Company expects up to six-month review period per FDA guidelines New longer-term clinical data demonstrating durable positive brain biochemical ...
Stocktwits on MSN
Rare stock slips 3% today — what’s up with Ultragenyx’s gene therapy for Sanfilippo syndrome?
Ultragenyx now expects a 6-month review period for the therapy and a likely decision from the FDA regarding potential ...
Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due ...
(Yicai) Jan. 28 -- Chinese medical researchers have made a breakthrough in gene therapy that overcomes the limitations of ...
Researchers developed a topical gene therapy that corrects disease-causing mutations in human skin models using lipid ...
Meghan McLean Poon Ph.D., Karen Potter Ph.D., Yuying You Ph.D. Cell and gene therapies represent a transformative frontier in modern medicine, offering potential cures for previously untreatable ...
AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, recently announced that the United States Food and Drug Administration (FDA) has ...
An AAV vector genome integration event associated with PLAG1 overexpression was identified in a preliminary analysis of the resected tumor.
BURLINGTON, Mass., May 19, 2025 (GLOBE NEWSWIRE) -- Fractyl Health, Inc. (GUTS) (the Company), a metabolic therapeutics company pioneering pattern-breaking treatments for obesity and type 2 diabetes ...
Creative Biolabs is helping researchers overcome challenges of gene therapy research by providing analytical testing ...
Pharmaceutical Technology on MSN
Sensorion announces $71.9m reserved offering to expand gene therapy pipeline
Sanofi’s participation will support the progression of Sensorion’s genetic medicine pipeline.
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