Mutations in the F9 gene cause factor IX (FIX) deficiency, leading to severe bleeding in people with hemophilia B. Current therapies rely on frequent FIX infusions or newer agents that rebalance ...
CLEVELAND, Dec. 08, 2025 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced the first commercial patient treatment with FDA-approved ZEVASKYN (prademagene zamikeracel), a ...
An investigational gene therapy showed sustained clinical efficacy for children born with ADA-SCID. In a study of 62 children, overall survival after treatment was 100% and event-free survival was 95% ...
CLEVELAND, Oct. 30, 2025 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (ABEO), a commercial-stage biopharmaceutical company developing cell and gene therapies for serious diseases, today announced that ...
This week marked the first time a patient with recessive dystrophic epidermolysis bullosa (RDEB) received prademagene zamikeracel (pz-cel, Zevaskyn), a first-of-its-kind autologous gene therapy to ...
BOSTON and LONDON, April 27, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the first patient has been dosed in an open-label, ...
AGC Biologics Partners with Rarity PBC to Advance Life-Saving Gene Therapy for "Bubble Baby Disease"
AGC Biologics Partners with Rarity PBC to Advance Life-Saving Gene Therapy for "Bubble Baby Disease" Collaboration will leverage AGC Biologics’ commercial manufacturing experience and proven platforms ...
Cell and gene therapies are transforming modern medicine by enabling targeted treatments that use living cells or genetic ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results