Two Japanese medical institutions are planning to develop tailored drugs for patients with genetic disorders in the hope of finding treatment for extremely rare diseases, a leading project member said Sunday.
The project involving Tokyo Medical and Dental University and the National Center of Neurology and Psychiatry will search for rare diseases that have the potential to be treated by nucleic acid drugs, a new emerging class of therapeutics, from a database of around 2,250 patients.
The development will be carried out based on technology that targets the expression of disease-causing proteins in the mutated gene. Pharmaceutical companies are reluctant to develop medicines for extremely rare diseases because of the costs and difficulties regarding business viability. Such drugs have already been administered in the United States.