Who We Are
We are pioneers in understanding GNE Myopathy (GNEM). We have catalyzed every major breakthrough on GNEM in the past 20 years, including gene-hunting, animal models, therapeutic discovery, and clinical trials. We are a community of GNEM doctors, patients, and scientists with a calling to cure GNEM and other muscle wasting illnesses.
What We Do
We are developing gene therapies that target the arm and leg muscles. Our first clinical program involves a non-viral gene therapy approach that allows re-dosing to achieve therapeutic gene expression within the limb skeletal muscles, while protecting the vital internal organs. This approach increases the chance for an effective treatment, reduces the risk of unwanted or life threatening side effects, and further paves the path towards developing curative gene therapies for GNEM and other distal myopathies.