PK10

PharmaKrysto is developing PK10 (L-cystine bis(N'-methylpiperazide)), a small molecule inhibitor of cystine crystal growth for the treatment of cystinuria. Cystinuria is a rare genetic disorder characterized by high concentrations of cystine in the urine, leading to the formation of painful and recurrent kidney stones. PK10 works by binding to the surfaces of developing cystine crystals, disrupting their growth pattern and preventing the formation of larger stones through a molecular recognition mechanism. The technology was licensed from Rutgers University and New York University and has received orphan drug designation from both the European Medicines Agency (EU/3/18/2036) and the FDA. The drug is currently in preclinical development, with PharmaKrysto preparing for first-in-human studies.

Other names

L-cystine bis(N'-methylpiperazide)L-CDNMPLH708LH-708LH 708