By Karen Roman Biopharmaceutical company Medera Inc. said it completed patient enrollment for Cohort B of its clinical trial ...

Abu Dhabi successfully completes its first gene therapy for genetic blood disorders, revolutionizing treatment approaches for ...

A new CRISPR approach can control genes without cutting DNA, opening a safer path for treating genetic diseases. A newly ...

Expected rapid advances in CAR T and other cellular therapies may expand treatment options not only in cancer but also in ...

An experimental gene therapy seems to slow the progression of Huntington’s disease by about 75 per cent, and researchers are ...

A new CRISPR breakthrough shows scientists can turn genes back on without cutting DNA, by removing chemical tags that act ...

Gene therapy advancements necessitate improved delivery infrastructure and comprehensive data collection across age groups to address logistical complexities and policy gaps. Multidisciplinary ...

A 10-month-old baby who sparked nationwide headlines after receiving a first-of-its kind gene-editing treatment was released from the hospital this week.

MSK is the first hospital in New York City to offer gene therapy through its pediatric cancer program MSK Kids. Exagamglogene autotemcel (exa-cel) (Casgevy®) is a gene therapy for treating sickle cell ...

This image shows three brain cells with the faulty protein that causes Huntington’s disease. The bright yellow cell in the middle has built up a clump of this protein inside it. The blue spots in the ...

Editor’s note: On June 3, 2025, KJ Muldoon was discharged from the hospital and is now at home with his family. When a baby born in Philadelphia was announced as the first person to get a gene therapy ...

With a pair of Phase III trial flops, Ultragenyx will explore cost reductions as analysts turn attention to an upcoming ...