Scotland-based biotech company PharmaKrysto, http://www.pharmakrysto.co.uk/ announced it would undertake a research collaboration with Rutgers University in New Jersey “to develop patient trials for PK10, a ‘potentially revolutionary’ compound to treat people with cystinuria-an inherited disease that causes large, painful and debilitating crystals of amino acid to form in the kidneys.”
First a digression to introduce cystinuria. A rare disorder, It affects 1 in 10,000. The orphan drug alpha- mercaptopropionyl glycine, also known as tiopronin (Thiola) has been approved as a treatment for cystinuria. This drug is manufactured by Mission Pharmacal. Thiola has been shown to lower the level of cystine in the urine of patients with cystinuria. Another medicine that is used at times is captopril.
It is reported that the company received a clearance by both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). TrialSite News staff learned through the preliminary research that the company is actively trying to raise investment funds to prepare for patient trials. From what we understand, cystinuria is a debilitating ailment and the success of Mr. Howell’s fundraising hopefully can lead to a treatment in the future.
View article hereSource: Herald Scotland