Treatment with exagamglogene autotemcel (exa-cel) led to robust and sustained improvements in quality of life for patients ...

He had just become the first patient to receive a gene therapy for sickle cell since it was approved — a therapy that is expected to free him from the ravages of the disease.

From birth to gene-edited in 6 months: Custom therapy breaks speed limits The N-of-1 accomplishment provides a template for swift, personalized genetic therapies.

A Georgetown woman with sickle cell disease is looking for a new lease on life from a gene-editing therapy being tested at Medical University of South Carolina.

Vertex Pharmaceuticals Inc and CRISPR Therapeutics AG's one-dose gene editing therapy for sickle cell disease would be cost effective if priced at up to $1.9 million, an influential U.S. drug ...

The FDA on Friday also approved a second treatment for sickle cell disease, called Lyfgenia, a gene therapy from drugmaker Bluebird Bio.

The personalized CRISPR treatment could be the future of gene therapy, but hurdles remain before everyone has access.

A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for him.

Researchers hope to test the product's ability to clear symptoms in patients with the rare genetic disorder driven by RPS19 ...