LEIDEN, Netherlands, April 25, 2016 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (NASDAQ:PRQR) today announced that the company will present pre-clinical data for QR-110 which is being developed for Leber’s congenital amaurosis Type 10 (LCA10) at the 2016 Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) in Seattle, WA, May 1 – 5, 2016. QR-110 is the second molecule that will move into clinical development using ProQR’s RNA approach.
The p.Cys998X mutation which is a cause of LCA10 is a point mutation in the CEP290 gene resulting in an activation of a cryptic splice site. QR-110 is a single-stranded, chemically modified RNA oligonucleotide designed to skip the cryptic splice site and thus result in mRNA that codes for a wild-type CEP290 protein. Following intravitreal injection in vivo, QR-110 is demonstrated to reach the outer nuclear layer of the retina, the target tissue. QR-110 is also demonstrated to increase wild-type mRNA in cells with the p.Cys998X mutation. The preclinical data will be presented on poster #1123 - B0295 on May 1st, 3:15 – 5:00pm PT. The abstract is published on the ARVO 2016 annual meeting website.
Investor and analyst event at ARVO
On May 3rd at 7:15pm PT, ProQR management invites investors and analysts to a dinner with Michael Cheetham, PhD, Professor of Molecular and Cell Biology at the Institute of Ophthalmology, University College London to discuss a novel 3-D stratified retinal organoid model for the development of therapeutics for diseases of the retina. For more information or to RSVP, please contact Ronen Abergel at email@example.com.
QR-110 is a first-in-class oligonucleotide, designed to address the underlying cause of Leber’s congenital amaurosis Type 10 due to the p.Cys998X mutation in the CEP290 gene. The p.Cys998X mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional Cep290 protein. QR-110 is designed to restore wild-type CEP290 mRNA leading to the production of wild-type CEP290 protein by binding to the mutated location in the pre-mRNA causing normal splicing of the pre-mRNA. QR-110 is intended to be administered through intravitreal injections in the eye.
ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe orphan diseases such as cystic fibrosis and Leber’s congenital amaurosis. 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 about QR-110 and its clinical development and therapeutic potential, and the ARVO annual meeting. 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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