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)

NCTID	NCT06735755 (View at clinicaltrials.gov)
Description	This is a Phase 1/2, multicenter, open-label, single-ascending-dose study to evaluate the safety, tolerability, and efficacy of BEAM-301 in adult patients with GSDIa homozygous or compound heterozygous for the G6PC1 c.247C\>T (p.R83C) variant and to determine the optimal biological dose. (Show More)
Development Status	Active
Indication	Glycogen Storage Disease Type Ia
Disease Ontology Term	DOID:2749
Compound Name	BEAM-301
Sponsor	Beam Therapeutics Inc.
Funder Type	Industry
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	36
Results Posted	Not Available

Target Gene/Variant	G6PC1 (p.R83C)
Therapy Type	Gene editing
Therapy Route	In-vivo
Mechanism of Action	Mutation correction
Route of Administration	Intravenous
Drug Product Type	MRNA, LNP
Target Tissue/Cell
Delivery System	Lipid encapsulation
Vector Type	LNP
Editor Type	ABE
Dose 1	Undisclosed dose escalation
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States

Has US IND	True
FDA Designations
Recent Updates	Dosing anticipated to commence in early 2025