Dimension Therapeutics announced it will discontinue the development of DTX101, an investigational AAVrh10-based gene therapy product in development for the treatment of moderate/severe-to-severe hemophilia B. The decision followed the review of the emerging DTX101 Phase 1/2 clinical study data, including the data as of the beginning of May 2017, and the observation that the data would not meet […]
Sangamo Therapeutics, Inc. and Pfizer Inc. recently announced an exclusive, global collaboration and license agreement for the development and commercialization of gene therapy programs for Hemophilia A, including SB-525, one of Sangamo’s four lead product candidates, which Sangamo expects will enter the clinic this quarter. Click here to read the full press release from Sangamo Therapeutics.
uniQure recently presented data at the American Society of Gene and Cell Therapy (ASGCT) 20th Annual Meeting in Washington, D.C. on successful repeated hepatic gene delivery with their AAV5 vector following a proprietary immunoadsorption procedure in non-human primates (NHPs). The results described successful sequential transduction with the two reporter transgenes and highlighted the progress made in readministration protocols […]
Kedrion Biopharma announced a return to normal supply levels of Koāte in the 1000 vial size and a series of large-scale improvements to their overall operations following a temporary period of limited availability of product. Click here to read the full contents of letter provided by Kedrion Biopharma regarding this matter.
uniQure recently received the designation of a “priority medicine” from the European Union for a potential gene therapy treatment for Hemophilia B. From the press release: uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that AMT-060, its proprietary, investigational gene therapy in patients with severe […]
Today, on World Hemophilia Day, many pharmaceutical companies joined in the celebration by recognizing and supporting the hemophilia community. Linked below are statements from a wide group of companies on today’s celebration of World Hemophilia Day: Grifols Press Release Bioverativ Press Release Novo Nordisk on supporting women with bleeding disorders Shire Press Release CSL Behring […]
Drug manufacturer, Novo Nordisk recognized World Hemophilia Day 2017 by celebrating the theme of the year; Hear Their Voices, by showing support for women with bleeding disorders. To learn more, read Novo Nordisk’s press release in it’s entirety. here.
Clotting factor manufacturer, Shire, is celebrating World Hemophilia Day in part by hosting an awareness rally in Chicago, Illinois. The rally is aimed mainly to educate the public on the dramatic estimate of a hemophilia bleeding episode occurring worldwide every 3-15 seconds. See more on their website. Read a full statement from Shire recognizing World Hemophilia […]
As part of World Hemophilia Day celebrations, Grifols has announced a large donation of clotting factor to the World Federation of Hemophilia (WFH) Humanitarian Aid Program. This announcement is a continuation of the company’s three-year commitment from 2014, bringing the total humanitarian aid commitment to more than 200M IU of Factor VIII over eight years Read the […]
On April 11, Catalyst Biosciences, a clinical-stage biopharmaceutical company focused on developing novel medicines to address hematology indications, reached a key milestone towards starting human trials with completion of the CB 2679d/ISU304 toxicology studies. Their press release can be read in full here.
Assisting and Advocating for the Bleeding Disorders Community