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