ProQR Announces R&D Day and Provides Update on Innovation Portfolio

First Investor R&D Day on March 14, 2016 in New York, NY

LEIDEN, Netherlands, Nov. 30, 2015 (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 orphan diseases such as cystic fibrosis (CF) and Leber's congenital amaurosis (LCA), today presents an update on the innovation pipeline and announces an R&D day for investors. ProQR has been investing in its Innovation Platform to discover RNA medicines for severe genetic disorders. Under the leadership of Chief Innovation Officer, Gerard Platenburg, this team has made significant progress in multiple therapeutic areas, which will be presented at the R&D day in more detail.

“We founded ProQR with the goal of developing a drug for CF based on a unique RNA technology that was invented at Massachusetts General Hospital. In the 3 years since incorporation, this early invention is developed into a product that is now being investigated in two global clinical studies in CF patients. During this time we have launched our second development program for LCA and have significantly expanded our understanding of the potential of RNA therapeutics. We are just starting to scratch the surface of what RNA technologies can mean to the future of medicine,” said Daniel de Boer, CEO of ProQR. “Our innovation platform has been very efficient and has yielded a wealth of new opportunities, establishing ProQR at the forefront of this novel therapeutic approach. We are laying out a strategic path forward for these core and non-core programs to maximize the potential.”

The R&D day will be led by the Company’s Chief Innovation Officer Gerard Platenburg, co-founder of ProQR. Under his leadership ProQR has initiated a broad effort to apply existing and novel RNA technologies to other severe genetic diseases to discover new potential therapies.

“We have seen the field of RNA therapies mature significantly over the last decade. To unlock the promise of this field we initiated our Innovation Platform in mid-2014,” said Gerard Platenburg, Chief Innovation Officer at ProQR. “With the broad experience in the field at our disposal, I believe we are at the verge of a new range of possibilities to treat genetic diseases. At ProQR, our innovation platform is focused on delivering the potential of this new class of therapeutics in several disease areas, including respiratory, CNS, ophthalmology and skin disorders. Within these areas, we target diseases with a good understanding of the causality, a feasible delivery route and a high unmet medical need.  At our inaugural R&D day, we will present the progress made on a selection of the innovation programs we are pursuing, including programs targeting epidermolysis bullosa, Usher syndrome, Friedreich’s ataxia, Fuchs endothelial corneal dystrophy (FECD), Huntington’s disease and Alzheimer’s disease.”

R&D Day for investors

On March 14, 2016 ProQR will organize its first R&D day in New York, NY. The day will feature a presentation from key executives in ProQR and Key Opinion Leaders in some of the therapeutic areas. We request people that are planning to attend to register at


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 the therapeutic potential of our RNA technology, our innovation programs and our R&D day. 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.

Sariette Witte
Investor Relations
T: +1 213 261 8891

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