ProQR has spun out Amylon Therapeutics, a company that will treat central nervous system (CNS) disorders with a lead candidate based on RNA technology.
Amylon Therapeutics’ lead program, AT-010, is an RNA-based treatment that modulates the splicing of the mRNA that encodes the amyloid precursor protein, resulting in a protein that lacks the amyloid-beta (Aβ) peptide, which can cause disease due to its tendency to aggregate.
HCHWA-D (Katwijk’s disease)
Amylon will further develop ProQR’s research into beta amyloid related disorders, initially by going after the stroke-causing disease hereditary cerebral hemorrhage with amyloidosis of the Dutch type (HCHWA-D) or Katwijk’s disease. Further down the line, Amylon plans to target other beta amyloid related disorders, such as cerebral amyloid angiopathy (CAA). There is currently no treatment for both of the diseases.
Treating HCHWA-D, a rare genetic disease
That rough R&D strategy follows the well-trodden path of treating a rare genetic disease before expanding into a broader, related indication. HCHWA-D is a genetic subtype of CAA, a disorder that also causes strokes but isn’t confined to people with certain mutations. CAA affects anyone with buildups of beta amyloid in their brain’s vascular system.
If Amylon’s science works in the well-defined HCHWA-D patient population, it may also help the many more seniors and Alzheimer’s disease patients who are at risk of stroke.
Founder Thomas de Vlaam
The Netherlands-based ProQR is facilitating this work by granting Amylon exclusive license to develop treatments for beta amyloid related disorders in return for milestones and royalties. ProQR has taken a majority stake in Amylon, but has also enlisted the support of institutional and private investors to put together seed funding for the startup. The company will be led by CEO and founder Thomas de Vlaam, who played a key role in the development of the lead program for CNS indications at ProQR.
Sources: Labiotech.eu, FierceBiotech.com | September 12, 2017