Overall and event-free survival of 100 and 95%, respectively, seen with autologous CD34+ hematopoietic stem-cell lentiviral gene therapy. HealthDay News — Autologous CD34+ hematopoietic stem-cell ...
For decades, scientists have been trying to find ways to cure disorders that can be traced back to an error in one gene. One of the first diseases that researchers ...
MILAN, BOSTON, and LONDON, Sept. 12, 2023 /PRNewswire/ -- Fondazione Telethon, one of the main Italian biomedical charities, and Orchard Therapeutics, a global gene therapy leader, today announced the ...
Over a decade ago, UCLA physician-scientists began using a pioneering gene therapy they developed to treat children born with a rare and deadly immune system disorder. They now report that the effects ...
GAITHERSBURG, Md.--(BUSINESS WIRE)--Leadiant Biosciences, Inc. today announced that the Food and Drug Administration (FDA) has granted approval to Revcovi™ (elapegademase-lvlr) injection in the U.S.
Rarity PBC, a Public Benefit Corporation dedicated to expanding the availability of transformative gene therapies for rare diseases, today announced the closing of a $4.6 million seed financing led by ...
A blood stem cell gene therapy co-developed by UCLA’s Dr. Donald Kohn restored immune function in 59 of 62 children with ADA-SCID, a rare and fatal immune disorder, with no serious complications ...
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