Uniqure NV, of Amsterdam, reported data from an ongoing phase I/II trial of its gene therapy, AMT-060, showing that AAV5-mediated gene transfer was possible in three patients with hemophilia B who have pre-existing anti-AAV5 neutralizing antibodies. Read More
Helix Biopharma Corp., of Toronto, entered a collaboration agreement with Moffitt Cancer Center to extract radiomics data using its Healthmyne platform from computed tomography scans of late-stage lung cancer patients treated with L-DOS47. Read More
Nemus Bioscience Inc., of Costa Mesa, Calif., updated its private placement arrangement with Schneider Finance LLC for the sale of 1 million shares of series E preferred stock for gross proceeds of $20 million, stating that Schneider did not provide funding to close the transaction on July 10, as required under the securities purchase agreement and has requested an extension of the closing date. Read More
The FDA released draft standardized pharmaceutical quality/chemistry manufacturing and control data elements and terminologies in an effort to transform submitted data into a readily useable electronic format. Read More
The structure of the human brain's cannabinoid receptor 1 (CB1), which binds to the main psychoactive ingredient in marijuana, delta 9-tetrahydrocannabinol (D9-THC), has been elucidated by Chinese researchers, which could have major implications for drug discovery and development. Read More
HONG KONG – India's Central Drugs Standard Control Organization (CDSCO) has been streamlining regulations to standardize its rules on the market for pharmaceuticals. Read More
The FDA's green light for the submission of Amicus Therapeutics Inc.'s new drug application (NDA) for migalastat in Fabry disease followed what John Crowley, chairman and CEO, called a "data-driven, science-based discussion" with "largely the same reviewers" within the same FDA review division who previously looked askance at the company's data package. Read More
With multiple phase II readouts ahead this year, Arena Pharmaceuticals Inc. is "putting all the pieces on the table and we'll assemble them in a way that brings the astronauts back to earth," CEO Amit Munshi told BioWorld. Read More
LONDON – The pharma industry in the U.K. is turning to the courts to prevent a new "budget impact" test being used to bring down drug prices. Read More
