KJ Muldoon, a 10-month-old baby, was diagnosed with the genetic disease carbamoyl-phosphate synthetase 1 deficiency after he ...

New gene therapy improves the life of 18-year-old with Sickle Cell Disease which affects 5,000 New Jersey residents living ...

Gene therapy treatments ... sickle cell disease and beta thalassemia, which affect tens of thousands of patients in the U.S. Those treatments are sold by major pharmaceutical and biotechnology ...

The personalized CRISPR treatment could be the future of gene therapy, but hurdles remain before everyone has access.

Vertex Pharmaceuticals Incorporated today announced data across multiple studies demonstrating positive clinical and quality of life benefits of treatment with CFTR modulators and, in particular, ...

Cathie Wood is bullish on biotech companies using gene sequencing, AI, and CRISPR tech to cure previously untreatable ...

Glioblastoma has been deadly and hard to treat, but researchers say they’ve made progress with bivalent CAR T-cell therapy ...

Researchers have identified an early postnatal window that allows gene transfer to circulating blood stem cells, advancing ...

It’s back to the drawing board for bluebird bio and its discussions with NICE, which has rejected its beta thalassaemia gene therapy Zynteglo for regular NHS use in first draft guidance.

Scientists at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, have found that gene editing using ...