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.

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 ...

Just a few weeks after conception, stem cells are already orchestrating the future structure of the human brain. A new ...

A team of researchers from the Keck School of Medicine of USC has developed an advanced tool for analyzing chimeric antigen ...

Siren Biotechnology is trying to raise $5 million via the type of crowdfunding round that’s rarely used by young drugmakers.

London hospital gene therapy breakthrough gives boy with ‘death sentence’ disease a normal life - Eisa Hussein took part in a ...