NEW YORK – Solve GNE, a nonprofit focused on research and fundraising for GNE myopathy cures, on Monday said it has raised more than $2.5 million to support academic and private sector researchers developing gene therapies for the rare disease.
GNE myopathy, or hereditary inclusion body myopathy (HIBM), is a rare genetic disorder in which mutations to the GNE gene cause progressive muscle weakness, ultimately resulting in loss of mobility. Solve GNE, based in Los Angeles, is doling out the funds to four programs in the hopes of quickly bringing multiple drug candidates to the US Food and Drug Administration.
The four awardees are biotech companies Genosera and Gradalis, both of which are designing gene therapies; a research team at Stanford University developing a novel adeno-associated virus delivery system for gene therapy and a technique for analyzing HIBM disease progression from MRIs of skeletal musculature; and a research team at Johns Hopkins University School of Medicine developing a CRISPR prime-editing approach to correct the genetic mutation.
As part of research agreements, Genosera and Gradalis said they will start in-human clinical trials of their candidates within 12 to 18 months.
Nationwide Children's Hospital in Ohio will be the primary clinical site for the programs that progress to clinical trials, with Stanford as a secondary site on the West Coast. Solve GNE also continues to search for alternative sites in Los Angeles and New York.