ProQR and Galapagos announce research collaboration on Fibrosis targets using ProQR’s Axiomer® technology

Monday, January 8, 2018

ProQR Therapeutics N.V. announced that it has entered into a research collaboration agreement with Galapagos N.V. Under the agreement the two companies will work together to discover novel Axiomer Editing Oligonucleotides (EONs) against fibrosis targets selected by Galapagos. The targets that will be pursued in the collaboration and financial details about the collaboration are not disclosed.

ProQR is developing its RNA platform technology in areas of ophthalmology, cystic fibrosis and dystrophic epidermolysis bullosa. Through a collaboration with Amylon Therapeutics, the platform is also being developed for CNS indications including HCHWA-D. Recent progress in the validation and expansion of the platform included positive results in clinical trials in cystic fibrosis and the start of a clinical trial in Leber’s congenital amaurosis.

“In this collaboration we will explore a novel application of our Axiomer® technology to Galapagos’ fibrosis targets,” said Daniel A. de Boer, CEO of ProQR. “We look forward to working closely with the team at Galapagos. With their leadership in the discovery of novel drug targets and our platform technology and know-how, we can make a meaningful impact together.”

Onno van de Stolpe, CEO at Galapagos added: “With our vast experience in the discovery of novel drug targets, we believe ProQR’s Axiomer technology can be applied to strengthen the validation of novel targets from our platform. We look forward to collaborating with ProQR and its exciting RNA editing technologies.”

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*

About Galapagos

Galapagos (Euronext & NASDAQ:GLPG) is a clinical-stage biotechnology company specialized in the discovery and development of small molecule medicines with novel modes of action. Galapagos' pipeline comprises Phase 3 through to discovery programs in cystic fibrosis, inflammation, fibrosis, osteoarthritis and other indications. Galapagos has demonstrated proof of platform with filgotinib targeting JAK1 in inflammatory conditions (collaboration with Gilead), GLPG1690 targeting autotaxin in IPF, and MOR106 targeting IL-17C in atopic dermatitis (collaboration with MorphoSys). Galapagos is focused on the development and commercialization of novel medicines that will improve people's lives. The Galapagos group, including fee-for-service subsidiary Fidelta, has approximately 550 employees, operating from its Mechelen, Belgium headquarters and facilities in The Netherlands, France, and Croatia. More information at www.glpg.com.

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® “Editing Oligo Nucleotides”, or EONs, recruit an endogenously expressed RNA editing system called ADAR, which it can direct to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G).

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