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.”
Pfizer has signed on to fund the launch of a hunt for a slate of small molecules that can degrade proteins, a key therapeutic pathway that’s been playing a role in prostate cancer and other areas.
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.
The new total-body PET/CT scanner could revolutionize the understanding and treatment of disease through analysis of better imaging data from the whole body.
US-based gene sequencing firm Illumina has signed an agreement with Guangzhou KingMed Diagnostics Group to co-develop new oncology and hereditary disease testing applications in China.
Johnson & Johnson (J&J) has signed 15 new partnerships to explore, develop and advance new medical devices, therapeutics and consumer health solutions.
An experimental drug being developed by Shire to treat cytomegalovirus (CMV) infection has been given a boost in the US with a ‘Breakthrough Therapy Designation’.
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