Gene Therapy Initiative

NF1 results from mutations or deletion of the neurofibromin 1 (NF1) gene, which encodes a negative regulator of the Ras signal transduction pathway called neurofibromin. To date, drug development has primarily focused on addressing the molecular abnormalities that arise downstream of the NF1 gene mutation/deletion. There are no therapies that address the underlying issue of this genetic disease; present therapies do not cure the disease. Thus the mission of the Gilbert Family Foundation’s Gene Therapy Initiative (GTI) is to develop curative therapies that address the underlying genetic abnormalities in NF1 patients.

In its first phase, the Gene Therapy Initiative will explore the feasibility of various gene-targeting therapeutic strategies for NF1 as well as novel or enhanced in vivo gene delivery systems. Areas of interest thus include:

  • Gene replacement
  • Gene editing
  • RNA editing
  • Exon skipping
  • Mutation suppression
  • Gene delivery systems

Initial Gene Therapy Initiative awards were announced in December 2018 and the second cycle will be announced in January 2022.

Development Pipeline for Funded Projects

Discovery
(21)
In Vivo Proof of Concept
(4)
IND Enabling
(0)
Clinical Trial Phase 1
(0)
Clinical Trial Phase 2
(0)
Clinical Trial Phase 3
(0)

Pre-clinical development of AAV9-NF1 gene therapy for neurofibromatosis type 1

In Vivo Proof Of Concept

RAFT-polymerized nanoparticles for delivery of NF1 cDNA to Schwann cells

Discovery

Suppression of NF1 Nonsense Mutations by RNA-guided RNA Pseudouridylation

Discovery

Targeted delivery of full length NF1 cDNA using non-viral vectors

Discovery

Testing the efficacy of new NF1 gene therapy candidates in human cell types

Discovery

GTI Advisory Board

Barry Byrne, MD, PhD

University of Florida

Leah Byrne, PhD

University of Pittsburgh

School of Medicine

Jeffrey Chamberlain, PhD

University of Washington

Manuela Corti, PhD

University of Florida

Kleopas Kleopa, MD

Cyrus Institute of Neurology and Genetics

Verena Staedtke, MD, PhD

Johns Hopkins University

School of Medicine

Barry Byrne, MD, PhD

University of Florida

Leah Byrne, PhD

University of Pittsburgh

School of Medicine

Jeffrey Chamberlain, PhD

University of Washington

Manuela Corti, PhD

University of Florida

Kleopas Kleopa, MD

Cyrus Institute of Neurology and Genetics

Verena Staedtke, MD, PhD

Johns Hopkins University

School of Medicine