Vertex Pharma has teamed up with Orum Therapeutics to develop a conditioning regimen, used to prepare patients to receive gene-editing medicines, that it hopes will offer a more tolerable a
The FDA has lost no time in approving Vertex Pharma and CRISPR Therapeutics pioneering gene-editing therapy Casgevy for its second use, approving the drug for transfusion-
The first-ever CRISPR-based gene-editing therapy has been approved for marketing in the UK for inherited blood disorders sickle cell disease (SCD) and transfusion-dependen