Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...
On Thursday, Bluebird Bio Inc. (NASDAQ:BLUE) reported a third-quarter EPS loss of 31 cents, compared to a loss of 80 cents a ...
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.
StitchR makes it possible to deliver larger payloads with current vectors, expanding the list of diseases targetable by gene therapies.
PTC Therapeutics’ gene therapy, Kebilidi to treat AADC deficiency receives US FDA approval: Warren, New Jersey Friday, November 15, 2024, 10:00 Hrs [IST] PTC Therapeutics, Inc., ...
Put up against placebo in the phase 3 EMBARK trial, delandistrogene moxeparvovec (Elevidys) did not significantly improve ...
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Spirovant Sciences doses first subject in cystic fibrosis gene therapy trialSpirovant Sciences has announced the commencement of its SAAVe Phase I/II study, with the first patient receiving a dose of ...
Bluebird has just two quarters until it’s out of cash. Executives are looking for financing to extend that runway to a ...
The FDA has accepted for review the resubmitted BLA for prademagene zamikeracel for recessive dystrophic epidermolysis bullosa.
As novel gene therapies emerge, there is a complex interplay among their potential benefits, the burdens they may impose and ...
Understanding what to patent versus what to keep as a trade secret is an increasingly important and challenging decision ...
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