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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) Homozygous or Compound Heterozygous for the G6PC1 C.247C>T (p.R83C) Variant

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Trial Profile

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) Homozygous or Compound Heterozygous for the G6PC1 C.247C>T (p.R83C) Variant

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 23 Dec 2024

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At a glance

  • Drugs BEAM 301 (Primary)
  • Indications Glycogen storage disease type I
  • Focus Adverse reactions
  • Sponsors Beam Therapeutics

    • Most Recent Events

    • 23 Dec 2024 New trial record

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