Beam Therapeutics to Present Initial Data from Phase 1/2 Trial of BEAM-301 in Glycogen Storage Disease Type Ia (GSDIa) at the ESGCT 33rd Annual Congress
Beam Therapeutics announced initial Phase 1/2 data for BEAM-301 in glycogen storage disease type Ia, achieving clinical proof of concept for base-editing correction of the R83C mutation. Both evaluable 40 mg patients achieved the protocol-defined maximum of ≥15 hours of controlled fasting, consistent with restored endogenous glucose production, and starch intake fell 71-92% in that cohort. Safety was consistent with LNP-based therapies, though three Grade 3 infusion-related reactions occurred; the steroid premedication regimen has since been optimized and no dose-limiting toxicities or deaths were reported.
Key figures
- Drug Name
- BEAM-301
- Phase Of Trial
- Phase 1/2
- Grade3 irrs
- 3
- Cohort doses mg
- [20,40]
- Patients treated
- 6
- Day36 median tth 20mg
- 4.3 hours
- Starch reduction 20mg
- ["74% (Day 128)","71% (Day 227)","11% (Day 387)"]
- Starch reduction 40mg
- ["71% (Day 50)","92% (Day 93)"]
- Controlled fasting 40mg
- ≥15 hours
- Baseline median tth 20mg
- 2.9 hours
- Baseline median tth 40mg
- 2.5 hours
- Median followup days 20mg
- 313
- Median followup days 40mg
- 53
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