The Hereditary Disease Foundation is thrilled to announce the recipients of our inaugural Transformative Research Awards! Congratulations to the two all-star teams, one led by Beverly Davidson, PhD of Children’s Hospital of Philadelphia and the University of Pennsylvania, and the other led by Ricardo Mouro Pinto, PhD of Massachusetts General Hospital and Harvard Medical School. They will each receive funding of $1 million over the next two years to advance their work toward developing treatments for Huntington’s disease. Dr. Davidson’s and Dr. Mouro Pinto’s teams each use cutting-edge methods but take different approaches to advance Huntington’s disease research that, if successful, could lead to truly transformative therapies for Huntington’s disease.
The Transformative Research Awards were made possible through a partnership between the Hereditary Disease Foundation and a generous group of anonymous donors. They are the largest grants ever awarded by HDF.
“These new awards are designed to move the most innovative work in Huntington’s disease from concept to practice,” said Meghan Donaldson, CEO of the Hereditary Disease Foundation. “We are funding collaborative research teams who are focused on creating new ways to move toward a disease-modifying treatment and provide transformational new insights to the HD research field. We thank our donors whose extraordinary generosity and vision have made the Transformative Research Awards possible, and we congratulate our first recipients – Bev, Ricardo, and their teams.”
Dr. Davidson will collaborate with Leslie Thompson, PhD from the University of California, Irvine, and Jang-Ho Cha, MD, PhD from Latus Biosciences. Their project, “Translational Studies on PIAS1 and MSH3 Knockdown for Huntington’s Disease,”will target two proteins using an adeno-associated virus (AAV) that can be injected into the brain at very small volumes and achieve wide distribution throughout the brain. MSH3 is involved in a biological phenomenon called “somatic instability,” which is the molecular stutter in the CAG repeat that causes HD to expand over time in vulnerable tissues, particularly the brain. PIAS1 has been shown to significantly improve symptoms in mice that model HD.
Beverly Davidson (principal investigator), Leslie Thompson and Jang-Ho Cha (co-investigators)
Dr. Davidson has been working on Huntington’s disease for decades, most recently specializing in developing and improving viral delivery strategies for HD therapeutics. She also has experience moving promising drug targets to primate models. Dr. Thompson is a renowned leader in stem cell research and has extensive experience working with various models of HD mice. Dr. Cha has worked in the pharmaceutical sector for decades and is well-versed in taking drugs from conception to clinic. Both Drs. Thompson and Cha were key members of the research team who traveled to Venezuela annually on a quest to find the gene that causes Huntington’s disease.
Ricardo Mouro Pinto (principal investigator), James Gusella, Benjamin Kleinstiver, David Liu (co-investigators), Benjamin Deverman, Joseph Nabhan, Cathleen Lutz, and Vanessa Wheeler (collaborators)
Dr. Mouro Pinto will work with a large team of experts including James Gusella, PhD and Benjamin Kleinstiver, PhD from Massachusetts General Hospital and Harvard Medical School; David Liu, PhD and Benjamin Deverman, PhD from the Broad Institute of MIT and Harvard; Joseph Nabhan, PhD from Vesigen Therapeutics; Cathleen Lutz, PhD, MBA from the Jackson Laboratory; and Vanessa Wheeler, PhD from Massachusetts General Hospital and Harvard Medical School. Their project, “Therapeutic Targeting of Somatic CAG Expansions with Precise CRISPR Base Editing,” will develop genome editing therapeutics that target the cause of the CAG repeat expansion in Huntington’s disease. Their goal is to permanently halt somatic instability with a one-and-done (gene editing) approach to the brains of mice that model Huntington’s disease.