ProQR Appoints Thaddeus Dryja, M.D. to Scientific Advisory Board  

Key Updates

• Dr. Thaddeus (Ted) Dryja, a pioneer in the field of retinal genetic diseases, joins the Company’s Scientific Advisory Board.
• Dr. Dryja will play a key strategic role in the development and advancement of ProQR’s growing ophthalmology pipeline.

LEIDEN, The Netherlands, Nov. 09, 2017 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR) today announced the appointment of Thaddeus Dryja, M.D. to the Scientific Advisory Board (SAB). Dr. Dryja is a member of the faculty at Massachusetts Eye and Ear and Harvard Medical School, and previously served as the Global Head of Ophthalmology Research at the Novartis Institutes for BioMedical Research.  With the appointment of Dr. Dryja, the Company’s SAB, which is chaired by Gerard Platenburg, Chief Innovation Officer of ProQR, now consists of six members including: Drs. Art Levin, Annemieke Aartsma-Rus, Phil Zamore, Cy Stein and Scott Armstrong. 

“Ted is a pioneer in retinal genetic diseases, having made a number of key discoveries during his decades-long career.  His extensive background in academia and the clinic will be of substantial value to ProQR as we continue developing our RNA based therapeutics for a growing number of ophthalmic indications,” said Gerard Platenburg, Chief Innovation Officer at ProQR.

“ProQR’s RNA-based approach offers significant potential for treating rare and debilitating ophthalmologic indications,” said Dr. Dryja. “I look forward to working with ProQR’s management team and fellow members of the Scientific Advisory Board to investigate the clinical value of the approach in a scientifically rigorous manner.”

Thaddeus (Ted) Dryja, M.D. is a Clinical Professor of Ophthalmology at Harvard Medical School and a faculty member at the Massachusetts Eye and Ear Infirmary.  From 2006 – 2009, Dr. Dryja served as the Global Head of Translational Medicine in Ophthalmology and from 2009-2017 as the Vice President and Global Head of Ophthalmology research at the  Novartis Institutes for Biomedical Research (NIBR).  In 1983, he joined the Massachusetts Eye and Ear Infirmary and the faculty of the Department of Ophthalmology at Harvard Medical School.  From 1983-2006, he led a research team studying the molecular genetics of hereditary diseases of the retina as well as practicing general ophthalmology and ophthalmic pathology. In 1992, he became director of the David G. Cogan Pathology Laboratory at the Massachusetts Eye and Ear Infirmary and in 1993 he became the Cogan Professor of Ophthalmology at Harvard. His research discoveries included finding compelling evidence for the recessive nature of oncogenic mutations at tumor suppressor genes like the retinoblastoma gene, the identification and cloning of the retinoblastoma gene, and the identification of 16 different genes responsible for forms of retinal degeneration and retinal dysfunction.  Dr. Dryja is a magna cum laude graduate of Yale College, earned his medical degree from the Yale University School of Medicine, and completed a residency in ophthalmology and fellowships in ophthalmic pathology and molecular genetics at Harvard Medical School.  In 1996, he became a member of the U.S. National Academy of Sciences.

ProQR’s ophthalmology pipeline includes the following:

• QR-110 for Leber’s congenital amaurosis 10 (LCA 10) due to the p.Cys998X mutation, which received IND and CTA clearance and is in clinical development (PQ-110-001 Phase 1/2 safety and efficacy study) with interim data expected in 2018, and full-data in 2019.  QR-110 was also granted Fast Track designation by the FDA and orphan drug designation by the FDA and EMA.    
• QR-421 for Usher syndrome type 2 due to exon 13 mutations in the USH2A gene, for which a clinical candidate has been selected and is ready for IND enabling development studies.
• QR-411 for Usher syndrome type 2 due to the c.7595-2144A>G in the USH2A gene, for which a clinical candidate has been selected and is ready for IND enabling development studies. QR-411 also received orphan drug designation by the FDA and EMA.
• QRX-1011 for Stargardt’s disease due to c.5461-10T>C mutations in the ABCA4 gene, which is in optimization phase.
• QRX-504 for Fuchs endothelial corneal dystrophy (FECD), for which a clinical candidate has been selected and is ready for IND enabling development studies.

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 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.
*Since 2012*

FORWARD-LOOKING STATEMENTS

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ProQR Therapeutics N.V.:
Investor and Media Contact:
Bonnie Ortega
T: +1 858 245 3983
ir@proqr.com