Leiden-based Amylon Therapeutics has raised €300.000 from proof-of-concept fund UNIIQ for the development of a RNA-based therapy for rare genetic and incurable disease which leads to strokes at mid-adulthood. This so-called Hereditary Cerebral Haemorrhage with Amyloidosis of the Dutch type (HCHWA-D) is also called the ‘Katwijkse Ziekte’ or ‘Katwijkse Disease’ named after the Dutch city Katwijk. The announcement of the investment was made by Robert Strijk, alderman at the municipality of Leiden.
Patients of the Katwijkse Disease contract cerebral haemorrhages around the age of 40. The culprit is the toxic protein Amyloïde bèta, which damages cerebral veins. Amyloïde bèta is caused by a mutation of the DNA of patients. The disease is sometimes considered to be a ticking time bomb, because the hamorrages can lead to paralysis, dementia and in some cases to dead. Currently there is no treatment for the Katwijkse Disease.
Thomas de Vlaam, CEO and founder Amylon Therapeutics says to StartupJuncture that the funding will be used to further the development of the ‘exon skipping’ technology. De Vlaam: “This is technology will enable us to prevent the production of toxic Amyloïde bèta and therewith te development of cerebral hemorrhages. Right now there are test being done in the preclinical phase. Our focus is to answers questions like: what’s the right dosage and the length of performance of the treatment.” De Vlaam didn’t want to name a timeframe for when the treatment might be available on the market. “This would be pure speculation on my part and I don’t think I would help patients with any speculations.”
If the Amylon Therapeutics succeeds in developing a cure for the Katwijkse Disease its significance can be profound. The protein Amyloïde bèta plays a significant role in causing Alzheimer and Cerebrale Amyloïde Angiopathie, which is the most common form of bleeding in the brain.
Big Unmet Need
Amylon Therapeutics is a spin-off of ProQR. It left the mothership in September. As part of the transaction, ProQR has granted an exclusive license to Amylon to develop therapeutics for beta amyloid related disorders. ProQR retains majority ownership in Amylon and is entitled to future milestones and royalties from the products developed by Amylon.
Vlaam is supported by biotechnology veterans such as Dink Valerio and Thomas Wisniewski. Valerio is the former founder and CEO of Crucell, which was acquired for $2.4 billion in 2011 by Johnson & Johnson and currently the chairman of the supervisory board of ProQR. Wisniewski is director of the Alzheimer’s disease research centre at NYU. Both Valerio and Wisniewski are members of the supervisory board of Amylon Therapeutics.
“I am glad to announce that today we can add another promising biotech company form Leiden to our portfolio,” says Liduina Hammer, fundmanager at UNIIQ. Adding: “Amylon is focusing on a target group with a big unmet need: currently there is no medicine available on the market for the Katwijkse Disease. Our proof-of-concept funding enables Amylon to take the first steps to clinical trials.”
Photo by Hal Gatewood on Unsplash
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