Phase I
Forendo Commences Phase Ia Study for FOR-6219 for Endometriosis
Single intravenous dose phase 1 trial showed that AIC649 was safe and well tolerated in all dose groups
Part 1 top-line data of the NOX-A12 trial to be presented at the September ‘Fourth International Cancer Immunotherapy Conference’
SpringWorks Therapeutics and BeiGene, Ltd. have teamed up to develop therapeutics that will target advanced solid tumors that contain RAS mutations, as well as other MAPK aberrations.
Shares of Synlogic, Inc. are climbing this morning after the New York Times highlighted the company’s proprietary synthetic biotics being used to treat patients with PKU. The company also released positive interim data from an ongoing study of its PKU treatment.
Cambridge, Mass.-based Biogen and Tokyo, Japan-based Eisai Co. announced results from an analysis of their ongoing long-term extension (LTE) Phase Ib trial of aducanumab for Alzheimer’s disease.
Gene therapy has been a dream for many researchers for decades—inserting a healthier gene into patients’ cells to replace defective genes and curing diseases. One disease that would seem to be a good target for gene therapy is hemophilia.
Bexion Pharmaceuticals is not a household name as far as drugmakers go. But, this week the company and its investigational cancer treatment, BXQ-350, is receiving lots of mainstream media coverage as a potential game-changer.
Cambridge, Mass.-based Sarepta Therapeutics expanded its gene therapy pipeline up to 11 in an equity deal with Lacerta Therapeutics. Investing $30 million into Alachua, Florida-based Lacerta Therapeutics, Sarepta gained access to the company’s AAV-based CNS-targeted gene therapies.
An investigational gene therapy treatment for hemophilia A has seen a 97 percent response rate after one treatment for the 12 trial participants, Spark Therapeutics announced this morning.
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