Orphina Biotechnology is a private biotech company that grew out of the non-profit organization HIBM Research Group (HRG). Orphina was founded by Dr. Daniel Darvish to accelerate the development of treatments for orphan diseases. We are a clinical-stage biologics development company. Our current focus is to develop treatments that are considerably superior to placebo. We have completed IND-enabling studies for our lead product (H001). We believe that rapid innovation is the most rewarding business model for creating meaningful value for all stakeholders.
What Is GNE Myopathy?
Unmet Medical Need
Currently, there is no approved intervention for GNEM, and treatment is limited to supportive or palliative care. Early diagnosis helps patients receive current optimal care. Periodic physical therapy sessions, along with a balanced physical activity, may slow progression in some patients.
Potential for Human Health
Orphan Drug Act of 1983 makes a good business decision to have a for-profit company because it increases the chance of success and suitability by a large margin based on historical data. We can reinvest the tax credit back into the company for more rapid innovation of treatments.
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