Gilbert Family Foundation Announces $12 Million Gene Therapy Initiative to Address Underlying Cause of NF1
- The Gilbert Family Foundation's Gene Therapy Initiative is the first in a series of the Foundation's medical initiatives to eradicate NF1.
- The initiative will award funding to institutions and researchers exploring developmental and curative therapies to address the underlying genetic abnormalities of NF1.
December 12, 2018 [DETROIT] – The Gilbert Family Foundation, a private foundation established by Jennifer and Dan Gilbert, today announced its first major initiative around gene therapy research.
The Gilbert Family Foundation’s goal is to accelerate the development of curative therapies that address the underlying genetic abnormalities in neurofibromatosis type 1 (NF1) patients. Over the next three years, $12 million in research grants from the Gilbert Family Foundation will fund several renowned multi-disciplinary research teams to identify which gene therapy techniques are most promising for NF1. The Foundation utilized a rigorous and extensive process, including peer review, to identify top researchers and projects in each of the initiative’s gene therapy focus areas.
The distinguished researchers and institutions chosen include:
- Charles Gersbach, PhD, Duke University
- Eric Pasmant, PhD, Paris Descartes University
- Andre Leier, PhD, University of Alabama
- David Bedwell, PhD, University of Alabama
- Deanne Wallis, PhD, University of Alabama
- Robert Kesterson, PhD, University of Alabama
- Allan Jacobson, PhD, University of Massachusetts
- Miguel Sena-Esteves, PhD, University of Massachusetts
The Gilbert Family Foundation believes that directly repairing, replacing, or bypassing the NF1 gene mutation is an important piece to curing NF.
“We are committed to funding revolutionary and big-idea research,” said Dan Gilbert, Co-Founder of the Gilbert Family Foundation. “The teams we have partnered with share this vision and our family is honored to be working closely with this talented group of researchers and physicians.”
NF1 drug development has previously focused on addressing the symptoms caused by the disease. This important initiative takes an alternative approach by working to develop therapies that address the underlying genetic causes of NF1. This includes gene replacement, gene editing, RNA editing, exon skipping, and nonsense mutation suppression.
The Gilbert Family Foundation launched with a $150 million commitment from Dan and Jennifer Gilbert to fund groundbreaking, cutting-edge research to accelerate a cure for NF1, as well as community efforts in the city of Detroit. In partnership with the Milken Institute Center for Strategic Philanthropy, the Gilbert Family Foundation has established initial focus areas guiding its research efforts.
NF1 causes tumors to form on nerves anywhere in the body, which can lead to disfigurement, blindness and cancer. NF1 can also result in learning disabilities, bone deformities and cardiovascular issues. The course of the disease is both unpredictable and variable among individuals.
“We have been deeply impacted ever since our oldest son Nick was diagnosed with NF1 as a toddler, and believe this research will benefit numerous families affected by NF1 around the globe, as well as our son,” said Jennifer Gilbert, Co-Founder of the Gilbert Family Foundation.