A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated children, offering a lasting, donor-free solution to ADA-SCID, known as the ...
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 ...
"Strimvelis returns home." With these words, Alessandro Aiuti, MD, PhD, head of the Pediatric Immunohematology Unit at the Scientific Institute for Research and Healthcare (IRCCS) San Raffaele ...
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 ...
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 ...
Adenosine deaminase (ADA) deficiency is a rare autosomal recessive disorder that underpins a severe form of combined immunodeficiency (SCID), resulting in the accumulation of toxic metabolites that ...
DUBLIN – Bidding to become the second European firm to win a gene therapy approval, Glaxosmithkline plc filed a marketing authorization application (MAA) with the EMA for GSK2696273, a treatment for ...
FRIDAY, Oct. 17, 2025 (HealthDay News) -- Autologous CD34+ hematopoietic stem-cell lentiviral gene therapy shows sustained clinical efficacy for severe combined immunodeficiency (SCID) due to ...
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