H001

About Orphina Biotechnology

Orphina Biotechnology is a private, pre-revenue company founded in 2021 with a mission to cure GNE Myopathy and other muscle-wasting illnesses. The company's lead program, H001, is a non-viral gene therapy for GNEM designed for re-dosability and targeted delivery to limb muscles, currently in the pre-clinical/IND-enabling stage. Orphina's strategy leverages deep community engagement with patients and clinicians to advance a pipeline that addresses significant unmet needs in rare neuromuscular diseases. The company is led by its founder, Dr. Daniel Darvish, who is both a physician and a patient, driving a patient-centric R&D approach.

Therapeutic Areas

Other GNE Myopathy (GNEM) Drugs