Solve GNE Highlights New Research, Expands Funding Efforts, and Advances Toward a Phase I Clinical Trial for GNE Myopathy
LOS ANGELES, March 16, 2026 (GLOBE NEWSWIRE) -- Solve GNE, a nonprofit foundation dedicated to accelerating treatments for GNE myopathy, has announced major progress across its research, fundraising, and regulatory efforts. To date, the non-profit has raised more than $6.5 million in support of programs aimed at moving clinically viable therapies closer to approval.
A newly published preclinical paper highlights the recent progress in one of our clinical development programs for GNE myopathy1. The study, which was carried out in collaboration with Gradalis Inc., describes a series of in vitro and in vivo data associated with a dual-function plasmid called GNEwt/bi-shRNA-GNEM743T lipoplex, designed to simultaneously deliver the wild-type GNE gene (GNEwt) and shut off the expression of GNEM743T, a GNE mutation that is highly prevalent in the Iranian Jewish community. Gradalis researchers showed that this approach leads to clinically relevant expression levels of functioning GNE protein as well as increased sialic acid production. The lack of sialic acid production is a hallmark of GNE Myopathy and is suspected to be the leading cause of muscle loss in these patients. Animal models showed that the engineered plasmid was successfully delivered to muscle tissue after intravenous administration using a DOTAP-cholesterol lipoplex formulation. Together, these findings support continued development of this gene therapy and help lay the foundation for future clinical testing in GNE myopathy.
Solve GNE and its collaborators are also pursuing several grant opportunities to support the next phases of research and development in GNE Myopathy. These grant applications are focused on the development of safer AAV based gene therapies, as well as novel blood-based biomarkers, gene editing approaches, the validation of a diagnostic platform to help predict immune responses to AAV-based therapies and further supporting the development of a clinically scalable dbRNA lipoplex treatment. If successful, these grant applications represent approximately $10.2 million in potential federal funding with the possibility of additional funding in the future.
Funding from Solve GNE is also being directed at the filing of an IND application by Gradalis Inc. in 2026. The FDA reviewed and provided a series of comments to our pre-IND application. Our team is currently finalizing the IND application for the first gene therapy targeting GNE Myopathy and we anticipate that a Phase I clinical trial may begin within the next 12-months.
Solve GNE remains focused on turning scientific progress into real momentum for GNE Myopathy patients and their families. Our commitment to funding additional research will help build a stronger path toward future treatment options for this rare disease.
About Solve GNE
Solve GNE is a nonprofit foundation dedicated to advancing research and treatment development for GNE myopathy. Through fundraising and scientific partnerships, Solve GNE works to accelerate progress toward therapies for patients and families affected by this rare muscle disease.
Media Contact:
Alexander Monsef
AlexM@solveGNE.org
____________________________
1 Preclinical assessment of GNEwt/bi-shRNA-GNEM743T lipoplex product development for GNE myopathy. Future Science OA, 12:1, 2635722, DOI: 10.1080/20565623.2026.2635722
Legal Disclaimer:
EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.
