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A community dedicated to finding a cure for GNE Myopathy

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Advances in Hereditary Inclusion Body Myopathy

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Press Release September 29, 2026

Solve GNE and Vecgen Awarded NIH Fast-Track STTR Grant to Develop a Diagnostic Predicting Immune Response to AAV Gene Therapy for GNE Myopathy

The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) has funded a joint Solve GNE–Vecgen project to validate a test that predicts, before treatment, how a patient's immune system will respond to an AAV gene-therapy vector — a key step toward safer patient selection for a GNE myopathy gene therapy, with potential reach across other AAV-based programs.

  • $333,689 Phase 1 funded & underway
  • ~$2M Phase 2 anticipated from Sept 2027
  • NIAMS Awarding NIH institute

Only Phase 1 funding has been obligated by the NIH. Phase 2 amounts are anticipated, not committed, and depend on NIH milestone review and availability of funds. Research reported here is supported by NIAMS of the National Institutes of Health under Award Number R42AR087918; content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH.

GNE Myopathy (aka Hereditary Inclusion Body Myopathy or HIBM) is an ultra rare genetic disorder that affects roughly 1 in 5 million individuals worldwide.

Patients with GNE Myopathy suffer from progressive muscle weakness and unfortunately become wheelchair bound as the disease progresses. HIBM is highly prevalent in the Iranian Jewish community, with almost 1 in 10 individuals having a single copy of the mutated gene known as M712T (or M743T).

GNE Myopathy is an autosomal recessive disease, meaning two copies of the mutated gene must be present in order for the symptoms to manifest. Married couples who are both asymptomatic carriers have a 75% chance of passing the defective gene to one of their children.

Research

We are collaborating with some of the world’s leading experts in order to streamline and advance the most cutting edge discoveries from the laboratory to the clinic.

A decade ago if someone talked about inserting a gene into a human cell, or removing and correcting a single mistake in our DNA with surgical precision, most scientists would have laughed. What was science fiction then is reality now.

Join us on this journey and follow our progress as we continue to update the community with our latest developments and success stories.

Johns Hopkins University School of Medicine Researchers spearheaded by Dr. Greg Newby are the recipients of a multi-year grant from Solve GNE. 

Using latest-generation CRISPR tools – base editors and prime editors – the team will attempt to directly correct the genetic mutation in laboratory models.

Collaborations

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Advancing a new therapy through human clinical trials is a complex, expensive and time consuming process that involves the coordinated efforts of patients and their families, physicians, scientists and federal officials.

This mountain may seem tall and out of reach but it’s certainly not impossible to summit.

With careful planning and proper funding we CAN find a path forward and thanks to the latest advances in molecular biology, we can reach our goal in a reasonably short period of time.

Blogs

Single HIBM Patient Treated

Gregory Nemunaitis, John Nemunaitis and colleagues from the Mary Crowley Cancer Research Centers in Texas have reported the first and only example of a single

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Dr. Paul Martin

Dr. Paul Martin and colleagues at Nationwide Childrens Hospital in Ohio have been exploring a novel approach to treating GNE myopathy using a bicistronic vector

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Prof. Stella Mitrani-Rosenbaum

Professor Stella Mitrani-Rosenbaum from Hadassah University Medical Center is a leading international expert in GNE Myopathy and has published extensively on the topic. Read more

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