ProQR Spins Out Amylon Therapeutics as Rare CNS Disease Company

September 12, 2017

Rare Daily Staff

The Netherlands-based ProQR Therapeutics said it has spun out Amylon Therapeutics as a privately-held company focused on the development of therapies for rare central nervous system disorders.

Amylon is backed with initial funding from a group of institutional and private investors. As part of the transaction, ProQR granted an exclusive license to Amylon to develop therapeutics for beta amyloid related disorders. The aggregation of the protein beta amyloid in the brain has been associated with neurodegenerative conditions. ProQR holds a majority ownership in the company and will get future milestones and royalties from the products developed by Amylon.

Thomas de Vlaam, who joined ProQR in 2015 to incubate CNS activities with the goal of spinning out a new company, will serve as Amylon’s founding CEO. The new company will initially focus its efforts on developing an RNA-based treatment for Hereditary Cerebral Hemorrhage with Amyloidosis of the Dutch type (HCHWA-D) a rare genetic disease that leads to strokes at mid-adulthood.

HCHWA-D is a genetic subtype of cerebral amyloid angiopathy, a condition in which a point mutation leads to the accelerated onset of disease. The condition is characterized by a progressive loss of intellectual function, stroke, and other neurological problems starting in mid-adulthood. Most affected individuals die within a decade after signs and symptoms first appear, and after onset the quality of life diminishes quickly and severely. There is currently no intervention available nor in development to battle the cause and/or symptoms of HCHWA-D.

ProQR has working to discover and develop RNA therapies for severe genetic rare diseases since its creation in 2012. Its discovery development programs are harbored in separate business units with dedicated teams of specialists focused on cystic fibrosis, eye diseases, skin diseases and CNS diseases.

“As part of our corporate strategy to bring as many of our programs to patients as possible, we built ProQR as a group of focused business units that enable us to attract external funding directly into the individual development programs or ultimately spin programs out as we have now done with Amylon,” said Daniel de Boer, CEO of ProQR. “Through spinning out Amylon, we position the beta amyloid program for optimal success with a dedicated team and the funding to develop treatments for patients suffering from these devastating brain diseases.”

September 12, 2017

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