An influential US drug pricing watchdog raised the price estimate of two experimental gene therapies from Vertex Pharmaceuticals/CRISPR Therapeutics and bluebird bio for sickle cell disease.
Vertex Pharmaceuticals and CRISPR Therapeutics s one-dose gene editing therapy for sickle cell disease would be cost effective if priced at up to $1.9 million, an influential US drug pricing group said on Wednesday.
One year after infusion of a gene-transfer product, patients with the bleeding disorder had significant improvements in clotting factor production and dramatic reductions in factor concentrate use.