Gene therapies are an undeniably exciting new frontier for medicine. From Kymriah's genetically modified, cancer-hunting immune cells, to Luxturna's vision-restoring healthy gene replacement, these therapies are fundamentally unlike 20th-century medicines which generally require ongoing drug treatment. Gene therapies can be one-off doses that in effect, cure a specific disease.
Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced that its New Drug Application (NDA) for inotersen has been accepted for Priority Review by the U.S. Food and Drug Administration (FDA). Inotersen is an investigational drug for the treatment of patients with hereditary TTR amyloidosis (hATTR). Priority Review is granted by the FDA to drugs with the potential to address a serious condition and, if approved, would provide a significant improvement in safety or effectiveness of the treatment, prevention, or diagnosis of a serious condition. The FDA has set a Prescription Drug User Fee Act (PDUFA) date of July 6, 2018. “TTR amyloidosis is a progressive, fatal disease with very limited treatment options. Receiving Priority Review in the U.S. and Accelerated Assessment in the EU for inotersen shows that the regulatory agencies recognize the high unmet need and the urgency to identify effective therapies to treat patients with this devastating disease,” said Sarah ...
The fear that machines will replace humans in the workplace is not a new one. In 1930, economist John Maynard Keynes conjectured that in the years to come, modern economies would face a new kind of affliction: what Keynes called “technological unemployment.”
Despite the availability of endoscopic therapy, many patients in the United States undergo surgical resection for non-malignant colorectal polyps. We aimed to quantify and examine trends in the use of surgery for non-malignant colorectal polyps in a nationally representative sample.
Shares of Belgium-based TiGenix NV shot up more than 72 percent in pre-market trading after Japan’s Takeda Pharmaceuticals opted to acquire the company with which it has a developmental partnership for $630 million.
Sangamo Therapeutics, Inc. and Pfizer Inc. announced a collaboration for the development of a potential gene therapy using zinc finger protein transcription factors to treat amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) linked to mutations of the C9ORF72 gene.
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