About Orphina

Founded by pioneers in understanding and catalyzing progress in GNE Myopathy (GNEM), …

Orphina's Name

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.

Our Misson

Using both novel and ignored technologies to maximize therapeutic benefit to human quality of life.

Our Vision

Build a top-tier independent biotechnology company that gathers the resources needed to accelerate the clinical development of very effective treatments.

Our Core Values

Commitment to Patients, Innovation and Discovery, Sense of Urgency, Open Culture, Passion for Excellence.