April 28, 2022 [DETROIT, MI] – The Gilbert Family Foundation, a private foundation established by Jennifer and Dan Gilbert to accelerate a cure for neurofibromatosis type one (NF1) and build economic opportunity and equity in the city of Detroit, today announced more than $18 million in grants to fund a new three-year campaign as part of the organization’s Gene Therapy Initiative (GTI).

Four grants totaling $4.4 million will be invested into further developing initial research that began in 2018, while an additional $13.8 million will be invested into 12 new research projects.

“We are committed to finding a cure for neurofibromatosis by supporting remarkable researchers and physicians as part of our Gene Therapy Initiative,” said Jennifer Gilbert, co-founder of the Gilbert Family Foundation. “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.”

The researchers continuing from the 2018 cohort include:

  • Charles Gersbach, PhD, Duke University
  • Robert Kesterton, PhD, University of Alabama at Birmingham
  • Casey Maguire, PhD, Massachusetts General Hospital
  • Miguel Sena-Esteves, PhD, UMass Chan Medical School

“The Gilbert Family Foundation’s support of a broad exploration of molecular therapeutic approaches for NF1 has yielded many promising avenues for further development,” said Dr. Sena- Esteves. “Previously, we developed an adeno-associated virus gene therapy approach in an animal model of NF1. I am excited to enter this next phase of the project so that we may demonstrate the efficacy and safety of AAV-NF1 gene therapy, and support the first ever AAV gene therapy clinical trial for NF1 patients.”

The new GTI researchers include:

  • Allison Bradbury, PhD, Abigail Wexner Research Institute at Nationwide Children’s Hospital
  • D. Wade Clapp, MD, Indiana University School of Medicine
  • Cherry Gupta, PhD, Battelle Memorial Institute
  • Matthew Hartman, PhD, Virginia Commonwealth University
  • Natalia Higuita-Castro, PhD, Ohio State University
  • Gabsang Lee, PhD, DVM, Johns Hopkins University
  • Leszek Lisowski, PhD, University of Sydney, Children’s Medical Research Institute
  • J. Elliott Robinson, MD, PhD, Cincinnati Children’s Hospital Medical Center
  • Kenneth Sims, PhD, Battelle Memorial Institute
  • James Walker, PhD, Massachusetts General Hospital
  • Deeann Wallis, PhD, University of Alabama at Birmingham
  • Yi-tao Yu, PhD, University of Rochester

“Gene therapies have always shown a tremendous promise for the correction of NF1, however, proper deployment of such therapies remains a significant challenge,” said Dr. Higuita-Castro. “The advancements derived from our work will allow us to deliver different types of NF1 gene therapies, which will be key to enabling collaborations with the wider NF1 research and clinical care community to help find an effective treatment for this condition.”

This new round of commitments brings the Gilbert Family Foundation’s total investment into curing NF1 to $72.5 million, with additional grant announcements to come later this year.

The Gilbert Family Foundation launched GTI in December 2018 to fund research focused on developing therapies that address the underlying cause of NF1, which causes tumors to form on nerves anywhere in the body.  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.

GTI supports research to develop various types of therapies, including gene replacement, gene editing, RNA editing, exon skipping, and nonsense mutation suppression.  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.