<img alt="" src="https://secure.hiss3lark.com/174537.png" style="display:none;">

Cystinuria Orphan Drugs

Company Presentation

Company Description

PharmaKrysto is an early-stage research and development company — our mission is to develop new treatments for people with Cystinuria, and improve their quality of life. There is a group of adults and children who experience severe, frequent pain and complicated kidney disease, caused by deposits of large amino acid crystals in their kidneys. This medical condition is called Cystinuria. Our goal is to improve the quality of life of people with Cystinuria.

This rare, inherited disease affects children and adults, causing recurrent, large kidney stones that are hard to treat; resulting in frequent pain, repeated surgeries and kidney damage.

We will do this by developing a novel, patented molecule that has already shown great potential in animal models in stopping stones from forming. The current treatments for this condition are poorly effective and have significant side effects. Our development plan makes full advantage of the significant unmet need and Orphan status. We anticipate reaching commercial sales within 6 years of funding and achieving peak sales of over £150M just within adult US and core EU markets.