To eradicate the negative consequences of NF1 via drug treatment
The mission of iNFixion Bioscience, a San Diego based drug discovery company, is to develop drug therapies to treat and prevent the progression of all symptoms associated with NF1, a rare genetic disorder impacting approximately 1 in 3000 people worldwide.
NF1 is caused by one of many possible genetic mutations in the NF1 gene. Mutations in this gene can be inherited (50% of cases), or can result from a spontaneous new mutation at conception. The NF1 gene is responsible for making a large and multifunctional protein called Neurofibromin which is produced in many cells throughout the body. Neurofibromin is known to regulate many important functions, including the control of cell division (i.e. tumors), as well as the proper functioning of brain, skeletal, muscular- and cardiovascular systems. As a consequence of NF1 mutations, tumors can grow along nerves anywhere in the body and other serious symptoms such as skin lesions, neurocognitive/social deficits, chronic pain, and bone abnormalities are common. Furthermore, people with NF1 mutations have an increased risk of both cancer and cardiovascular disease.
iNFixion’s unique approach to NF1 is to correct a key underlying genetic ‘mechanism of action’, namely the lack of functional Neurofibromin protein, by enhancing the affected person’s natural ability to both produce and maintain healthy Neurofibromin protein. By correcting this root cause mechanism, we believe that a fundamental and preventive impact on disease symptoms, and disease progression, will be achieved.