uniQure Announces First Patient Treated in Dose-Confirmation Study in Patients with Hemophilia B
The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure, a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, announced it has treated the first patient in its Phase IIb dose-confirmation study of AMT-061, an investigational AAV5-based gene therapy […]
U.S. District Court in Delaware rules against Shire in preliminary injunction
On Aug. 7, 2018, the U.S. District Court in Delaware ruled against Shire in its request for a preliminary injunction against Roche AG from creating, transporting and marketing its recently-approved drug Hemlibra in the U.S., which received FDA approval to treat people with hemophilia A with inhibitors in 2017. This ruling means there are no […]
Spark Therapeutics investigational trail moves into Phase 3
The following is an excerpt from a press release from Spark Therapeutics. Read the press release in its entirety here. Investigational SPK-8011 moving to Phase 3 As of the July 13 data cutoff, 12 participants in the Phase 1/2 trial have received a single administration of investigational SPK-8011, including two at a dose of 5×1011 […]
Catalyst Biosciences releases update on trial of Factor IX treatment of severe hemophilia B
The following is a summary of a press release from Catalyst Biosciences by Dr. Robert Sidonio, a trusted medical advisor. Read the full press release here. On June 18, Catalyst Biosciences provided an update of the progress from its Phase ½ trial investigating the use of FIX variant product, called CB 2679d/ISU304, which is a […]
Grifols Voluntarily Recalls Profilnine
Grifols Biologicals is initiating a voluntary product recall of one lot of Profilnine. Grifols Biologicals has determined there is low risk to the patient associated with this issue; however, Grifols Biologicals is requesting the lot to be returned out of caution. Therapy Plasma Product NDC Number Lot Number Size Packaging Expiration Date 68516-3208-2 A1PBB00072 1000 […]
FDA Grants Priority Review to Genentech's HEMLIBRA for People with Hemophilia A without Factor VIII Inhibitors
Genentech, a member of the Roche Group, announced the U.S. Food and Drug Administration has accepted the company’s supplemental Biologics License Application and granted Priority Review for HEMLIBRA Â for adults and children with hemophilia A without factor VIII inhibitors. The sBLA is based on data from the Phase III HAVEN 3 study. The FDA is […]
BioMarin Presented an Update on the Phase 1/2 Study of Valoctocogene Roxaparvovec at WFH's 2018 World Congress in Scotland
The following is an excerpt from a press release from BioMarin. Read the entire press release here. BioMarin Pharmaceutical Inc. announced an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy treatment for severe hemophilia A. The updated results were presented during […]
Novo Nordisk Announced New Data at WFH's 2018 World Congress in Scotland
The following is an excerpt from a press release from Novo Nordisk. Read the full press release here. Adults with haemophilia B who received a single dose Refixia® (nonacog beta pegol; N9-GP) achieved greater total factor IX exposure than those treated with rFIXFc (recombinant factor IX-Fc fusion protein). The head-to-head paradigm7 trial also observed a […]
uniQure Presents New Data Demonstrating Clinical Benefits at American Society of Gene and Cell Therapy Annual Meeting in Chicago
The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure N.V., a leading gene therapy company advancing transformative therapies for patients with severe medical needs, presented data showing successful liver transduction with the AAV5 vector in both non-human primates and humans with pre-existing anti-AAV5 neutralizing antibodies (NABs). […]
Bioverativ Presents Preliminary Phase 1/2a Data at WFH's 2018 World Congress in Scotland
The following is a press release from Bioverativ. Read the full press release here. Bioverativ Inc., a Sanofi company dedicated to transforming the lives of people with rare blood disorders, presented initial clinical data for BIVV001, a novel and investigational von Willebrand factor (VWF)-independent factor VIII therapy for people with hemophilia A. Preliminary safety and […]