Today, the FDA approved two gene therapies to treat sickle cell disease, marking the biggest day in the history of gene therapy and a seminal moment in the history of biotechnology and human health.
Casgevy, developed by CRISPR Therapeutics and Vertex Pharmaceuticals, represents the first-ever approval of a gene-editing medicine in the United States (it was previously approved in the United Kingdom and Bahrain). Lyfgenia, developed by bluebird bio, joins Casgevy as the first two gene therapies ever approved in the United States to treat sickle cell disease.
The dual approvals represent durable and potentially curative treatment options for a patient population that has been overlooked for far too long – and one that is the largest to date for a gene therapy. Over 100,000 people in the United States currently live with sickle cell disease, approximately 20,000 of whom are estimated to be eligible for the two therapies.
This incredible moment showcases the power of science and human ingenuity and the culmination of decades of groundbreaking research. Today’s milestone will also pave the way for a coming wave of next-generation gene therapy treatments for a range of diseases, from other rare disorders to cancers.
Casgevy and Lyfgenia represent the fourth and fifth gene therapies for rare genetic diseases approved by the FDA in 2023 alone, doubling the number of such therapies available in just one year.
