Polycystic kidney disease (PKD) affects kidney along with other organs, in which development of cyst interrupts the ability to filter the waste products from blood. Various symptoms are being observed in the patients suffering from the disease; such as pain in the abdomen, back or flank, and it is thought to be caused by enlargement of the cysts in the kidney, bleeding, urinary tract infection, renal colic, or coincidental hypernephroma. The main feature observed in the patients with Autosomal Dominant Polycystic Kidney Disease (AKPKD) is bilateral progressive increase in the number of cysts, which may lead to end stage renal disease. It has been hypothesized that cells are protected by the allele inherited from the parent without ADPKD due to which hepatic cysts, cerebral aneurysms, and cardiac valvular abnormalities may also occur.
PKD THERAPEUTICS UNDER DEVELOPMENT (2017)
There are many pharmaceutical companies that are using different technologies for better development of drug candidates. These technologies can be helpful in the development of drugs as combination therapies or single agent therapies. They also allow the development of specific targeted therapies with better drug delivery. For instance, Syros’ technology platform dedicated to the regulatory genome to systematically identify disease-causing alterations in gene expression and create medicines that control the expression of genes with the aim of providing a profound and durable benefit for patients with diseases that have largely eluded other genomics-based approaches.
As of November 2017, the polycystic kidney disease therapeutics pipeline comprises of approximately 16 drug candidates in different stages of development.
Some of the key players developing drugs for the treatment of PKD therapeutics include Otsuka Pharmaceutical Co., Ltd., Exelixis, Inc., and XORTX Pharma Corp.