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.
HONG KONG – With $13.2 million in new financing, Tokyo-based oncology drug developer Anaeropharma Science Inc. now plans to focus on research and development of drug candidates that target hypoxic microenvironments of tumors by using recombinant Bifidobacterium.
Kala Pharmaceuticals Inc., of Waltham, Mass., has filed its form S1A to offer to sell 6 million shares of its common stock, priced between $14 and $16 per share, in an IPO.
Inmed Pharmaceuticals Inc., of Vancouver, British Columbia, said it entered a research and development collaboration with Atera SAS, of France, a tissue engineering company specializing in the development of advanced human tissue models.
DUBLIN – Shire plc is challenging Roche Holding AG’s imminent disruption of the hemophilia A market by slapping a preliminary injunction on the Swiss pharma, alleging that the latter’s description of adverse events that occurred in a phase III trial of emicizumab is “inaccurate and misleading” and could compromise patient safety.
Pluristem Therapeutics Inc., of Haifa, Israel, said Austria’s regulatory agency cleared the firm to begin enrollment for a pivotal phase III trial of PLX-PAD cell therapy in critical limb ischemia (CLI).
Alnylam Pharmaceuticals Inc., of Cambridge Mass., and Sanofi Genzyme, a unit of Paris-based Sanofi SA, reported data from an ongoing phase II open-label extension study testing fitusiran in 33 patients with hemophilia A and B, with or without inhibitors.
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