Rare Diseases

ProQR spins out CNS assets to form RNA rare disease biotech

Amylon Therapeutics CEO Thomas de Vlaam

ProQR has spun out its CNS assets to form Amylon Therapeutics. The offshoot starts life with seed funding and an RNA-based program targeting a rare genetic disease that causes strokes.

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). Further down the line, Amylon plans to target other beta amyloid related disorders, such as cerebral amyloid angiopathy (CAA).

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…