Drugs / BEAM-301
last change Dec 2024 re-read 3 minutes ago

BEAM-301

targets G6PC1 via restoration

Developed for
glycogen storage disease due to glucose-6-phosphatase deficiency type IA
Investigated by
Beam Therapeutics Inc.

Trials 1

2025202620272028
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/2 NCT06735755 Dec 2024 → Dec 2027 expected glycogen storage disease due to glucose-6-phosphatase deficiency type IA Beam Therapeutics Inc. Recruiting No outcome recorded

News releases announcing trial results or a regulatory action · 3

DateIssuerRelease
2024-08-06 Beam Therapeutics Inc. Regulatory Beam Therapeutics Reports Pipeline Updates and Second Quarter 2024 Financial Results beamtx.com ↗
In our genetic disease franchise, we’re pleased to announce the clearance of our U.S. investigational new drug (IND) application for BEAM-301, our first U.S. in vivo regulatory filing.
2023-10-25 Beam Therapeutics Inc. Results Beam Therapeutics Presents Preclinical Data Highlighting Utility and Durability of BEAM-301 to Correct a Glycogen Storage Disease Type I Deficiency Disease-Causing Mutation at ESGCT beamtx.com ↗
today reported new preclinical data demonstrating the ability of its in vivo drug candidate, BEAM-301, to directly correct the R83C mutation, one of the primary disease-causing mutations of glycogen storage disease type Ia (GSDIa).
2022-05-02 Beam Therapeutics Inc. Results Beam Therapeutics to Present New Preclinical Data from Base Editing Pipeline at ASGCT 25th Annual Meeting beamtx.com ↗
Single, systemic administration of BEAM-301 mitigates fasting hypoglycemia and restores metabolic function

Evidence & citations 4 cited values

Every value below carries the sentence it was read from. 1 source stands behind the page.

FieldValueCited text
Known as BEAM-301 ClinicalTrials.gov intervention name — accepted as the source's own label NCT06735755 ↗
Action Restore “resulting in restoration of G6Pase-α catalytic activity” NCT06735755 ↗
Route Intravenous “for intravenous (IV) administration” NCT06735755 ↗
Target G6PC1 “BEAM-301 is designed to correct the G6PC1 c.247C\>T allele” NCT06735755 ↗