Gilbert Foundation invests $18M toward Gene Therapy Initiative
The Gilbert Family Foundation, a private fund established by Dan and Jennifer Gilbert, announced Thursday it is funding more than $18 million in grants toward a cure for Type 1 neurofibromatosis.
The money will fund a three-year campaign as part of the organization's Gene Therapy Initiative. Four grants totaling $4.4 million will be invested into further developing research that initially began in 2018, while the additional $13.8 million will be invested into 12 new research projects.
Commonly referred to as NF, neurofibromatosis is a condition that causes tumors to form on nerves anywhere in the body. Type 1 usually appears in childhood, while types 2 and 3 appear in early adulthood.
The Gilberts have been passionate advocates in the fight to end NF since their oldest son, Nick, was born with NF1. Diagnosed at 15-months, Nick has suffered through multiple brain surgeries, loss of vision and rounds of chemotherapy. In 2018, he underwent an eight-hour operation and spent more than a month in recovery. The disease can be inherited, but none of the Gilbert's other four children have been diagnosed.
Jennifer Gilbert, who co-founded the Gilbert Family Foundation in 2015 to increase NF research, said they are committed to finding a cure for the disease. Since then, the family has committed $72.5 million to curing NF1.
"NF1 affects 1 in 3,000 people throughout the world, including our son Nick. The progress from our last research cycle and the promise of this one continues to give us hope that anyone enduring NF1 will see a cure in our lifetime," Jennifer Gilbert said in a statement.
The foundation launched its Gene Therapy Initiative in December 2018 to fund research focused on developing therapies that address the underlying cause of NF1. Historically, NF1 drug development addressed only the symptoms caused by the disease, demonstrating the need for organizations like the Gilbert Family Foundation, Children’s Tumor Foundation and others who are working toward a cure.
The Gene Therapy Initiative is researching to develop various types of therapies, including gene replacement, gene editing, RNA editing, exon skipping and nonsense mutation suppression, officials said. GTI is also invested in developing methods to improve the accuracy and efficiency of delivering NF1 gene therapies to their intended parts of the body.
Type 1 NF can cause bone deformities, learning disabilities and high blood pressure. Type 2 can cause hearing loss, vision loss, and difficulty with balance. Type 3 can cause chronic pain throughout the body.
Their lead Ph.D. researchers include Charles Gersbach of Duke University, Robert Kesterton of the University of Alabama at Birmingham, Casey Maguire of Massachusetts General Hospital and Miguel Sena-Esteves of the University of Massachusetts.