Beam Therapeutics Reports Initial Clinical Data from Phase 1/2 Trial of BEAM-301, Demonstrating Proof of Concept for Genetic Correction in Glycogen Storage Disease Type Ia (GSDIa) at ESGCT 33rd Annual Congress
Beam Therapeutics reported initial Phase 1/2 data for BEAM-301 in glycogen storage disease type Ia, showing proof of concept for genetic correction of the R83C mutation via a single infusion. In the 40 mg cohort, two of three patients achieved 15 hours of controlled fasting, improved glucose control, and no longer required daily starch supplementation; median time to fasting hypoglycemia rose from 2.5 to 15.0 hours at Day 36. Safety was consistent with LNP-based therapies, with no dose-limiting toxicities or Grade 4/5 events, though four of six patients had serious treatment-related TEAEs including three Grade 3 infusion-related reactions; premedication and infusion regimens were modified.
Key figures
- Drug Name
- BEAM-301
- Phase Of Trial
- Phase 1/2
- Tth 20mg
- median 2.9h baseline to 4.3h at Day 36
- Tth 40mg
- median 2.5h baseline to 15.0h at Day 36
- Patients dosed
- 6
- Starch reduction
- 100% reduction in daily starch supplementation in 2 of 3 patients in 40 mg cohort
- Median followup months
- 7.6
- Controlled fasting hours
- 15 hours achieved by 2 of 3 patients in 40 mg cohort
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