ProQR Announces Conference Presentations for Axiomer® RNA Editing Technology and QR-313 for DEB
LEIDEN, the Netherlands, April 30, 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, today announced that the Company will present at two upcoming conferences in May.
Presentation at the TIDES 2018 Conference
ProQR will deliver a presentation on the Axiomer® RNA editing technology during the TIDES: Oligonucleotide and Peptide Therapeutics Conference to be held on May 7 – 10, 2018 in Boston, MA, USA.
|Abstract title:||Axiomer® technology: Therapeutic oligonucleotides for directing site-specific A-to-I editing by endogenous ADAR enzymes|
|Presenter:||Antti Aalto, Ph.D., senior scientist protein interactions of ProQR|
|Oral Presentation:||Thursday, May 10, 11:45am – 12:15pm ET|
|Session:||Track 5: mRNA, CRISPR and Hot Topics in Oligonucleotides|
Presentation at the IID 2018 Meeting
ProQR will present data from the QR-313 program for dystrophic epidermolysis bullosa at the International Investigative Dermatology (IID) 2018 meeting to be held on May 16 – 19, 2018 in Orlando, FL, USA.
|Abstract title:||QR-313, an antisense oligonucleotide, restores expression of functional type VII collagen in DEB patient cells|
|Presenter:||Ilse Haisma, PH.D., senior scientist dermatology of ProQR|
|Poster Presentation:||Saturday, May 19, 11:45 am – 1:45 pm ET|
|Poster #:||984 – 1474 EVEN/ 493 – 981 ODD|
|Oral Presentation:||Saturday, May 19, 3:15 - 5:45pm ET|
About 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® “Editing Oligonucleotides”, or 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® EONs are designed to recruit an endogenously expressed RNA editing system called ADAR, which can direct the change of an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G).
QR-313 is a potential first-in-class RNA-based oligonucleotide designed to address the underlying cause of dystrophic epidermolysis bullosa (DEB) due to mutations in exon 73 of the COL7A1 gene. Mutations in this exon can cause loss of functional collagen type VII (C7) protein. Absence of C7 results in the loss of anchoring fibrils that normally link the dermal and epidermal layers of the skin together. QR-313 is designed to exclude exon 73 from the mRNA (exon skipping) and produce a functional C7 protein, thereby restoring functionality of the anchoring fibrils. QR-313 has been granted Orphan Drug Designation in the United States and the European Union.
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.
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 our participation at the TIDES: Oligonucleotide and Peptide Therapeutics Conference and the International Investigative Dermatology meeting. 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.:
Chief Financial Officer
T: +1 415 231 6431