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 […]

Spark Therapeutics and Pfizer Announced Data at WFH's 2018 World Congress in Scotland

The following is an excerpt from a press release from Spark Therapeutics. Read the full press release here. Spark Therapeutics, a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, and Pfizer, announced, with a cumulative follow-up of more than 18 patient years of observation, all 15 participants in the ongoing […]