ProQR Announces Presentations at ECFS on Eluforsen for F508del Cystic Fibrosis and at CFF Research Conference
LEIDEN, The Netherlands, June 04, 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 upcoming presentations at the European Cystic Fibrosis Society (ECFS) conference and the CFF Research Conference.
Presentation at ECFS Conference
ProQR will deliver a short talk (ePoster) and a poster presentation on eluforsen during the ECFS: European Cystic Fibrosis Society Conference to be held on June 6 – 9, 2018 in Belgrade, Serbia.
| Abstract title: | Exploratory immune assays distinguish healthy volunteer from CF patient cohorts and were validated in a dose escalation study of QR-010 in subjects with cystic fibrosis homozygous for the F508del CFTR mutation | |
| Presenter: | Miriam Bujny, Ph.D., director biomarkers of ProQR | |
| Poster Presentation: | Friday, June 9 14:00 – 15:00 CET | |
| Poster #: | EPS3.02 | |
| ePoster Presentation: | Session: New treatments: from genes to protein. Thursday, June 7, 14:00-15:00 CET |
Presentation at 2018 CFF Research Conference – New Technologies Advancing Toward a One-Time Cure
ProQR’s scientific advisory board (SAB) member, Prof. Yi-Tao Yu from the University of Rochester Medical Center (URMC), will give a presentation at the upcoming CFF Research Conference – New technologies Advancing Towards a One-Time Cure. The conference will be held on June 18 -21, 2018 at the Snow King Resort in Jackson, WY USA.
| Presentation title: | Suppression of nonsense mutations in the CFTR gene by targeted RNA pseudouridylation | |
| Presenter: | Yi-Tao Yu, Prof. University of Rochester Medical Center | |
| Session: | Session 2: Approaches for Targeting Nonsense Mutations | |
| Schedule: | Tuesday, June 19 10:55 – 11:20 MT |
About eluforsen
Eluforsen, formerly known as QR-010, is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of the disease by targeting the mRNA in CF patients that have the F508del mutation. The technology was exclusively licensed from Massachusetts General Hospital. The F508del mutation results in the production of a misfolded CFTR protein that does not function normally. Eluforsen is a single agent designed to bind to the defective CFTR mRNA and to restore CFTR function. Eluforsen is designed to be self-administered via an optimized eFlow® Nebulizer (PARI Pharma GmbH). eFlow® is a small, handheld aerosol delivery device which nebulizes eluforsen into a mist inhaled directly into the lungs. Eluforsen has been granted orphan drug designation in the United States and the European Union and fast-track status by the FDA. The eluforsen project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 633545.
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*
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. Such statements include those relating to our participation at the European Cystic Fibrosis Society Conference and the CFF Research Conference. 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.:
Smital Shah
Chief Financial Officer
T: +1 415 231 6431
ir@proqr.com
