The Food and Drug Administration recently approved gene therapy to treat sickle cell disease among children ages 2 and older, ...
Learn how gene editing is transforming sickle cell disease treatment and why expanding access to younger children matters.
A new treatment for children aged 2 or older with sickle cell disease has been approved by the U.S. Food & Drug ...
A new study describes a gene therapy strategy that uses the brain's own glymphatic transport system to distribute engineered viral vectors throughout the brain.
Stem cell transplantation (also called bone marrow transplantation) and gene therapy are among the most powerful curative ...
The National Institute on Aging of the National Institutes of Health has awarded a five-year grant initially worth $8.37 ...
The firm announced it has aligned with the US Food and Drug Administration on the design of the Phase III study, which could support a biologics license application.
The 500,000-square-foot facility, designed as a global gene therapy manufacturing complex, spans a full city block along ...
Genespire, in collaboration with researchers at the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), has today ...
An 11-year-old boy from Türkiye is receiving care for Elevidys, a one-time gene therapy for Duchenne Muscular Dystrophy (DMD) ...
Fast, local cryopreservation is a consistent bottleneck for autologous cell therapy developers, even in major cell and gene therapy hubs.
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