There is a group of adults and children who experience severe, frequent pain, kidney damage and a risk of kidney failure, caused by deposits of large amino acid crystals in their kidneys, bladder and urinary tract. This chronic medical condition is called Cystinuria.
PharmaKrysto is an early-stage research and development company with a mission to improve the outcomes for Cystinuria sufferers by saving kidney function and preventing unwanted pain.
Cystinuria is the result of faulty genes causing kidneys to leak amino acids into urine. High concentrations of an amino acid called cystine –a building block of most proteins – are left unabsorbed in the urine. The cystine then crystallises in the kidneys, bladder and urinary tract, forming large crystal-stones which block the flow of urine, causing intense pain and infection. Surgery is often the only way of removing them and this can damage the delicate structure of the kidneys, severely affecting function. Repeated infection can cause progressive and permanent kidney failure.
Cystinuria is a chronic condition affecting patients of all ages, with sufferers having many kidney stone events each year, causing significant disruption to their work, educational and family life. There are about 45,000 people in the US and 100,000 people in Europe with Cystinuria. Most patients have their first experience of cystine stones causing pain and infection in their teens or early 20’s.
Patients have not benefitted from any new Cystinuria treatments for over 30 years. Current methods used old technology to prevent cystine stones which are only effective in up to half of the patients that take them. These older treatments also have significant side effects, which can include skin rashes, as well as liver and bone marrow disorders.
PharmaKrysto’s mission is to prevent pain and the risk of losing kidneys in people with Cystinuria. We have been working with leading scientists who have discovered a completely new method of blocking the growth of cystine crystals.
This patented technology exclusively available to PharmaKrysto – which has been granted orphan drug designation in both the USA and Europe – works in a completely novel way to current therapies and we believe it could be further developed to become an easy-to-use frontline therapy, without either the complexity or severe side effects of current treatments.
We are a highly experienced team of pharmaceutical industry professionals with deep and broad knowledge of novel product development and commercialization. We were inspired to set up PharmaKrysto when we learnt about the experiences of Cystinuria patients and how a new and effective therapy could change their lives in a positive way.
We are now preparing the technology for the first studies in humans, through developing the manufacturing process and adding to our knowledge of the mechanism of action and preclinical safety of the molecules we have developed for candidate selection.
Jackie, Julian and Carl are the founders of PharmaKrysto and have worked together on various projects over the last 15 years. Their collective experience covers all the major aspects of product development and commercialisation with experience gained in companies ranging from small bio-pharma through to some of the largest pharmaceutical companies in existence.
With more than two decades of industry experience, Julian trained as a doctor in the UK before starting his industry career in pharmaceutical development with SmithKline Beecham and Roche and more recently as the Chief Medical Officer and CEO at smaller biopharma companies.
Julian has been involved in multiple new medicines approvals in the US and EU and has led development teams across a range of therapy areas. He completed an MBA at Cranfield University.
Carl, who originally trained as a biochemist and toxicologist, also has more than two decades of senior, executive and non-executive board level experience in pharmaceutical development and commercialisation.
Previously, Carl held leadership roles at United Therapeutics and Encysive Pharmaceuticals, working on innovative therapies for the treatment of pulmonary arterial hypertension, after which he founded Shield Therapeutics and for 10 years led the financing, acquisition, development, approval and initial commercialisation of the company’s lead product, Feraccru.
Jackie has over 20 years’ experience in regulatory affairs and early-stage development. She holds an MA in biochemistry from Oxford University, where she also obtained a doctorate in immunology and molecular biology.
Jackie has worked in senior roles in regulatory affairs and clinical development for large, medium and small pharmaceutical companies, including Boehringer Ingelheim and Abbott. She has been involved in and led a broad range of global, EU and national new medicines approvals in the USA and Europe, including Kaletra, Humira and Feraccru/Accrufer.
For more information please contact:
info@pharmakrysto.com