ORPHINA

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... 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..

Also known as: Orphina Biotechnology

Primary location: Calabasas United States