Beam Therapeutics has initiated a clinical development program in glycogen storage disease type 1a.  Beam is using base editing to potentiall correct the causative genetic change in G6PC: initially the R83C variant, which is the most common mutation in GSD1a.  Base editing is an emerging class of precision genetic medicines designed to overcome the limitations of existing approaches and expand the potential of genetic medicine.  By rewriting a single base, base editors may correct disease-causing point mutations and potentially create life-long cures for patients suffering from serious diseases. Click below to read about their preclinical data.

Ultragenyx Pharmaceuticals is evaluating DTX401 to establish normal glucose metabolism and reduce or eliminate the need for cornstarch to maintain normal glucose levels.  The company is currently in phase 3 of clinical trials.