Recent Updates
Recently added Catalysts

BEAM-301

Phase 1

Glycogen Storage Disease Type Ia | Small molecule | Rare Disease |Beam Therapeutics Inc.|Last Updated: Dec 15, 2025

Target and mechanism

ModalitySmall molecule

Also known as BEAM-301: Single dose of BEAM-301 administered by IV, BEAM-301: Single dose of BEAM-301 administered by

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment36

FDA Designations

No designations recorded

Clinical trial landscape

BEAM-301 · 1 trial · 1 indication

Phase 1 1
NCT06735755A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa)Glycogen Storage Disease Type Ia
RECRUITING36 Analytics
PHASE1RECRUITING
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)
Glycogen Storage Disease Type Ia
NCT0673575536
Unlock trial analytics

Study Endpoints

Primary Endpoints

To evaluate the safety and tolerability of BEAM-301 in patients with GSDIa with a G6PC1 c.247C>T (p.R83C) variant by measuring incidence of treatment-emergent adverse events (TEAEs) including serious adverse events (SAEs) and dose-limiting toxicities (DL
BEAM-301 administration through month 24

Secondary Endpoints

Absolute and change from baseline in time to hypoglycemia or metabolic decompensation during a controlled fasting challenge
Baseline to month 24
Changes from baseline in starch supplementation dose
Baseline to month 24
Level of serum glucose and metabolic parameters (lipid profile and uric acid) over time
Baseline to month 24
Unlock Study Endpoints

Study Design & Arms

AllocationNA
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
BEAM-301EXPERIMENTALBEAM-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.

Interventions

NameTypeDescription
BEAM-301: Single dose of BEAM-301 administered by IVDRUGBEAM-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.
Unlock Study Design Details

Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites3

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 ...

Countries:United States
Unlock Eligibility Criteria