Orphina LLC is a pioneering biotechnology company based in Chatsworth, CA, dedicated to preserving and prolonging muscle health. With a focus on understanding and finding solutions for GNE Myopathy (GNEM) and other muscle wasting illnesses, Orphina has been at the forefront of major breakthroughs in gene-hunting, animal models, therapeutic discovery, and clinical trials.
Driven by a strong community of GNEM doctors, patients, and scientists, Orphina is developing gene therapies that aim to preserve and prolong muscle health. Their innovative non-viral gene therapy approach allows for re-dosing to achieve therapeutic gene expression within limb skeletal muscles while safeguarding vital internal organs, increasing the chances of developing safe and effective treatments for muscle diseases.
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