ANNUAL REPORT 2015 It’s in our RNA ProQR Therapeutics N.V. T: +31 88 166 7000 W: www.proqr.com E:
[email protected] Office Leiden: Darwinweg 24, 2333 CR Leiden, The Netherlands Office Palo Alto: 543 Bryant Street, Palo Alto, CA 94301, US Including magazine PAGE I PAGE II Magazine Magazine ANNUAL REPORT 2015 ANNUAL REPORT 2015 quartered in the bioscience park in Growth numbers Leiden, the Netherlands. Surround- ed by several other innovative 105 organizations, ProQR’s scientific staff pioneer in the development of medicines in its relentless efforts to find medicines to fight rare genetic 5 diseases. ProQRians Since its inception in 2012, ProQR 8 quickly advanced an RNA repair technology for CF that was discov- ered by a world-renowned RNA sci- 1 entist from Massachusetts General Hospital and called their molecule Programs QR-010. A second program called 100 ProQR QR-110 was launched to fight the Game-changing innovator most common cause of genetic blindness in children, a disease in the interest of patients called Leber’s congenital amaurosis. 1 This program is based on a tech- Targets under evaluation nology discovered by a university in For decades there was little hope for people with a rare genetic disease. the Netherlands. With pre-clinical 140M data for the programs in hand, the Due to the innovative efforts of several companies, people with rare genetic company completed a successful diseases like cystic fibrosis (CF) may cherish hope. Since the discovery of RNA IPO on the Nasdaq Stock Market in 520K 2014. modification, a future beckons in which DNA errors can be corrected at the Capital raised in € RNA level, overcoming some of the challenges that are associated with, for ProQR’s focus 10M ProQR has shown remarkable exe- example, gene therapies.