Phase 1
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.
Now that July is over, the U.S. Food and Drug Administration (FDA) appears to be back in full swing with a full docket of drug decisions to make. There are four therapeutics with PDUFA dates this week involving six biopharma companies. Here’s a look.
Navitor Pharmaceuticals, headquartered in Cambridge, Massachusetts, announced that Thomas E. Hughes will be the company’s new chief executive officer.
Cellectis announced it had appointed Stefan Scherer to the role of senior vice president Clinical Development and deputy chief medical officer.
Impact Therapeutics, headquartered in Nanjing, China, completed a Series C financing round worth $30 million. The round was led by Decheng Capital with participating from Lilly Asian Ventures (LAV).
Denali Therapeutics stock climbed 11 percent after the release of positive data from its Phase I clinical trial of DNL201 in Parkinson’s disease.
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