A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa)

Inclusion criteria

• Males or females age ≥18 years of age at the time of consent.
• Diagnosis of GSDIa and homozygous or compound heterozygous for the G6PC1 c.247C>T (p.R83C) variant (confirmed by genetic testing).
• History of at least 1 episode of hypoglycemia <60 mg/dL within the 2 years prior to signing the ICF.

Exclusion criteria

• Liver transplant recipient/on waiting list for liver transplant or known history of liver cirrhosis.
• Presence of liver adenoma >5 cm in size based on liver MRI or liver ultrasound performed at screening or within 6 months prior to screening.
• Presence of liver adenoma >3 cm and ≤5 cm with a documented annual growth rate of ≥0.5 cm per year.
• Have aspartate transaminase or alanine transaminase >upper limit of normal (ULN).
• Total bilirubin levels >ULN; if documented Gilbert's Syndrome, total bilirubin >2 × ULN.
• Previous treatment with any cell-, gene-, or viral vector-derived therapy Hospitalization for management of hypoglycemia within 4 weeks prior to signing the ICF.
• Have triglycerides >1000 mg/dL or a history of pancreatitis or a history of acute pancreatitis within 5 years that does not have a known and corrected etiology (eg, cholecystectomy for gallstone pancreatitis).