, FDA, Uric acid, DSM-IV codes, IND, Therapy, Glycogen storage disease, Biotechnology, The First Half of My Life, Congress, Liver, Kidney, Systemic administration, G6PC, Mouse, Hypoglycemia, Cell, Risk, Gene, Triglyceride, LNP, ESGCT, Glucose, European Society for Medical Oncology, Patient, Blood, Cholesterol, Coma, Pharmaceutical industry, Vaccine, Medical imaging, Silviculture, Beam
CAMBRIDGE, Mass., Oct. 25, 2023 (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 in vivo drug candidate, BEAM-301, to directly correct the R83C mutation, one of the primary disease-causing mutations of glycogen storage disease type Ia (GSDIa). The data were presented today in an oral presentation titled “A Single, Systemic Administration of BEAM-301 Mitigated Fasting Hypoglycemia One Year after Dosing in a Transgenic Mouse Model of Glycogen Storage Disease Type-Ia” at the 30th Annual European Society of Gene & Cell Therapy (ESGCT) Congress in Brussels.
Key Points:
- “GSDIa is a devastating disease that significantly impacts an individual’s quality of life and puts them at consistent risk of hypoglycemia, coma and potentially death.
- BEAM-301 is a liver-targeting lipid-nanoparticle (LNP) formulation containing base editing reagents optimized to correct the R83C mutation.
- These findings support the potential of BEAM-301 to directly correct the disease-causing R83C mutation with a single dose.
- Beam plans to submit a U.S. IND application in the first half of 2024 for authorization to initiate clinical trials of BEAM-301.