Goldfinch’s therapeutic programs are underscored by strong genetic validation and a deep familiarity of the biological pathways that cause disease. Our initial efforts are focused on focal segmental glomerulosclerosis (FSGS) and polycystic kidney disease (PKD).
FOCAL SEGMENTAL GLOMERULOSCLEROSIS (FSGS)
Focal Segmental Glomerulosclerosis (FSGS), a rare kidney disorder, is a histopathologic diagnosis characterized by severe scarring of the kidney's filtering units, or glomeruli, leading to an excess of essential proteins spilling into the urine, called proteinuria. FSGS is a leading cause of kidney failure, and patients with diagnosed FSGS are at increased risk of heart attack, stroke and premature death.
There are currently no FDA approved treatments for FSGS. Existing treatments are non-specific, do not alter the progression of disease and carry significant toxicities.
Goldfinch is leading the charge to develop treatments targeting the molecular causes of FSGS to drastically improve the lives of patients suffering from this devastating, life-shortening condition.
POLYCYSTIC KIDNEY DISEASE (PKD)
Autosomal dominant polycystic kidney disease (ADPKD) is one of the most common single gene diseases, affecting more than 600,000 people in the US and 12 million worldwide. The disease is passed on from parent to child, with each child of an affected parent having a 1-in-2 chance of inheriting the disorder, which does not skip generations.
ADPKD patients develop fluid filled cysts in both kidneys that increase in number and size over time, culminating in end stage kidney disease in half of affected individuals by middle age. ADPKD commonly causes liver cysts which arise from bile ducts and can sometimes grow large enough to cause symptoms as well. In the United States, 1 in 20 individuals either on dialysis or with a kidney transplant has ADPKD.
There are no FDA approved therapies specifically targeting ADPKD and no means by which to alter the course of the disease.
Goldfinch is leveraging scientific discoveries based on the known genes that cause ADPKD to discover treatments to alter the course of ADPKD and drastically improve the lives of patients suffering from this chronic and debilitating disease.