In a study co-led by the University of Maryland School of Medicine (UMSOM), researchers have identified a "master regulator" gene, ZNFX1, that may act as a biomarker to help guide treatment in future ...

Neurona Therapeutics has circled up $102 million to push its off-the-shelf cell therapy for epilepsy through phase 3 testing.

Knocking out a single gene reprograms part of the large intestine to function like the nutrient-absorbing small intestine. In ...

Doing more to incorporate caregiver burden into value assessments and cost-effectiveness research of Duchene muscular dystrophy treatments was the focus of a recent International Society for ...

The Swiss drugmaker, which holds European rights to Elevidys, suspended three trials while researchers investigate the death of a young man who died following treatment.

If preclinical findings were translated into the clinic, they could shorten the CAR-T generation lead time from two weeks to ...

In response to a request from the European Medicines Agency (EMA) following the death of a 16-year-old patient in the U.S., ...

Editas Medicine struggles with underwhelming data and lacks major partnerships. Find out why EDIT stock faces significant ...

None of the cell and gene therapies that reached the final regulatory submission stage were rejected by the FDA.

The European Medicines Agency asked for three clinical trials to be placed on hold until the exact cause of death of a US ...

Sarepta stock plunged Thursday after European officials put all studies of the company's gene therapy, Elevidys, on hold.