Maria Lowe, Ph.D., associate vice president of Pharmaceutical Intelligence at Institute for Clinical and Economic Review ...

Childhood-onset striatonigral degeneration is a rare genetic disorder that robs children of the ability to walk and speak by ...

Shares of Sarepta Therapeutics SRPT dropped more than 6% yesterday after the EMA placed a clinical hold on all studies ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) shared an update on Elevidys (delandistrogene moxeparvovec-rokl), the only approved ...

Sangamo Therapeutics has stacked up $18 million cash from Eli Lilly in a licensing deal that will let the pharma use the ...

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 ...

Laura Bobolts, PharmD, BCOP, senior vice president of clinical strategy and growth at OncoHealth, discusses strategies for managing costs of novel cellular and gene therapies in the oncology pipeline.

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

Head and neck cancers often begin in the mouth, throat, or voice box. They're among the most common cancers in the world, ...

Gene therapy, a technique that is revolutionizing the treatment of multiple genetic conditions, including eye and muscle diseases and blood disorders, requires efficient and specific delivery of the ...

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