Lupus Nephritis (LN) is the most common cause of morbidity and mortality in SLE. 50% of the 150-350k US SLE patients progress to or have LN. Focal Segmental Glomerulosclerosis (FSGS) is the major cause of nephrotic syndromes (NS) with a US prevalence of over 40,000. There are no FDA or EMA approved therapies
Amyotrophic Lateral Sclerosis (ALS)
ALS is a progressive neurodegenerative disease resulting from loss of motor neurons, muscle atrophy and subsequent paralysis. Median survival after symptom onset is 3 to 5 years. Current treatments have a minimal effect on slowing disease progression or improving survival.
Solid Organ Transplant
There are currently 23,000 renal & 9,000 liver transplants in the US per year, increasing by 10% annually. Current standard of care immunosuppression is associated with nephrotoxicity and kidney failure. 20% of yearly renal transplants are re-transplant due to transplant rejection.
Islet Transplant in Type 1 Diabetes (T1D)
Type 1 Diabetes (T1D) is an autoimmune disease with progressive loss of insulin-producing Beta cells in the pancreas and uncontrolled hyperglycemia. There is no effective treatment or cure.