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 ...
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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 U.S. Food and Drug Administration approved PTC Therapeutics' gene therapy to treat a potential fatal enzyme deficiency ...
StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.
The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...
At Neurogene, the developer of a gene therapy for Rett syndrome, expediency seems to be the priority, @adamfeuerstein writes.
Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review ...
StitchR makes it possible to deliver larger payloads with current vectors, expanding the list of diseases targetable by gene therapies.
The approval marks the first time gene therapy will be available to treat patients with aromatic I-amino acid decarboxylase ...
University of Wisconsin–Madison researchers targeting a group of hereditary neurodegenerative diseases have found success ...
The FDA has accepted for review the resubmitted BLA for prademagene zamikeracel for recessive dystrophic epidermolysis bullosa.
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