Tu172 - Immune Therapy of CHAPLE Syndrome (Complement Hyper Activation, Angiopathic Thrombosis and Protein Losing Enteropathy Syndrome)

Abstract Text: Problem: CHAPLE Syndrome (Complement Hyper activation, Angiopathic Thrombosis and Protein Losing Enteropathy) is an extremely rare life threatening genetic pediatric disorder. This is due to the deficiency of CD 55 receptor protein that is also known as Decay accelerator Factor, DAF or Cromer Blood Group located at gene chromosome 1 at Q32.2 ). The disease has an over all frequency of (Less than 1 in Million or < 1 / 1 000 000) cases. The total number of cases in USA are less than 10 and globally less than 100 cases are reported. FDA has recently approved the drug Pozelimab (Veopoz) under fast tract rare disease program and has a cost of $ 34,615.38 for single dose vial
The Need: A cost effective solution is urgently needed for this life threatening disease to reduce the burgeoning problem of rare diseases and its medical management cost structure.
Our Work: Based on "the recent advances in Genomics and Fundamentals of Immunology" we have identified a small molecular drug for factor D inhibitor that will reduce cost structure by 10 folds in next 5 years and further 10 folds long term (Kumar Shah, MD CHAPLE SYNDROME, Patent Pending USA) and will require extreme competitive medical management and clinical trials for the positive outcome