Consortium Focused on Hemophilia A Patients, Announces Significant Progress in Development of ‘First in World’ Regenerative Medicine Therapy

Sernova Corp, an Ontario-based clinical stage regenerative medicine company, reported significant scientific progress achieved in the development of a ‘first in world’ personalized regenerative medicine therapy for the treatment of Hemophilia A patients by the HemAcure Consortium and confirmation of the second phase of funding of the Consortium by the European Commission. The therapy being […]

Pharmaceutical Business News

Sangamo Therapeutics and Pfizer Announce Collaboration for Hemophilia A Gene Therapy

Sangamo Therapeutics, Inc. and Pfizer Inc. announced this week, 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. “Sangamo brings deep scientific and technical expertise across multiple […]

rare diseases

Capsule for Severe Bleeding Disorder Moves Closer to Reality

Motivated by the tribulations of hemophilia patients and their families, researchers funded by the National Institute of Biomedical Imaging and Bioengineering are working to develop a pill to treat this serious inherited bleeding disorder. Oral delivery of the treatment–clotting factor IX–would allow individuals with type B hemophilia to swallow a pill rather than be subjected […]

Biotechnology Gene Therapy

After One Dose of Gene Therapy, Hemophilia B Patients Maintain Near-Normal Levels of Clotting Factor

At ASH Meeting, CHOP Hematologist Leads Clinical Trial in Which All Subjects Safely Maintain Factor IX Expression that Curtails Disabling Bleeding Researchers are reporting the highest and most sustained levels to date of an essential blood-clotting factor IX in patients with the inherited bleeding disorder hemophilia B. After receiving a single dose of an experimental […]