Gilbert Family Foundation Announces $12 Million Gene Therapy Initiative in NF1

Rare Daily Staff

The Gilbert Family Foundation, a private foundation established by Jennifer and Dan Gilbert, said it has launched a $12 million grant program to support work towards a gene therapy to treat neurofibromatosis type 1.

Neurofibromatosis type 1 (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.

The Gilbert Family Foundation’s goal is to accelerate the development of curative therapies that address the underlying genetic abnormalities in NF1 patients. NF1 drug development has previously focused on addressing the symptoms caused by the disease. This 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.

Over the next three years, the foundation will provide $12 million in research grants to fund several 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 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

“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.”

December 13, 2018
Photo: Dan Gilbert, co-founder of the Gilbert Family Foundation