Neurogene is proceeding with a scaled-back study of its proposed NGN-401 gene therapy for the rare genetic neurological disorder Rett syndrome after a serious adverse event left a study participant in ...
At Neurogene, the developer of a gene therapy for Rett syndrome, expediency seems to be the priority, @adamfeuerstein writes.
Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...
On Thursday, Bluebird Bio Inc. (NASDAQ:BLUE) reported a third-quarter EPS loss of 31 cents, compared to a loss of 80 cents a ...
Intellia Therapeutics CEO John Leonard has, for the last couple years, found himself stuck in a financial Groundhog Day.
Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...
Medpage Today on MSN4d
First Brain-Delivered Gene Therapy Approved for AADC DeficiencyThe treatment is the first approved gene therapy for AADC deficiency, the FDA said. People with this rare disorder have ...
The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...
Nov 13 (Reuters) - The U.S. Food and Drug Administration approved PTC Therapeutics' (PTCT.O), opens new tab gene therapy to ...
Find out what is driving the slowdown in gene therapy investment, as the sector continues to struggle with widespread layoffs ...
StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.
University of Wisconsin–Madison researchers targeting a group of hereditary neurodegenerative diseases have found success ...
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