Investor Relations

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?

GNE myopathy (GNEM), also known as HIBM, Nonaka myopathy, IBM2, or distal myopathy with rimmed vacuoles, is a genetic muscle-wasting disease. The first signs of the disease appear between 20 and 40 years of age and affect males and females equally. This condition is characterized by progressive muscle weakness which typically worsens over time, decreased grip strength, and frequent loss of balance. GNEM often leads to becoming wheelchair-bound or bedridden by 30 to 50 years of age.

Our Technology

Our technology is designed to increase the expression of the GNE enzyme and production of N-Acetylneuraminate (NeuAc) within the skeletal muscles of limbs to normal or above normal levels. Our technology further protects the vital internal organs from unnecessary exposure to increased GNE expression or sialic production. This approach increases the chance for an effective treatment, reduces the risk of unwanted or life-threatening side effects, and further paves the path towards developing curative gene therapies for GNEM and other distal myopathies.

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

Tax Incentives

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.

For further information, please email us at investors@orphina.com