NHF 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.

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Engineered Regulatory T-cell Therapy for Tolerance to FVIII

Year:
-
Grants:
Career Development Award
Tags:
Hemophilia A (Factor VIII/F8)
Inhibitors
Author(s):
Moanaro Biswas

Moanaro Biswas, PhD, is currently an Assistant Professor in the Gene and Cell Therapy Program at the Herman B Wells Center for Pediatric Research at Indiana University, Indianapolis. She was appointed to this position in May 2018. She received her Masters and PhD in Biotechnology from India. She joined the University of Florida in 2013 as a Postdoctoral Research Associate and was subsequently appointed a faculty position in the department of Pediatrics at UF in 2017. She is mentored by Dr. Roland W. Herzog, a distinguished professor with extensive expertise in gene therapy for hemophilia. While at the University of FLorida, she received the Henry A. Kokomoor Award for excellence in Pediatric Research in 2017. Dr. Biswas has been the recipient of an early career investigator award from Bayer in 2016. Dr. Biswas is interested in studying cell and gene therapy-based treatments for combating inhibitor formation in hemophilia, As the 2018 recipient of the NHF/Novo Nordisk Career Development Award, which is made possible by a generous gift from Novo Nordisk, Dr. Biswas will be researching engineered Treg therapy for tolerance induction in hemophilia A. Chimeric antigen receptor (CAR) expressing Tregs, made specific for FVIII, will be tested in a murine model of hemophilia A. She will explore at the cellular and molecular level, interactions between antigen-specific CAR-Tregs and immune cell types involved in the development of inhibitors to FVIII.

von Willebrand Factor (VWF) Regulation in Blood Outgrowth Endothelial Cells from Individuals with Altered VWF Levels

von Willebrand Factor (VWF) Regulation in Blood Outgrowth Endothelial Cells from Individuals with Altered VWF Levels

Year:
-
Grants:
Career Development Award
Tags:
Von Willebrand Disease
Author(s):
Christopher J. Ng

The National Hemophilia Foundation (NHF) is pleased to announce Christopher J. Ng, MD, Assistant Professor of Pediatrics, University of Colorado Denver, as the recipient of the 2017 NHF/Novo Nordisk Career Development Award (CDA). The overall objectives of the CDA are to advance bleeding disorders research by promoting the development of innovative studies among established investigators. The award funds basic, pre-clinical or clinical research approaches to yielding scientific information or answers contributing to better treatments for inheritable bleeding disorders.

Dr. Ng's CDA project is on "von Willebrand Factor (VWF) regulation in blood outgrowth endothelial cells from individuals with altered VWF levels”. By using blood outgrowth endothelial cells, Ng will identify the transcriptional and epigenetic modifiers that play a role in the regulation of VWF levels. He will also be utilizing novel assays for characterizing the effects. The proposed studies should shed light on our molecular understanding of VWD, advance other areas of investigation and potentially lead to better diagnostic and prediction algorithms for bleeding in VWD. Ng will be mentored on this award by Jorge DiPaola, MD, Director of Basic and Translational Research in Pediatric Hemostasis and Thrombosis at University of Colorado Denver.

Dr. Ng received his medical degree in 2008 from the Keck School of Medicine at the University of Southern California and completed his pediatric residency at the University of Washington–Seattle Children’s Hospital. Dr. Ng has the distinction of having received a several previous awards from NHF and others during the early stages in his career. He is a former NHF-Shire Clinical Fellow, having received the award in 2013 while training under the mentorship of Dr. Marilyn Manco-Johnson, Director of the Hemophilia and Thrombosis Center at UCD and Dr. DiPaola (see below). Ng has been the recipient of NHF’s Judith Graham Pool Postdoctoral Research Fellowship in 2015 for his project on a “Multi-system evaluation of von Willebrand factor function in Type 1 von Willebrand disease mutations” (see below). Ng also received a 2013 HTRS Mentored Research Award, the CSL Behring Professor Heimburger Award and the Hemophilia Association of New York Research Award.

Ng’s immediate focus is to continue building his career as a physician-scientist, through basic and translational studies on VWF for enhancing knowledge of hemostatic and thrombotic disorders while continuing to treat patients and providing clinical leadership at the University of Colorado Denver’s Hemophilia and Thrombosis Center. For the longer term, Ng hopes to one day have an independent, NIH-funded laboratory studying VWF and the biological factors that lead to varied clinical phenotypes in hemophilia and VWD.

