Nov 13 (Reuters) - The U.S. Food and Drug Administration approved PTC Therapeutics' (PTCT.O), opens new tab gene therapy to ...
In another sign of biopharma's waning interest in the traditional ex vivo gene therapy approach, CSL Behring is shutti | CSL ...
Intellia presented data from its Phase I study of NTLA-2001 at the 2024 American Heart Association scientific meeting in ...
Based on a small trial with positive muscle performance data, Regenxbio is expected to pursue approval of its gene therapy ...
Neurogene has dropped plans to keep studying the high dose of its Rett syndrome gene therapy because of an adverse event that ...
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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 ...
Neurogene has halted patient enrolment in the high-dose arm of its trial of Rett syndrome gene therapy, sparking another run on its shares. Last week, the company reported that NGN-401 had shown ...
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 ...
Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...
University of Wisconsin–Madison researchers targeting a group of hereditary neurodegenerative diseases have found success ...
On Thursday, Bluebird Bio Inc. (NASDAQ:BLUE) reported a third-quarter EPS loss of 31 cents, compared to a loss of 80 cents a ...
The approval marks the first time gene therapy will be available to treat patients with aromatic I-amino acid decarboxylase ...
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