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Beam Therapeutics Reports Positive Phase 1/2 Data for Gene Editing Treatment BEAM-301

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Beam Therapeutics Inc. (BEAM) announced initial Phase 1/2 trial results for BEAM-301, an investigational base editing therapy for glycogen storage disease type Ia (GSDIa). The data demonstrate clinical proof of concept, showing that a single intravenous dose restored internal glucose production in patients carrying the R83C genetic mutation.

Patients living with GSDIa lack a functional enzyme needed to maintain normal blood sugar, requiring frequent starch intake to prevent severe hypoglycemia. In the study, patients receiving the higher 40 mg dose achieved controlled fasting for at least 15 hours and reduced their daily starch needs by up to 92 percent. BEAM-301 showed an acceptable safety profile consistent with lipid nanoparticle therapies, with no dose-limiting toxicities or treatment dropouts reported.

The company will present updated trial data at the European Society of Gene and Cell Therapy congress on October 28, 2026. Beam Therapeutics Inc. (BEAM) will also host a live investor webcast to discuss the findings on October 27, 2026, at 8:00 a.m. Eastern Time.

Key points

  • Initial Phase 1/2 data show BEAM-301 restored internal glucose production in GSDIa patients after a single treatment.
  • Patients in the 40 mg dose group achieved 15 or more hours of controlled fasting and reduced daily starch intake by up to 92 percent.
  • The therapy showed an acceptable safety profile with no reported dose-limiting toxicities, treatment discontinuations, or deaths.
  • Full trial details will be presented at a medical congress on October 28, 2026, alongside an investor webcast on October 27, 2026.

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Questions and answers

What is BEAM-301 and what does it treat?

BEAM-301 is an in vivo base editing therapy designed to correct the R83C mutation in patients with glycogen storage disease type Ia (GSDIa).

What were the key findings from the BEAM-301 trial?

Patients in the 40 mg cohort achieved at least 15 hours of controlled fasting and reduced daily starch requirements by up to 92 percent following a single dose.

When will Beam Therapeutics present the full trial results?

The company will present updated Phase 1/2 trial data at a medical congress on October 28, 2026, and host an investor webcast on October 27, 2026.