Through the CDA, Ng will receive $70,000 per year for up to three years. This award was selected through a process of peer review conducted by NHF's Research Review Committee. This volunteer committee is made up of highly experienced and respected physicians and researchers working in the field of hematology. NHF wishes to thank the reviewers as well as Novo Nordisk, Inc. for their very generous support of this research award.

PiggyBac Mediated Gene Transfer for Coagulation Disorders

PiggyBac Mediated Gene Transfer for Coagulation Disorders

Year:
-
Grants:
Career Development Award
Tags:
Gene Therapy
Author(s):
Janice M. Staber
Dr. Staber received her undergraduate e degree in biochemistry from the University of Iowa. She received her MD from the Carver College of Medicine at the University of lowa. She received strong mentorship under the guidance of Drs. Paul McCray and Steven Lentz during her post-doctoral research in gene therapy and hemophilia studies. She was subsequently appointed a faculty position at the University of Iowa Children's Hospital in 2010 and became Assistant Professor of Pediatrics in the Division of Hematology/Oncology in 2013.
Anti-fibrinolytic Strategies to Decrease Bleeding in Hemophilic Arthropathy

Anti-fibrinolytic Strategies to Decrease Bleeding in Hemophilic Arthropathy

Year:
-
Grants:
Career Development Award
Tags:
Hemophilic Arthropathy
Pain
Author(s):
Annette von Drygalski

Dr. von Drygalski's research focuses on better understanding the mechanisms operating the anti-fibrinolytic system and how this process works in patients with hemophilia and specifically with joint bleeding. Accelerated fibrinolysis and clot instability are becoming increasingly recognized as contributing factors to bleeding in hemophilia. One important molecule that prevents fibrinolysis is called TAFI (Thrombin Activatable Fibrinolysis Inhibitor). Dr. von Drygalski will be studying the dual anti-fibrinolytic and anti-inflammatory functions of TAFI in hemophilia arthropathy and develop a therapeutic TAFI-based approach to improving the efficacy of FVIIa based bypassing strategies for acute bleeding and joint protection. Her mentors and collaborators are John H. Griffin, PhD, Laurent O. Mosnier, PhD and Martin Lotz, MD, distinguished researchers at UCSD -The Scripps Research Institute. Dr. von Drygalski received her M.D. from the Universities of Erlangen/Nurnberg and Munich in 1995 and her Pharm.D. from the University of Munich in 1988. She joined the faculty in July 2011 as Assistant Clinical Professor at UCSD and Director of the Adult Hemophilia and Thrombosis Treatment Center, Division of Hematology/Oncology, Department of Medicine. She also has an appointment as Adjunct Assistant Professor at the Scripps Research Institute (TSRI). The funding for this award is made possible thanks to a generous grant from Novo Nordisk.

Identification of Chemical and Genetic Modifiers of Bleeding Disorders Using a Zebrafish Model System

Identification of Chemical and Genetic Modifiers of Bleeding Disorders Using a Zebrafish Model System

Year:
-
Grants:
Career Development Award
Tags:
Hemophilia A (Factor VIII/F8)
Hemophilia B (Factor IX/F9)
Author(s):
Jordan A. Shavit
Identification and Amelioration of T-cell Mediated Inflammatory Cytokines that Contribute to Anti-Factor VIII Inhibitor Formation in Hemophilia A

Identification and Amelioration of T-cell Mediated Inflammatory Cytokines that Contribute to Anti-Factor VIII Inhibitor Formation in Hemophilia A

Year:
-
Grants:
Career Development Award
Tags:
Hemophilia A (Factor VIII/F8)
Inhibitors
Author(s):
Keri C. Smith

Dr. Smith's research focuses on better understanding the development of factor VIII inhibitors. Specifically, she will focus on the effects of chemical signals, or cytokines, secreted by helper T cells on the development of inhibitor antibodies. She hypothesizes that certain cells, called Th17 cells, play an important role in the development of these antibodies by stimulating inflammation and driving the immune response toward inhibitor production. Dr. Smith received a BS from the University of Delaware before earning her Ph.D. from Montana State University. Prior to her appointment as an Assistant Professor at the University of Texas Medical School at Houston, she spent four years as a postdoctoral research fellow at the University of Michigan.

