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BEAM-301

Glycogen Storage Disease 1a (GSD1a)

PreclinicalIND-enabling

Key Facts

Indication
Glycogen Storage Disease 1a (GSD1a)
Phase
Preclinical
Status
IND-enabling
Company

About Beam Therapeutics

Beam Therapeutics is a clinical-stage biotechnology company developing a new class of precision genetic medicines based on its foundational base editing technology. The company's approach allows for the correction of point mutations, insertion or deletion of single bases, and modulation of gene expression with high efficiency and specificity. Beam is advancing a diversified pipeline of in vivo and ex vivo programs targeting sickle cell disease, alpha-1 antitrypsin deficiency, glycogen storage disease 1a, and various cancers. The company has established strategic collaborations with leading pharmaceutical partners and went public in February 2020.

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