ProQR Strengthens its Scientific Advisory Board with the Appointment of Dr. Phil Zamore, Dr. Cy Stein and Dr. Scott Armstrong


Key Updates

  • ProQR appoints three new members to the Company’s Scientific Advisory Board: Dr. Phil Zamore, Dr. Cy Stein and Dr. Scott Armstrong, who bring a tremendous amount of experience in the fields of RNA-based therapeutics and genetic pediatric diseases.
  • The new members will join Dr. Art Levin and Dr. Annemieke Aartsma-Rus on the Scientific Advisory Board that will play a strategic role in the development of the Company’s pipeline.
  • The Scientific Advisory Board will also play a key role in the advancement of the Company’s novel and proprietary next generation RNA editing technology, Axiomer®, that utilizes endogenous RNA editing processes to target a number of genetic disorders. 

LEIDEN, the Netherlands, Aug. 28, 2017 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR) today announced the appointment of three new members to the Company’s Scientific Advisory Board (SAB). The SAB members will serve as strategic advisors to the Company as it continues to develop its pipeline of RNA therapeutics for severe genetic rare diseases and novel and proprietary technology platforms. 

“We are very pleased to have such a talented and experienced SAB. Science is at the core of everything we do at ProQR, and with this group as our key advisors we can be more successful in translating innovative science into therapies for patients in need,” said Daniel de Boer, Chief Executive Officer of ProQR. “With the addition of Phil, Cy and Scott the SAB now has a tremendous level of scientific, medical and industry expertise and a significant track record of success that will be invaluable as we advance our R&D programs and our broader RNA technologies, including the Axiomer® RNA editing platform that was recently introduced at the Company’s R&D Day.”

The SAB now consists of five members, Dr. Art Levin, Dr. Phil Zamore, Dr. Cy Stein, Dr. Scott Armstrong, and Dr. Annemieke Aartsma-Rus, and is chaired by Gerard Platenburg, Chief Innovation Officer of ProQR.

Art Levin PhD, has more than two decades of experience in RNA drug development from discovery through drug registration, both in large pharma and biotech companies. He has been key to the development of numerous of oligonucleotides, including the first approved antisense medicines, and the first microRNA-targeted therapeutic in clinical trials. He is currently the Executive Vice President, Research and Development at Avidity Biosciences, Dr. Levin has published over 60 scientific articles and served as a director of the Oligonucleotide Therapeutics Society.

“ProQR has been working on cutting-edge RNA technologies since its inception,” said Art Levin. “They continue to innovate and find important new mechanisms that can be exploited for therapeutic benefit. One of the most interesting is an RNA process used in nature called A to I editing or ADAR. ProQR's Axiomer® platform takes advantage of this natural process to modulate expression of disease-related genes in a completely novel way.”

Phil Zamore PhD is a Howard Hughes Medical Institute Investigator, Gretchen Stone Cook Chair of Biomedical Sciences, Professor of Biochemistry and Molecular Pharmacology, and Chair of the RNA Therapeutics Institute at the University of Massachusetts Medical School. He is also a co-founder of Alnylam Pharmaceuticals and Voyager Therapeutics. Dr. Zamore and his coworkers have played a role in nearly all of the major breakthroughs in the study of RNA silencing. 

Cy Stein PhD, MD, is a Professor of Medical Oncology and Molecular and Cellular Biology and the former chair of the Department of Medical Oncology & Therapeutics Research at City of Hope, overseeing the department’s clinical and research programs. He was co-editor-in-chief of Nucleic Acid Therapeutics for over 20 years and the series editor for Perspectives in Antisense Science. Dr. Stein has been named a top physician in his field by New York Times Magazine and New York Magazine, he has been awarded 11 patents, and he has published widely in prominent journals, including Science, Nature Biotechnology and Annals of Internal Medicine.

Scott Armstrong MD, PhD, is an internationally renowned pediatric clinician and scientist. He is the Chair of the Department of Pediatric Oncology at Dana-Farber Cancer Institute, and the David G. Nathan Professor of Pediatrics at Dana-Farber Cancer Institute, Boston Children’s Hospital and Harvard Medical School. He is also Associate Chief of the Division of Hematology and Oncology at Boston Children’s Hospital. Dr. Armstrong previously held a variety of roles at Memorial Sloan Kettering Cancer Center in New York, NY, including in the Cancer Biology and Genetics Program and the Department of Pediatrics. His research has been recognized with many awards and he is a fellow of the American Association for the Advancement of Science (AAAS) and a member of the Association of American Physicians (AAP).

Annemieke Aartsma-Rus PhD, professor of Translational Genetics at the Leiden University Medical Center, has played an important role in the development of the antisense mediated exon skipping therapy for Duchenne muscular dystrophy (DMD). In 2015 and 2016 she was selected as the most influential scientist in DMD in the past 10 years by Expertscape. Dr. Aartsma-Rus has coordinated stakeholder meetings involving academics, industry, regulators and patient representatives, including two hosted by the European Medicine Agencies in April 2015 and November 2016. Amongst others, she is chair of the COST Action “Networking towards clinical application of antisense-mediated exon skipping for rare diseases”, past chair of the executive board of the TREAT-NMD alliance, and President-Elect of the Oligonucleotide Therapeutics Society.

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. 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. These forward-looking statements include, but are not limited to, statements regarding our SAB members, and statements regarding our ongoing and planned discovery and development of our pipeline and the advancement of our novel and proprietary technologies. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and 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.

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