[1/2] A sign hangs in front of the world headquarters of Vertex Pharmaceuticals in Boston, Massachusetts, U.S., October 23, 2019.
REUTERS/Brian Snyder/File Photo Acquire Licensing RightsOct 31 (Reuters) - A panel of advisers to the U.S. health regulator said on Tuesday Vertex Pharmaceuticals (VRTX.O) and CRISPR Therapeutics (CRSP.BN) could assess potential safety risks of their sickle cell disease gene therapy after approval.
If the therapy is approved, Vertex has proposed a 15-year follow up of patients to evaluate the safety outcomes of the therapy.
Patients who were treated with the therapy were free from severe vaso-occlusive crisis for 12 months from the infusion of exa-cel.
"It was made pretty clear that any theoretical off-target editing concerns do not outweigh the benefits of drug approval," said Salim Syed, analyst at Mizuho.
Persons:
Brian Snyder, Scot Wolfe, Salim Syed, Sriparna Roy, Krishna Chandra
Organizations:
Vertex Pharmaceuticals, REUTERS, CRISPR Therapeutics, Staff, U.S . Food, Drug Administration, UMass Chan Medical, Analysts, FDA, Mizuho, Thomson
Locations:
Boston , Massachusetts, U.S, Bengaluru