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View Annual Report 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. Undoubtedly, further development of this technolo- cution power. It has taken the initial gy offers hope of a better life for millions of patients. This is the world where idea of the QR-010 program for CF targeted at the ∆F508 mutation ProQR Therapeutics is focusing. 49K into two global clinical studies in Patient potential a total of 80 CF patients to date. It Initially, ProQR was founded to beat a cure. ProQR originated from the advanced the second program, QR- 2012 2013 2014 2015 cystic fibrosis in just one child. Now, drive of Dutchman Daniel de Boer, 110, which targets Leber’s congeni- ProQR’s quest leads far beyond, as who was looking for a treatment tal amaurosis due to the p.Cys998X its mission is to develop treatments for his son. He eventually founded mutation and is moving this second for all patients with rare genetic ProQR together with preeminent program towards the clinic in 2016. diseases. leaders in the biotech space: Dinko With the start of it’s innovation unit, RNA and our genes, in short Valerio, Henri Termeer and Gerard the company has been actively The company, with offices and labs Platenburg. expanding the pipeline to utilize its Genetic diseases are caused by a defect in our genes, our DNA. in Leiden (the Netherlands) and RNA technologies to target severe These broken genes cause downstream effects on the proteins Palo Alto (CA, US), is a result of what RNA modification – the name of genetic disorders. ProQR now has which cause the diseases. To create proteins our cells make a is globally known as ‘patient-driven the game promising programs in five thera- copy of our genes, called the RNA, which functions as the blue- drug development’. It is a great Only four years after the company’s peutic areas. print for proteins. ProQR’s technologies aim to repair the defects example of people impacted by a formation, ProQR is part of the in the RNA to restore protein function and take away the underly- disease starting their own quest for thriving global biotech sector, head- ing cause of a disease. PAGE III PAGE IV Magazine Magazine ANNUAL REPORT 2015 ANNUAL REPORT 2015 RNA MODULATION an elegant approach The ProQR way tempting to fix the underlying defect at the genetic level small and do not need a vector or vehicle to be deliv- • Dystrophic epidermolysis bullosa, a severe disorder Historically, there have not been many treatment through DNA modification, which permanently changes ered to the target organ. The molecules are specifically that causes fragile skin options for severe genetic diseases. Over the past few the genetic makeup of patients and is hard to deliver to designed for the disease and its particular defect and • Usher syndrome, the most common cause of com- decades, many new approaches have been developed the right cells and organs. ProQR aims to repair genetic therefore can have potential wide applicability. ProQR bined deaf and blindness but there is still a clear unmet need for many patients. defects through RNA modification, which potentially has developed a toolbox of RNA technologies to make • Fuchs endothelial corneal dystrophy (FECD), a com- These new approaches include looking at an alternative removes the underlying cause of genetic diseases, but molecules with which the company can potentially treat mon disease causing vision loss way to add the functionality lost by the genetic defect does not permanently alter cells. Furthermore, the many genetic disorders. • Friedreich’s ataxia, results in progressive damage to by adding the missing proteins, but the applicability is repair only takes place in cells that express the gene the nervous system limited to some diseases only. Another approach is at- that is causing the disease and the RNA molecules are How does the QR-010 molecule work? • Huntington’s disease, affecting muscle coordination Unlike any other CF treatment currently in develop- and cognition ment, QR-010 aims to repair the ∆F508 mutation in the • Beta-amyloid related disorders including Alzheimer’s RNA. After the RNA is repaired, a normal healthy CFTR disease, the most common form of dementia. protein can be formed that is expected to have normal Research and development pipeline function. The goal of ProQR’s QR-010 is to stop the progression of cystic fibrosis. How does the QR-110 molecule work? QRX-704 QRX-313 As with all of ProQR’s molecules, QR-110 aims to repair The molecules are Huntington’s disease Epidermolysis bullosa the mutation at the RNA level, in this case restoring CEP290 function. To target the p.Cys99X mutation in specifically designed the CEP290 gene ProQR needed a different approach for the disease and QRX-504 QRX-411 than the one used for QR-010 for CF. Therefore, a sec- Fuchs (FECD) Usher syndrome ond technology was licensed and QR-110 was designed. its particular defect QR-110 QR-010 and therefore can QRX-604 QRX-203 LCA cystic fibrosis Other molecules in development Friedreich's ataxia Alzheimer’s disease >2,000 patients >49,000 patients ProQR has a pipeline with molecules targeting more have potential wide diseases than just CF and LCA. Other diseases that the applicability company is working on include: Innovation Pre-clin Phase 1 & Pivotal development clinical PoC studies PAGE VI Cystic fibrosis at a glance ANNUAL REPORT 2015 Indy Klaver (14) Cystic fibrosis at a glance Cystic fibrosis is a life-threatening, genetic disease ria leading to infections, extensive lung damage and that causes persistent lung infections and progres- eventually, respiratory failure. In the pancreas, the sively limits the ability to breathe. In people with CF, mucus prevents the release of digestive enzymes a defect in the CFTR gene causes the production of that allow the body to break down food and absorb faulty CFTR protein causing a thick, build-up of mu- vital nutrients. The most common CF mutation is cus in the lungs, pancreas and other organs. In the the ∆F508 mutation that affects about 70% of the Dreaming of lungs, the mucus clogs the airways and traps bacte- 70,000 CF patients worldwide. a care-free life with severe coughing and lung in- knows the whole routine is nec- not there all the time. A life without fection. “Once or twice a year, when essary to lead a fairly normal life. the medication, without the rules a bacteria of some sort makes me “But it is just too much sometimes. I that are all ‘for your own good’. I A group of teenage girls on bikes enters a street in Oostzaan, a pretty rural sick, breathing becomes difficult. hate it, get angry, take it out on my hear that too often. I don’t want to It’s as if I am breathing through mom.” be the problem child. I wish there town just north of Amsterdam. They chatter and laugh on the way home after a straw. I sometimes experience was some sort of pill that would a long day at school in nearby Zaanstad. For a neutral observer it would be breathing difficulties, for example ‘Fairly normal’ means that Lin- get rid of CF for good.” In short, a when I try to run up the stairs at da and Indy can go on day trips, healthy, hassle-free life. difficult to distinguish the girl in the group who suffers from cystic fibrosis school. I have to stop and catch my shopping in Amsterdam or even to (CF), a rare genetic disease with a high mortality rate. breath. Having an infection means an amusement park. Linda: “But Looking outside from the living I have to go to hospital. I need we will need to bring the bag with room, with dinner almost ready, Meet Indy Klaver, who just got hooked up to an oxygen tank. a week or two with intravenous medication, nebulizer and other Indy spots a ray of sunshine over home.
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