Lysosomal Disorders

Around the Helix: Gene and Cell Therapy Company Updates - January 19, 2022

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in gene and cell therapies.

FDA Extends BLA Review for bluebird bio’s β-Thalassemia and CALD Gene Therapies

Eli-cel remains on clinical hold in cerebral adrenoleukodystrophy after a report of persistent anemia.

Fabry Disease Gene Therapy Shows Efficacy in First 2 Patients

The first patient with Fabry disease dosed with FLT190 2 years ago continues to have elevated, sustained α-Gal A.