$12 Million Grant Awarded for Hemophilia A Gene Therapy Research


The grant, given to Indiana University School of Medicine, will fund 3 major projects to improve different aspects of hemophilia gene therapy.

The National Heart Lung and Blood Institute has awarded a $12 million grant to researchers from Indiana University (IU) School of Medicine to develop improved gene therapies for hemophilia A.1,2

Leading the research is Roland Herzog, PhD, Riley Children's Foundation professor of immunology and professor of pediatrics, and director, Gene and Cell Therapy Program, IU School of Medicine. The grant was awarded with the goal of better understanding hemophilia gene therapies as well as decreasing toxicity and improving durability.

“Several companies have taken this forward into clinical trials, and in some of these trials, the patients initially looked like they were cured,” Herzog said in a statement.2 “But what they all have in common is that they need to deliver a lot of the virus in order to get the desired results, and over time clotting factor levels started to decline. So, it’s clear that we need to further study the biology of this phenomenon.”

The grant is funding 3 main projects focused on different themes in hemophilia A gene therapy. The first project, led by Randal J. Kaufman, PhD, director, Degenerative Diseases Program, Sanford Burnham Prebys Center for Genetic Disorders and Aging Research, will focus on cellular toxicity and stress that can be induced by FVIII protein production.

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The second project will focus on molecular virology and is beingled by Weidong Xiao, PhD, professor of pediatrics, IU School of Medicine, with major contributions from Junping Zhang, PhD, and Anh Lam, PhD, both research scientists at IU. This project aims to improve the development of gene therapy viral vectors.

The last project is jointly led by Herzog and Ype de Jong, MD, PhD, assistant professor of medicine, Sanford I. Weill Medical College, Cornell University. This project will examine the immune system and its role in the interference of FVIII production over time.

An internal and external scientific advisory board will serve to counsel the grant program. The grant will also fund platforms to generate human liver cells and gene therapy vectors.

“This is an incredibly significant and urgent medical question, and it requires the synergy of multiple groups with different expertise to come together and solve a problem that they wouldn’t be able to solve on their own,” said Herzog.2 “My hope is that our studies will help the field as a whole move toward curing hemophilia A.”

1. Toward safer gene therapy for hemophilia A. NIH Reporter. February 5, 2022. https://reporter.nih.gov/search/ijSAalprwUiljAHsZ9Ping/project-details/10333185
2. Researchers receive $12 million for safer hemophilia treatments. News release. University of Indiana School of Medicine. March 8, 2022. https://medicine.iu.edu/news/2022/03/hemophilia-treatment-grant
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