ProQR Therapeutics recently presented an update on its drug portfolio and introduced its new proprietary RNA platform technology, Axiomer, during the company’s R&D day in New York. This new RNA editing tool has the potential to drive the production of a new class of therapies targeting genetic diseases including cystic fibrosis (CF).
“Since last year we have made good progress on executing on our strategy to develop life-changing therapies for patients in need, through a diversified pipeline with a balanced risk profile,” Daniel A. de Boer, ProQR CEO, said in a press release.
Human genetics is based on a genetic code composed of only four different nucleotides — guanine, adenine, thymine, and cytosine — that when combined in triples produce amino acids, which in turn are translated into proteins. A small change in one of the nucleotides in the genetic code can be harmless, but it also can cause serious medical conditions.
All cells have natural gene-editing molecular machinery. ProQR’s next-generation RNA technology, Axiomer, is able to alter a single nucleotide base of an RNA molecule in a specific and targeted manner. This new strategy could create the ability to correct small mistakes in our genetic code that lead to diseases.
Axiomer “Editing Oligo Nucleotides,” or EONs, was designed to recruit a naturally expressed RNA editing system called ADAR. This mechanism can directly change an adenosine in the RNA code into an inosine — a nucleoside that is read as a guanosine.
“We are pleased to show the progress we have made in our RNA therapeutics pipeline at our second R&D day. Following QR-010 for CF, QR-110 is now in clinical trials for LCA 10 [Leber’s congenital amaurosis Type 10], and our third molecule, QR-313 for DEB [dystrophic epidermolysis bullosa] will move to clinical trials early next year. We are focusing on three important genetic diseases, all with high unmet needs, and all which we believe could greatly benefit from our unique RNA oligonucleotide approach. Within the next 18 months we will have generated clinical data in patients in all these programs,” de Boer said.
Apart from these three disorders, the company is also developing therapies in the ophthalmology field.
“While our focus and priority is on clinical development of our most advanced RNA-based therapeutics to help patients with CF, LCA 10, and DEB, we continue to innovate in RNA science. This innovation effort has led to the discovery of a novel RNA editing technology that we believe can address the underlying cause of a broad range of genetic defects at the RNA level,” de Boer added.
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