News at a glance: New cancer institute head, NIH ’s applied research tilt, and bias-free clinical algorithms

BIOMEDICINE U.K. approves CRISPR therapy for sickle cell In a world first, U.K. regulators last week approved a therapy that uses CRISPR, the Nobel Prize–winning gene-editing tool invented in 2012. The treatment has been shown to help people with beta thalassemia and sickle cell disease, both inherited blood disorders that involve defects in the oxygen-carrying protein hemoglobin. It relies on removing blood stem cells from patients, using CRISPR to turn on the gene for a fetal form of hemoglobin, then reinfusing the cells. The therapy, developed by the companies Vertex Pharmaceuticals and CRISPR Therapeutics, has been tested in clinical trials with dozens of patients. Almost all those with sickle cell stopped having debilitating pain “crises,” and most beta thalassemia patients were able to forgo the blood transfusions they previously needed. The treatment is expected to cost at least $2 million, raising questions about whether the United Kingdom’s National Health Service and U.S. insurance companies will cover it. U.S. regulators are expected to approve the therapy for sickle cell disease by 8 December and for beta thalassemia by 30 March 2024. BIOSAFETY House OKs gain-of-function ban Although it may never become law, the U.S. House of Representatives last week approved an amendment that would ban National Institutes of Health (NIH) funding for studies that might make an actual or possible human pathogen more...
Source: Science of Aging Knowledge Environment - Category: Geriatrics Source Type: research