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Also known as BEAM-301: Single dose of BEAM-301 administered by IV
BEAM-301 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| BEAM-301 | EXPERIMENTAL | BEAM-301 consists of adenine base editor messenger ribonucleic acid (mRNA) and single guide ribonucleic acid (gRNA) formulated in lipid nanoparticles (LNPs) for intravenous (IV) administration. |
| Name | Type | Description |
|---|---|---|
| BEAM-301: Single dose of BEAM-301 administered by IV | DRUG | BEAM-301 is designed to correct the G6PC1 c.247C\>T allele via an A:T-to-G:C base-pair substitution, resulting in restoration of G6Pase-α catalytic activity. |
Inclusion Criteria: 1. Males or females age ≥18 years of age at the time of consent. 2. Diagnosis of GSDIa and homozygous or compound heterozygous for the G6PC1 c.247C\>T (p.R83C) variant (confirmed by genetic testing). 3. History of at least 1 episode of hypoglycemia \<60 mg/dL within the 2 years ...
BEAM-301 is an investigational small molecule developed by Beam Therapeutics Inc. (ticker: BEAM) for the treatment of Glycogen Storage Disease Type Ia (GSDIa), a rare inherited metabolic disorder. It is being studied as a single dose administered by intravenous (IV) infusion and remains in clinical development.
BEAM-301 is being developed to treat Glycogen Storage Disease Type Ia (GSDIa), a rare disease caused by impaired glucose metabolism in the liver. The Phase 1/2 study enrolls adults 18 years and older with GSDIa in the United States. It is investigational and has not been approved for any use.
BEAM-301 targets the R83C mutation, a specific disease-causing mutation associated with Glycogen Storage Disease Type Ia. By addressing this mutation, the small molecule is designed to act on the underlying cause of GSDIa rather than only managing symptoms.
BEAM-301 is being developed by Beam Therapeutics Inc., which trades on the Nasdaq under the ticker BEAM. The company is running the clinical program for the asset in Glycogen Storage Disease Type Ia.
BEAM-301 is in Phase 1 clinical development. It is investigational and has not been approved by the FDA or any other regulatory agency. The ongoing study is a Phase 1/2 dose-exploration trial evaluating safety and efficacy in patients with Glycogen Storage Disease Type Ia.
BEAM-301 is being studied in NCT06735755, a Phase 1/2 dose-exploration study evaluating its safety and efficacy in patients with Glycogen Storage Disease Type Ia. The trial is currently recruiting, plans to enroll 36 participants, is uncontrolled, and is conducted in the United States with adults 18 years and older.
Yes. BEAM-301 is also referred to as a single dose of BEAM-301 administered by IV. These names describe the same investigational small molecule from Beam Therapeutics being studied in Glycogen Storage Disease Type Ia, given as one intravenous dose rather than a repeated regimen.