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 ...
StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.
Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...
StitchR makes it possible to deliver larger payloads with current vectors, expanding the list of diseases targetable by gene therapies.
Put up against placebo in the phase 3 EMBARK trial, delandistrogene moxeparvovec (Elevidys) did not significantly improve ...
As novel gene therapies emerge, there is a complex interplay among their potential benefits, the burdens they may impose and ...
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., ...
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.
Spirovant Sciences has announced the commencement of its SAAVe Phase I/II study, with the first patient receiving a dose of ...
High cost is a major challenge. The global cell and gene therapy market is projected to expand by $6.56b, reflecting an ...