Founded by pioneers in understanding and catalyzing progress in GNE Myopathy (GNEM), …
Orphina is derived from “Orphan”, defined as “one deprived of protection or advantage”. We are the reason justifying the need for the Orphan Drug Act, and the need for rapid innovation towards development of cures.
Using both novel and ignored technologies to maximize therapeutic benefit to human quality of life.
Build a top-tier independent biotechnology company that gathers the resources needed to accelerate the clinical development of very effective treatments.
Commitment to Patients, Innovation and Discovery, Sense of Urgency, Open Culture, Passion for Excellence.
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.