Full Press Release Details
ProQR Therapeutics N.V.
Press Release September 21, 2017
ProQR Presents in vivo Proof of Concept Data for the
Axiomer RNA Editing Platform Technology
LEIDEN, the Netherlands, September 21, 2017 ProQR Therapeutics N.V. (Nasdaq: PRQR), today announced that a presentation titled Axiomer Technology: Therapeutic Oligonucleotides for Directing Site-Specific A-to-I Editing by Endogenous ADAR
Enzymes will be presented during the Oligonucleotide Therapeutics Society (OTS) Meeting to be held September 24-27, 2017 in Bordeaux, France. The presentation is part of session: Emerging Topics in RNA
Biology on September 25, 2017 starting at 3:00pm CET.
These data were also presented during a Drug Information Association (DIA) webinar
Oligonucleotide-Based Therapeutics in Rare Diseases that took place on September 19, 2017. A replay of the webcast is now available online (1). The presentation is titled
Axiomer Technology: Therapeutic Oligonucleotides for Directing Site-Specific A-to-I Editing by Endogenous
The study demonstrated that, in an in vivo research model of Hurler syndrome, treatment with the Axiomer EONs resulted in editing of RNA and partial restoration of the enzymatic activity that is missing in this syndrome. Moreover, the increase in enzymatic activity correlated well with reduced
levels of the enzyme s substrate, the accumulation of which results in the characteristics of the syndrome.
RNA editing is an exciting
therapeutic strategy that is rapidly evolving, said Phillip D. Zamore, PhD, 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. ProQR s RNA editing technology promises unique advantages over alternative approaches. This data represents significant progress in confirming the proposed
scientific rationale for this novel technology.
Gerard Platenburg, Chief Innovation Officer at ProQR added, We are the first to show
evidence of RNA editing using endogenous ADAR enzymes in an in vivo model. We see the results from this proof of concept study as a major milestone in the development of our
Axiomer technology for clinical application. With this data, we can continue to build the evidence to take the first EONs further into development.
ProQR Therapeutics N.V. | Zernikedreef 9, 2333 CK Leiden, The Netherlands | +31 88 166 7000 | info@proqr.com | www.proqr.com
Next-Generation Axiomer Platform Technology
ProQR is pioneering a next-generation RNA technology called Axiomer , which could potentially yield a
new class of medicines for genetic diseases. Axiomer EONS mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human
cells. The Axiomer Editing Oligo Nucleotides , or EONs, recruit an endogenously expressed RNA editing system called ADAR, which it can direct to change an Adenosine (A) to an
Inosine (I) in the RNA an Inosine is translated as a Guanosine (G).
About Hurler Syndrome
Mucopolysaccharidosis type I-Hurler (MPS I-H; Hurler syndrome) is a recessive
lysosomal storage disorder caused by mutations in the IDUA gene, which codes for the -L-iduronidase enzyme. The most common of these mutations is the G-to-A mutation resulting in the formation of a premature termination codon (W402X) and absence of functional
-L-iduronidase enzyme. The lack of the enzyme results in accumulation of its lysosomal substrates, glycosaminoglycans, which leads to severe defects in multiple
organs and a very limited life expectancy.
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.
FORWARD-LOOKING STATEMENTS
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 our 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 the therapeutic potential and clinical development of our Axiomer platform technology. 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.
T: +31 6 2970 4513 (NL)
T: + 1 213 261 8891 (US)
ProQR Therapeutics N.V. |
Zernikedreef 9, 2333 CK Leiden, The Netherlands | +31 88 166 7000 | info@proqr.com | www.proqr.com