Trace Neuroscience, which launched this week with $101 million, benefited from last year’s approval of Qalsody. Its CEO spoke ...
The FDA has accepted for review the resubmitted BLA for prademagene zamikeracel for recessive dystrophic epidermolysis bullosa.
Put up against placebo in the phase 3 EMBARK trial, delandistrogene moxeparvovec (Elevidys) did not significantly improve ...
Immunotherapy, which enhances the immune system's T cell response to eliminate cancer cells, has emerged as a key approach in ...
Why one trial participant's healthy birth after her CAR-T treatment for lupus is catching resesarchers' attention: ...
Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...
StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.
By exploiting the genetic variation in cancer cells, an already approved cancer drug demonstrated enhanced effects against cancer cells in specific patient groups.
The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus vector-based gene therapy indicated for the treatment of adult ...
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