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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
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