Developing technologies to improve muscle function
We are passionate about preserving and prolonging muscle health. We are pioneers in understanding GNE Myopathy (GNEM). We have catalyzed every major breakthrough on GNEM, 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.
We are developing gene therapies for preserving and prolonging muscle health. Our first clinical program involves a non-viral gene therapy approach that enables re-dosing to achieve therapeutic gene expression within the limb skeletal muscles, while protecting the vital internal organs. Our approach increases the chance for successful development of safe and effective treatments, and further paves the path towards developing curative gene therapies for muscle diseases.
Orphina is a biotechnology company at seed stage, focused on preserving and prolonging muscle health. Our initial clinical gene therapy program is for a rare disease indication of unmet medical need. Our technology allows re-dosing while limiting exposure to vital organs, thereby increasing therapeutic effect and reducing the risk of severe or life-threatening side effects.