From left to right: Tim de Jong (UNIIQ), Ivo de Nooijer (LURIS), Tsinat Hailu (Director nonclinical Development Amylon Therapeutics), Robert Strijk, Alderman for Accessibility, Economic Affairs, City Centre and Culture City of Leiden, Liduina Hammer (UNIIQ)
Katwijkse Ziekte and CAA
Amylon Therapeutics develops a therapy against Katwijkse Ziekte, formally called Hereditary Cerebral Hemorrhage with Amyloidosis, Dutch Type (HCHWA-D). Katwijkse Ziekte is a heritable orphan disease which originates from the Dutch city of Katwijk. Currently, there is no therapy for the Katwijkse Ziekte and there is no effective therapy available to prevent the development of the disease. Patients, between 40 and 60 years of age, suffer of severe bleedings in the brain as a result of the development of Amyloïd bèta plaques in the blood vessels walls. Depending on the location and the impact of these bleedings, they can lead to paralysis, dementia, and even death. The progression of the disease is significant: after every event, the risk of new bleeding increases and the condition of the patient worsens with each bleeding. On top of that, the moment of the next bleeding is unpredictable.
I am delighted that with its investment UNIIQ provides Amylon Therapeutics the opportunity to continue the development of a therapy against Katwijkse Ziekte and making the next step towards clinical development – Thomas de Vlaam, founder and CEO Amylon Therapeutics
Katwijkse Ziekte is a special (hereditary) variation of Cerebral Amyloid Angiopathy (CAA), a disease which is known to cause multiple hundreds of thousand of patients worldwide, which are affected by or die from the consequences of CAA every year. Another difference between Katwijkse Ziekte and CAA, apart from the fact that CAA is not hereditary, is that the time of onset of CAA is unpredictable. Besides these differences both diseases are similar. Therefore a development of a successful therapy against Katwijkse Ziekte could be a promising step towards finding a therapy for CAA and potentially Alzheimer’s disease, a disease which is also characterized by the formation of plaques in the brain.
Next step towards clinical development
Thomas de Vlaam: “Due to the hereditary dominant mutation we know for sure that Amyloïd bèta plaques will be formed in the brains of patients. By using our “exon skipping” technology Amylon will be able to stop the formation of the Amyloïd bèta. If we can stop the formation of the plaques we expect to stop the bleedings in the blood vessel walls as well.”. De Vlaam continues: “I am delighted that with its investment UNIIQ provides Amylon Therapeutics the opportunity to continue the development of a therapy against Katwijkse Ziekte and making the next step towards clinical development. Until now there is no cure or therapy for patients with Katwijkse Ziekte, we strive to make a change and help these people.”
“Today we have added another promising Leiden based biotech comapny to our portfolio”, mentioned Liduina Hammer, UNIIQ Fund manager. “By focusing on the development of a therapy for Katwijkse Ziekte Amylon will solve an unmet need: there is no cure or therapy against Katwijkse Ziekte. Using the UNIIQ investment to proof its concept Amylon will be able to make a step towards clinical development”.
Amylon Therapeutics was founded by the ambitious entrepreneur Thomas de Vlaam who is supported by experienced and successful biotechnology entrepreneurs such as founder and former CEO of Crucell, Dinko Valerio. In addition, as ProQR Therapeutics spin-off, Amylon Therapeutics has the profound expertise and experience of its shareholders at its disposal.