A common cold was enough to kill Cora Oakley. Born in Morristown, New Jersey, with virtually no immune system, Cora was diagnosed with severe combined immunodeficiency, a rare genetic condition that ...
Risto-cel, a gene-edited cell therapy formerly called BEAM-101, for severe SCD, benefits patients for nearly two years, new ...
Dr. Teresa Blankmeyer Burke is spending the fall semester in Florianópolis, a city in southern Brazil, to study the ethics of ...
ORLANDO -- A gene therapy that could transform CAR T-cell treatment achieved rapid and potentially durable measurable ...
Researchers at the University of Geneva, together with colleagues in Switzerland, France, the United States and Israel, ...
The National Academy of Inventors (NAI) today released the list of newly inducted fellows, and two Duke researchers have been ...
Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced activation of The University of Texas Medical Branch (UTMB) in ...
A cutting-edge therapy using base-edited immune cells is offering a major breakthrough for patients with one of the toughest ...
An experimental gene therapy treatment created at UCLA gave a "bubble girl" born with a rare genetic disorder a new life.
From rare diseases to chronic conditions, epigenetic editing unlocks new ways to control gene activity without altering the genetic code.
ROME, Italy, Dec. 10, 2025 /PRNewswire/ -- Fondazione Telethon announced today that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for Waskyra, an ex ...
Aradigm Health, a new benefits platform aimed at easing the financial sting of coverage for cell and gene therapies, has ...