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% ...
Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the long-term safety and efficacy of the curative treatment, in a study led by ...
An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has restored and maintained immune system function in 59 of 62 children born ...
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 ...
ORLANDO, Fla. — Exagamglogene autotemcel (exa-cel, Casgevy), a CRISPR/Cas9 gene-editing therapy, showed efficacy in children aged 5-11 years with transfusion-dependent beta-thalassemia (TDT) or sickle ...
A gene therapy study published recently in the journal Nature Medicine showed promising results in restoring hearing in patients of all ages with genetic hearing loss and congenital deafness. The ...
What Is Itvisma, and Why Does It Matter? Itvisma (onasemnogene abeparvovec-brve) is a gene therapy that the FDA has approved to treat spinal muscular atrophy (SMA) in adults, teens, and children 2 or ...
Gene therapy has allowed several children born with inherited deafness to hear. A small study published Wednesday documents significantly restored hearing in five of six kids treated in China. On ...
EAST HANOVER, N.J., Nov. 24, 2025 /PRNewswire/ -- Novartis today announced that the US Food and Drug Administration (FDA) has approved Itvisma® (onasemnogene abeparvovec-brve) for the treatment of ...
Gazi University in Ankara has officially launched the production process for Türkiye's first local and national gene therapy research product. The project, which targets rare genetic diseases, is ...
On Monday, the U.S. Food and Drug Administration (FDA) approved Novartis AG’s (NYSE: NVS) Itvisma (onasemnogene abeparvovec-brve) for children two years and older, teens, and adults with spinal ...
Scientists have made major progress in treating congenital deafness using gene therapy. This approach repairs hearing loss by fixing the root cause—a faulty gene known as OTOF. The OTOF gene creates ...
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