Beam Therapeutics reports early Phase 1/2 data for base editing therapy in GSD Ia, partners with Pfizer and Enodia

Beam Therapeutics disclosed early results from a Phase 1/2 study evaluating a base editing approach aimed at correcting the genetic defect that causes glycogen storage disease type Ia, a rare metabolic disorder.

The initial data indicate that the therapy can edit the target mutation in patient cells, with early signals of biochemical improvement, although the study is still ongoing and safety data are limited.

Beam also announced a collaboration with Pfizer and Enodia to advance the program, leveraging Pfizer's development expertise and Enodia's platform to move the candidate toward later stage testing.