Two Gene Therapies for Sickle Cell Disease Get FDA Nod, Including First Ever CRISPR-Based Therapy

Inside Precision Medicine December 11, 2023
Malorye Branca

In a landmark moment for sickle cell disease treatment and CRISPR, two gene therapies were approved for the condition by the U.S. FDA. One of them,Vertex and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel [exa-cel]), is the first ever FDA approved CRISPR/Cas9 genome-edited cell therapy. Simultaneously, Bluebird Bio received the FDA OK for Lyfgenia, its gene therapy for sickle cell.

Bluebird said their treatment would come with a $3.1 million price tag, compared with the $2.2 million price for Casgevy. This approval also marks Bluebird’s third ex vivo gene therapy approved by the FDA for a rare genetic disease and second FDA approval for an inherited hemoglobin disorder.

Last month, the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) was first to...

Today's Sponsors

Today's Sponsor