Solve GNE has been actively raising funds to support a consortium of academic and private sector researchers tasked with the development of various gene therapies
HIBM is a muscular disorder that causes progressive muscle weakness. (Credit: Towfiqu barbhuiya on Unsplash)
Solve GNE, a Los Angeles based non-profit organization, has raised over $2.5m and negotiated sponsored research agreements with multiple leading medical researchers to help develop a cure for Hereditary Inclusion Body Myopathy (HIBM).
Also known as GNE Myopathy (GNEM), HIBM is a muscular disorder that causes progressive muscle weakness which manifests in early adulthood – generally starting with the legs – until reaching eventual complete loss of mobility. While rare, this disease has a remarkably high incidence among Iranians of Jewish descent – with an estimated 10 to 15% of the population carrying at least a single copy of the mutated gene.
Solve GNE has been actively raising funds to support a consortium of academic and private sector researchers tasked with the development of various gene therapies. Solve GNE has committed to funding experts in HIBM in four leading organizations: Genosera, Gradalis, Johns Hopkins University School of Medicine and Stanford University. These agreements include detailed development budgets and a commitment from Genosera and Gradalis to start human clinical trials within 12 to 18 months.
The Solve GNE consortium is focusing on a common goal but implementing a parallel track strategy that should allow them to approach the FDA with multiple options to get patients enrolled in Phase 1 trials as rapidly as possible.
Additionally, Solve GNE has already identified Nationwide Children’s Hospital in Ohio as their primary clinical site. Stanford has agreed to serve as the secondary clinical site on the West Coast. Solve GNE is still researching alternative clinical sites in Los Angeles and New York in order to facilitate enrollment of patients who live in those cities.
Additional details will be provided by Solve GNE and researchers from Stanford, Johns Hopkins, Gradalis and Genosera during a global webcast presentation followed by a Q&A session on Sunday September 10th, 2023 at 11A New York time (8A Los Angeles time, 6P Tel Aviv time). For those who are unable to attend the live presentation, a recording of the webcast will be made available.
Source: Company Press Release