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ProQR Supports Rare Disease Day 2018 and Honors Henri A. Termeer

27 February 2018 at 7:00 AM EST

LEIDEN, the Netherlands, Feb. 27, 2018 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases including cystic fibrosis, Leber's congenital amaurosis 10 and dystrophic epidermolysis bullosa, today announced its support of Rare Disease Day 2018, to be held on February 28, 2018. On this day, patients and their families, patient organizations and healthcare providers come together to raise awareness of rare diseases.  ProQR is also co-organizing a commemorative event celebrating the legacy of the late Henri A. Termeer, co-founder of ProQR, being held on the same day in Boston, MA.

Henri Termeer, former CEO of Genzyme and a visionary and pioneer for rare diseases, was ProQR’s co-founder in 2012 and vice chairman of the supervisory board from January 2014 until he unexpectedly passed away on May 12th, 2017.  On Rare Disease Day, which is also Henri’s birthday, family, friends and associates will come together to celebrate the renaming of the “North” plaza to “Henri A. Termeer Square”, across from the Genzyme Center in Cambridge, Massachusetts, which was built by him.  In 2019, the Square will become home to a life-size sculpture of Henri Termeer.  To learn more, please visit   

In recognition of Rare Disease Day, ProQR will hold a luncheon at the Company’s headquarters in Leiden, the Netherlands, to host a special guest family in which all three young children are affected by Leber’s congenital amaurosis 10 (LCA 10).  The family will share their experiences of living with LCA and how this rare genetic disease has had an impact on their lives.  LCA 10 is a genetic eye disorder and leading genetic cause of childhood blindness.  ProQR is currently conducting a Phase 1/2 safety and efficacy trial in children and adults that suffer from LCA 10 due to one or two copies of the p.Cys998X mutation in the CEP290 gene.

“We fully support Rare Disease Day 2018, which will raise public awareness about rare diseases and bring attention to the importance of research to develop therapies for these patients who have no available treatment options,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “With an estimated 7,000 rare diseases known today and with less than 400 available therapies available, there is a dire need to quickly develop and bring to patients the medicines they need.  On this day, it is a privilege to pay tribute to Henri, a good friend and mentor and pioneer in rare diseases.  We plan to honor his legacy through completing the mission that we set out on together.”

About Rare Disease Day

Rare Disease Day is a global campaign to raise awareness of rare diseases.  Since its inception in 2008, led by EURORDIS-Rare Diseases Europe and its Council of National Alliances, thousands of events have taken place worldwide on the last day of February to bring attention to rare diseases and how they impact the lives of patients and loved ones.  The theme for 2018 is “Research, continuing on from Rare Disease Day 2017.” To learn more, please visit the Rare Disease Day website

About the late Henri A. Termer

Henri was ProQR’s co-founder, vice chairman and served on the Company’s supervisory board since January 2014. From October 1983 to June 2011, Henri served as chairman, president and chief executive officer of Genzyme Corporation. For ten years prior to joining Genzyme, Henri worked for Baxter International Laboratories, Inc., a manufacturer of human health care products. Henri resigned from Genzyme in June 2011 following the acquisition of Genzyme by Sanofi. Widely acknowledged for his contributions to the biotechnology industry and health care field, Henri was active in the areas of humanitarian assistance, policy issues, and innovation in providing access to health care. He was a member of the board of Massachusetts General Hospital and Partners HealthCare, and a member of the board of fellows of Harvard Medical School. Henri was also a member of the board of the Massachusetts Institute of Technology and serves on its Executive Committee and a board member of the Biotechnology Industry Organization (BIO). He was chairman emeritus of the New England Healthcare Institute, a nonprofit, applied research health policy organization he was instrumental in founding. Henri was a board member of ProQR Therapeutics N.V., Abiomed Inc., Aveo Pharmaceuticals, Verastem, Inc., Moderna Therapeutics and Medical Simulation, and was a board member of Allergan, Inc. from 2014 through its acquisition by Actavis in March 2015. In 2008, he was appointed to Massachusetts Governor Deval Patrick’s Council of Economic Advisors. Henri was chairman of the Federal Reserve Bank of Boston’s board of directors from 2010-2011. Henri studied economics at the Economische Hogeschool (Erasmus University, the Netherlands) and earned an MBA from the Darden School at the University of Virginia.

About Leber’s Congenital Amaurosis 10

Leber’s congenital amaurosis (LCA) is the most common cause of blindness due to genetic disease in children and consists of a group of diseases of which LCA 10 is the more severe forms. LCA 10 is caused by mutations in the CEP290 gene of which the p.Cys998X mutation is the most common. LCA 10 leads to early loss of vision causing most people to lose their sight in the first few years of life. To date, there are no treatments approved or other products in clinical development that treat the underlying cause of the disease. Approximately 2,000 people in the Western world have LCA 10 because of this mutation.

About ProQR

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such as cystic fibrosis, Leber’s congenital amaurosis 10 and dystrophic epidermolysis bullosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
*Since 2012*


This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such statements include those relating to Rare Disease Day 2018, and our clinical development and therapeutic potential of our product candidates.  Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.

ProQR Therapeutics N.V.:
Investor Contact:
Smital Shah
Chief Financial Officer
T: +1 415 231 6431

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Source: ProQR Therapeutics N.V.