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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
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.
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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
Enrollment Count
36
Results Posted
Not Available
Therapy Information
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
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2024-12-06
Completion Date
2027-12-30
Last Update
2024-12-16
Participation Criteria
Eligible Age
>=18 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
1
Locations
United States
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
Dosing anticipated to commence in early 2025
Resources/Links
News and Press Releases
Beam Therapeutics Announces Progress in Hematology and Genetic Disease Franchises and Outlines Key 2025 Anticipated Catalysts
SEC Form 10-K: 2024 Annual Report
Preclinical Publications
Base-editing corrects metabolic abnormalities in a humanized mouse model for glycogen storage disease type-Ia