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 pre-startup biotechnology company focused on the development of therapy for orphan diseases. Our current focus is preserving and improving muscle function through targeted proprietary gene therapy. Our technology allows treatment re-dosing while protecting vital internal organs from unnecessary treatments, thereby reducing the risk of severe or life threatening side effects.