Gene therapy has emerged as a promising approach to previously untreatable conditions, including Duchenne muscular dystrophy (DMD), a progressive disorder caused by DMD mutations that leads to early ...

The nonprofit says its pipeline offers more affordable and accessible therapeutics, including point-of-care CAR-T cell therapy for leukemia and lymphoma.

Researchers at the University of Minnesota have completed a first-in-human clinical trial testing a CRISPR/Cas9 gene-editing ...

HAYA Therapeutics, SA, a biotechnology company pioneering precision RNA-guided regulatory genome targeting therapeutics that reprogram disease-driving cell states for rare, common, chronic and ...

An investigational gene therapy has successfully restored immune function in all nine children treated with the rare and life ...

Scientists from the Keck School of Medicine at University of Southern California have developed a spectral flow ...