Three companies – Intellia Therapeutics, Editas Medicine and uniQure – are each anticipating a bright 2022 as the promises of gene therapy continue to be realized.
Sanofi Hits the Brakes on Its Hemophilia Program Published: Nov. 6, 2020 By Mark Terry BioSpace Three hemophilia organizations, the World Federation of Hemophilia, the European Haemophilia Consortium and the National Hemophilia Foundation issued a joint statement reporting that Sanofi Genzyme had placed a global dosing hold on its “full clinical development program for […]
London-based Freeline announced the closing of a $120 million extended Series C financing to advance the company’s gene therapy programs, and is considering additional capital fundraising options during 2020.
CSL Behring bolstered the company’s growing gene therapy portfolio with the acquisition of global rights to uniQure’s gene therapy program for hemophilia B that has a price tag of up to $2 billion.
Denmark’s Novo Nordisk paused three clinical trials of concizumab over safety issues. The studies are evaluating the drug for hemophilia A and B.
Bayer announced a three-year collaboration agreement with Children’s Hospital of Philadelphia (CHOP) for the discovery and development of small molecules to develop a first-in-class oral non-replacement therapy for the treatment of hemophilia A and B.
Pfizer Inc. is investing $500 million to expand a manufacturing facility in Sanford, North Carolina, that plays a central role in the company’s efforts to become a major player in gene therapy.
Roche is buying U.S.-based gene therapy specialist Spark Therapeutics for $4.3 billion after developments in this area convinced the Swiss drugmaker to “step up”, Chief Executive Severin Schwan said.
U.S. regulators approved expanded use of Roche’s drug Hemlibra to include almost all patients with hemophilia A.
Clinical data suggests that uniQure’s AAV5 gene therapy may be viable treatments for 97 percent of hemophilia B patients.