The cell and gene therapy sector is poised to deliver a wave of new therapies with the potential to cure rare and common diseases. As many as 13 new cell or gene therapies could be approved for use in the U.S., Europe, or both by the end of 2023. While manufacturing and regulatory challenges remain,…
Hemophilia gene therapy Hemgenix sets record for world’s most expensive drug
FDA has approved CSL Behring’s (ASX: CSL) Hemgenix (etranacogene dezaparvovec), an adenovirus-associated virus–based gene therapy for adults with hemophilia B. The price tag for the gene therapy — the first for hemophilia B — is $3.5 million. The drug is administered as a single infusion. Hemophilia B is a genetic bleeding disorder caused by missing or…