Childs Cure co-founders Jainu and Shruti Jogani founded the organization to address the genetic causes of rare diseases, ...

The nonprofit’s stem cell therapy was able to completely restore the cornea in one of the 14 patients on the Phase I/II trial ...

Hearing impairment affects millions globally, with genetics playing a major role in infant hearing loss. Early screening and ...

The changes at Spark follow Roche's decision last year to halt development of a gene therapy for hemophilia A that was the ...

Financially, Monopar has a cash runway until 2026 but will need additional funds for commercialization efforts. See why I'm ...

UNC Charlotte researchers are contributing to pioneering breakthroughs in cancer therapeutics, communicable disease ...

The European Medicines Agency’s Committee for Medicinal Products for Human Use recommended the approval of Vyjuvek for the ...

A new study led by Profs. Fan Kelong and Yan Xiyun from the Institute of Biophysics of the Chinese Academy of Sciences ...

Columnist Jennifer Lynne assesses the effects of Pfizer's decision to halt production of its hemophilia B gene therapy, Beqvez.

Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, today welcomed the European Medicines Agency's (EMA’s) announcement that its Committee for Medicinal ...

A study led by UMass Chan Medical School researchers has demonstrated that a gene therapy to correct a mutation that causes ...