No patients have received Casgevy, CRISPR Therapeutics and Vertex Pharmeceuticals’ recently approved sickle cell gene therapy ...

In the meantime current gene therapies remain expensive and challenging to scale up because each patient’s batch of cells has ...

Illuminating the experience of people living with sickle cell could improve patients’ lives and enhance all of medicine ...

Kendric Cromer, 12, is among the first patients to be treated with gene therapy just approved by the F.D.A. that many other ...

Despite the promise, the therapy developed by Vertex Pharmaceuticals ... Unlike most of the body’s cells, embryonic stem cells — which are created from human embryos — can give rise to ...

1. What is the term for a transplantation procedure in which cells or organs are transferred between individuals belonging to ...

Researchers at multiple AViDD centers confirm that the NIH has offered a 1-year, no-cost extension, but it doesn’t come with ...

Vertex Pharma has made its name ... The therapy takes the form of stem cell-derived human pancreatic islet cells, which secrete insulin in the body in response to rising blood glucose.

Aardvark Therapeutics, Inc., a biopharmaceutical company, announced the appointment of Manasi Sinha Jaiman, M.D., M.P.H. as Chief Medical Officer and Tom Bicsak, Ph.D., as Head of Regulatory Affairs ...

such as chelation drugs to remove excess iron from the body. Haematopoietic stem cell transplantation (HSCT) offers a chance of a cure, but is only possible when a donor with a matching human ...