ProQR Appoints ADAR Expert Dr. Peter A. Beal to Scientific Advisory Board to Focus on Axiomer® RNA A-to-I Editing Technology

Key Updates

  • Dr. Peter A. Beal joins ProQR’s scientific advisory board, bringing with him tremendous experience in the field of RNA-based therapeutics, particularly in the area of RNA editing.
  • As an expert in the field of ADAR and A-to-I editing, Dr. Beal will be instrumental in advancing the company’s novel and proprietary Axiomer® RNA editing technology.
  • The board now consists of seven members: Dr. Peter Beal, Dr. Art Levin, Dr. Thaddeus (Ted) Dryja, 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.

LEIDEN, the Netherlands, April 10, 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 Leber's congenital amaurosis 10, dystrophic epidermolysis bullosa and cystic fibrosis, today announced the appointment of Dr. Peter A. Beal to the company’s scientific advisory board (SAB).

“The field of RNA editing with ADAR is exciting and holds the potential to generate a whole new class of therapeutics,” said Dr. Peter Beal, professor at the University of California, Davis and SAB member of ProQR. “Through the collaboration with ProQR, we’ve been able to accelerate the understanding of the basic editing mechanism of A-to-I ADAR. In my new role as an SAB member, I look forward to helping ProQR to transform the unique Axiomer® technology using ADAR into therapies for patients.”

Dr. Beal is an influential leader in the field of RNA editing and Professor in the department of chemistry at UC Davis. His research focuses on the use of chemical approaches to the study of RNA biology with a particular emphasis on RNA editing and RNA interference. Dr. Beal received a Bachelor of Science degree from University of North Dakota, he received his Ph.D. at the California Institute of Technology and was an NIH postdoctoral fellow at Harvard University in the laboratory of Professor Stuart L. Schreiber. After, he became a Full Professor at the University of Utah.

ProQR and Dr. Beal have been collaborating extensively on the optimization of ProQR’s proprietary Axiomer® A-to-I editing technology. By the administration of just a single stranded editing oligonucleotide (or EON), the Axiomer® technology enables the recruitment of endogenous ADAR (adenosine deaminases acting on RNA) to edit individual nucleotides in the RNA, without the need for bacterial enzymes or viral vectors. The technology platform enables a patient’s body to repair its own RNA specifically and reversibly and could be applicable to over 20,000 disease-causing mutations.

“Given Dr. Beal’s exceptional background in RNA research, we are very pleased to welcome Peter to our scientific advisory board,” said Gerard Platenburg, Chief Innovation Officer at ProQR. “Particularly exciting for us is Peter’s pioneering work in A-to-I editing using the body’s own ADARs as RNA editing enzymes that will help us in harnessing the broad applicability of our Axiomer® platform.”

The other members of the SAB are:

Art Levin Ph.D., 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 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 and Dr. Levin has published over 60 scientific articles and served as a director of the Oligonucleotide Therapeutics Society.

Thaddeus (Ted) Dryja, M.D., is a Clinical Professor of Ophthalmology at Harvard Medical School and a faculty member at the Massachusetts Eye and Ear Infirmary. Previously, Dr. Dryja served as the Global Head of Translational Medicine in Ophthalmology and as the Vice President and Global Head of Ophthalmology research at the Novartis Institutes for Biomedical Research (NIBR). In 1983, he joined the Massachusetts Eye and Ear Infirmary and the faculty of the Department of Ophthalmology at Harvard Medical School and later became director of the David G. Cogan Pathology Laboratory at the Massachusetts Eye and Ear Infirmary and the Cogan Professor of Ophthalmology at Harvard. Dr. Dryja is a magna cum laude graduate of Yale College, earned his medical degree from the Yale University School of Medicine, and completed a residency in ophthalmology and fellowships in ophthalmic pathology and molecular genetics at Harvard Medical School.  In 1996, he became a member of the U.S. National Academy of Sciences.

Phil Zamore Ph.D., 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 Ph.D., M.D., 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 M.D., Ph.D., 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 Ph.D., 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 by Expertscape as the most influential scientist in DMD in the past 10 years. 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 Leber’s congenital amaurosis 10, dystrophic epidermolysis bullosa and cystic fibrosis. 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 product candidates, including our development and their therapeutic potential. 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.

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

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