Both Vertex Pharmaceuticals Inc.’s Casgevy (exagamglogene autotemcel, exa-cel) and Bluebird Bio Inc.’s Lyfgenia (lovotibeglogene autotemcel, lovo-cel) received U.S. FDA approval Dec. 8, providing 16,000 American sickle cell patients who have recurring vaso-occlusive events with access to the first cell-based gene therapies.
With the number of high-priced cell and gene therapies expected to grow dramatically over the next decade, U.S. Sen. Bill Cassidy (R-La.) is asking for input from patients, doctors, manufacturers, payers and policy wonks on how to pay for the treatments and ensure patient access when they become more mainstream across the ultra-rare disease spectrum.
Arriving on the gene therapy scene with an undisclosed seed funding sum, Alveogene is tackling respiratory diseases with high unmet need via a next-generation lentiviral delivery platform to advance into the clinic a candidate for rare inherited disorder alpha-1 antitrypsin deficiency.
