NBDF funds a broad range of research programs that seek to increase our understanding of the science behind bleeding disorders, how they affect people's lives, and pathways to better treatments and cures.
Establishing the Factors Responsible for Hepatocyte Permissiveness to AAV Vectors
Double-stranded Adeno-associated Virus (dsAAV) Vectors to Improve Efficacy and to Evaluate Safety of Factor IX Gene Therapy
Paul Monahan is Translational and Clinical Development Lead for Hematology Gene Therapy at Spark Therapeutics. Prior to 2015, Dr. Monahan was Professor of Pediatrics, Hematology/Oncology at the University of North Carolina at Chapel Hill, where he spent more than 20 years as an Investigator in the UNC Gene Therapy Center and treating children with bleeding disorders as an Attending Physician in the Harold Roberts Hemophilia Diagnostic and Treatment Center. He served on several clinical medical and scientific foundations and committees including the Board of Directors of the Hemostasis and Thrombosis Research Society. For ten years he served on the Medical and Scientific Advisory Committee of the National Hemophilia Foundation and as the Region IV Director for the US Hemophilia Treatment Center Network (CDC and MCHB). His basic science laboratory maintained a research focus on gene therapy for hemophilia as well as animal models for the study of hemophilic bone and joint disease, inhibitors in hemophilia B, and novel therapies from 1996 through 2016. In 2013 the National Hemophilia Foundation awarded Monahan the NHF Leadership in Research Award. Prior to joining Spark in Spring 2018, he performed preclinical research development and coordinated clinical trial initiation of hemophilia B and hemophilia A gene therapy trials in a collaborative partnership with Asklepios Biopharmaceuticals and as Medical Lead at Baxalta/Shire.
Retroviral Vector-Mediated Neonatal Gene Therapy for Hemophilia A
Nonviral Gene Delivery for Hemophilia
Gene Transfer of Factor VIII to the Liver in Vivo
Non-primate Lentiviral Vector-Based Gene Therapy for Hemophilia A
Gene Therapy for Hemophilia B by Direct Intramuscular Injections of Recombinant Adeno Associated Virus Serotype Vectors
Correction of Hemophilia with Lentiviral Vectors
Development and Characterization of Novel Genetically Engineered Factor VIII Molecules with Increased Activity and Stability
