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
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).
PROQR 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. 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 collaboration with Galapagos and the potential of our Axiomer platform. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with clinical development activities, including that positive results observed in prior and ongoing studies may not be replicated in later trials or guarantee approval of any product candidate by regulatory authorities, regulatory review or approval process, and that we may not be able to realize the potential benefits of the collaboration or the use of our Axiomer platform, and the risks, uncertainties and other factors in our filings made with the
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Source: ProQR Therapeutics N.V.