Pharma Focus Asia

ProQR Successfully Protects Axiomer™ IP Portfolio from New Challenge

Saturday, April 20, 2024

ProQR Therapeutics N.V. (Nasdaq: PRQR) has once again successfully defended a crucial patent for its groundbreaking RNA editing platform, Axiomer™. This achievement follows the company's victory in fending off opposition against its key patent, EP 3 507 366 B1, filed with the European Patent Office (EPO) in June 2021.

The patent focuses on editing oligonucleotides with specific chemical modifications, enhancing stability, and enlisting endogenous ADAR for editing purposes. It plays a vital role in ProQR's intellectual property portfolio, safeguarding its Axiomer RNA editing platform and its current generation of molecules. Following a public hearing on April 17, 2024, the EPO's Opposition Division upheld ProQR's robust IP position on chemically modified editing oligonucleotides.

The patent encompasses broad platform claims, protecting ProQR's proprietary ADAR-mediated RNA editing platform Axiomer. René Beukema, Chief Corporate Development Officer and General Counsel at ProQR, expressed confidence in the decision, emphasizing its reinforcement of the company's IP estate and the value it brings to advancing their science, platform, and pipeline.

ProQR initiated the utilization of endogenous ADAR in RNA editing in 2014, marking the beginning of a series of patent filings. Over the years, the company has expanded its patent estate, reinforcing its leading position in ADAR-mediated RNA editing. Presently, ProQR boasts extensive patent protection related to the Axiomer RNA editing platform, comprising 15 published patent families and a total of 28 patents. Additionally, the company continues to invest in expanding its IP estate concerning ADAR-mediated RNA editing.

Axiomer™ represents a revolutionary RNA base editing technology pioneered by ProQR, with the potential to revolutionize medicine across diverse disease types. Axiomer™ "Editing Oligonucleotides" (EONs) facilitate precise single nucleotide changes to RNA utilizing ADAR present in human cells. By directing endogenous ADARs to convert Adenosine (A) to Inosine (I) in RNA, Axiomer™ EONs hold promise in correcting disease-causing mutations, modulating protein expression, and introducing new protein functions to combat or prevent diseases.

This victory underscores ProQR's dedication to innovation and its commitment to advancing transformative RNA therapies through cutting-edge technologies like Axiomer™.



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