Pharmacogenomics of Hemophilia Therapy: Genetics of Inhibitor Antibody Response

Year:
-
Grants:
Career Development Award
Tags:
Inhibitors
Author(s):
Jay Lozier
Studies of VWF Function in VWF-Platelet and VWF-FVIII Interactions

Studies of VWF Function in VWF-Platelet and VWF-FVIII Interactions

Year:
-
Grants:
Career Development Award
Tags:
Von Willebrand Disease
Author(s):
Veronica Flood

Dr. Flood earned her undergraduate degree from Harvard University in 1995 and her doctorate from Tufts University in 1999. She is board certified in pediatrics as well as pediatric hematology/oncology. She has been serving as an Assistant Professor at the Medical College of Wisconsin, where she has been performing research and seeing patients since 2006.

As part of her NHF Career Development Award, Dr. Flood will study the common variants and interactions of the von Willebrand factor molecule as a means to enhance overall understanding and eventually improve upon current methods of von Willebrand disease testing. Using mouse models, she will investigate diagnostic alternatives by examining the interaction between ristocetin and its ability to bind to von Willebrand factor. As Dr. Flood states in her summary, "Improved testing for VWD will prevent patients with normal VWF function from receiving unnecessary treatment, while allowing more accurate assessment of patients with true defects in VWD."

Immune Response in Platelet-Derived FVIII Gene Therapy of Murine Hemophilia A

Year:
-
Grants:
Career Development Award
Tags:
Gene Therapy
Hemophilia A (Factor VIII/F8)
Platelets
Author(s):
Qizhen Shi

Recombinant Factor VIIa and RL Platelets as a Hemophilia Therapy

Year:
-
Grants:
Career Development Award
Tags:
Platelets
Hemophilia A (Factor VIII/F8)
Hemophilia B (Factor IX/F9)
Factor VII/F7
Author(s):
Alisa Wolberg

Self-Regulating HIV Vectors for Hemophilia A Gene Therapy

Year:
-
Grants:
Career Development Award
Tags:
Gene Therapy
HIV/AIDS
Hemophilia A (Factor VIII/F8)
Author(s):
Yashuhiro Ikeda

A Disease-Specific DNA Chip for von Willebrand Disease

Year:
-
Grants:
Career Development Award
Tags:
Von Willebrand Disease
Author(s):
Bradley E. Aouizerat

Platelets as Modifiers of Phenotype in Hemophilia A

Year:
-
Grants:
Career Development Award
Tags:
Hemophilia A (Factor VIII/F8)
Platelets
Author(s):
Donald L. Yee

Development of Nanoparticles for Non-viral Hemophilia Gene Therapy

Year:
-
Grants:
Career Development Award
Tags:
Gene Therapy
Hemophilia A (Factor VIII/F8)
Hemophilia B (Factor IX/F9)
Author(s):
Suzie Hwang Pun

Regulation of Factor V and VIII Secretion by an ER to Golgi Transport Receptor

Year:
-
Grants:
Career Development Award
Tags:
Factor V
Hemophilia A (Factor VIII/F8)
Author(s):
Bin Zhang

Individualized Phenotypic Prophylaxis for Hemophilia

Year:
-
Grants:
Career Development Award
Tags:
Hemophilia A (Factor VIII/F8)
Hemophilia B (Factor IX/F9)
Author(s):
Kathleen Brummel-Ziedins

Depletion of Immune Response to AAV-F.IX

Year:
-
Grants:
Career Development Award
Tags:
Hemophilia B (Factor IX/F9)
Gene Therapy
Author(s):
Huang-Ge Zhang

Establishing the Factors Responsible for Hepatocyte Permissiveness to AAV Vectors

Year:
-
Grants:
Career Development Award
Tags:
Gene Therapy
Hemophilia A (Factor VIII/F8)
Hemophilia B (Factor IX/F9)
Author(s):
Hiroyuki Nakai

Double-stranded Adeno-associated Virus (dsAAV) Vectors to Improve Efficacy and to Evaluate Safety of Factor IX Gene Therapy

Year:
-
Grants:
Career Development Award
Tags:
Gene Therapy
Hemophilia B (Factor IX/F9)
Author(s):
Paul E. Monahan
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

Year:
-
Grants:
Career Development Award
Tags:
Gene Therapy
Hemophilia A (Factor VIII/F8)
Author(s):
Lingfei Xu