Born in Morristown, N.J., with virtually no immune system, Cora was diagnosed with severe combined immunodeficiency, a rare ...

“I remember asking the doctor if my daughter was going to die,” said Chelsea Oakley, mother of Cora Oakley, a newborn who was diagnosed with ADA-SCID when she was just seven days old, in April 2017.

Following a positive opinion from the EMA, the transfer of marketing authorization of Strimvelis from Orchard Therapeutics has been approved by the European Commission Fondazione Telethon will now be ...

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 ...

100% overall survival and ≥95% event-free survival observed at two and three years following one-time treatment withlentiviral HSC gene therapy 50 total participants represent largest published ...

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.

An experimental gene therapy developed to treat children born with a rare immunodeficiency disease has demonstrated extraordinary efficacy according to a new long-term follow-up study published in the ...

Leadiant Biosciences, Inc. today announced that the Food and Drug Administration (FDA) has granted approval to Revcovi ™ (elapegademase-lvlr) injection in the U.S. Revcovi is a new enzyme replacement ...

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 ...

BOSTON and LONDON, May 11, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced data published in the New England Journal of Medicine (NEJM) ...