ProQR Strengthens Leading Intellectual Property Estate for ADAR-Mediated RNA Editing

ProQR Therapeutics N.V. recently announced it has further strengthened its leading intellectual property (IP) estate with the issuance of a new patent in the US and that it has successfully defended against an opposition to its IP in Japan.

“The new patent that the USPTO granted to ProQR further expands the protection of RNA editing using oligonucleotides to recruit endogenous ADAR. Our leading intellectual property portfolio protects our Axiomer ADAR-mediated RNA editing platform technology and more fundamentally the use of an oligonucleotide to recruit endogenous deaminating enzymes in the cell,” said René Beukema, Chief Corporate Development Officer and General Counsel. “Our patents have been challenged by multiple parties on several occasions and have been upheld by the European Patent Office and now also by the Japan Patent Office. We believe safeguarding our intellectual assets is pivotal to our long-term success and intend to continue to defend against challenges through our leading IP position.”

The United States Patent and Trademark Office (USPTO) granted a new patent to ProQR, under US patent No. 11,781,134 that relates to a method for RNA editing. Key claims in this patent pertain to use of any type of oligonucleotide that is at least sufficiently complementary to a target sequence that can recruit an endogenous Adenosine Deaminase Acting on RNA (ADAR), which then can deaminate (or edit) a target adenosine into an inosine in the target sequence. The scope of the patent further underpins that the broad concept of applying endogenous ADAR by administering antisense oligonucleotides for RNA editing is proprietary to ProQR.

Separately, the Japanese Patent Office issued a final communication indicating that the main claim in one of ProQR’s leading IP estates related to single-stranded antisense RNA editing oligonucleotides comprising one or more mismatches with the target sequence was upheld regardless of a third-party opposition against the granted patent (JP 7074345).

ProQR invented the use of endogenous ADAR in RNA editing in 2014 and filed a first patent application in that same year. Since then, ProQR has filed multiple additional patent applications on further improvements to form a leading patent estate that makes RNA editing with oligonucleotides that recruit endogenous ADAR proprietary to ProQR. Today ProQR has extensive patent protection related to its RNA editing platform, Axiomer, including more than 11 published patent families, that currently comprise a total of 29 patents. Beyond this, ProQR has several unpublished patent applications and continuously invests in expanding its IP estate around ADAR-mediated RNA editing.

ProQR is pioneering a next-generation RNA base editing technology called Axiomer, which could potentially yield a new class of medicines for diverse types of 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 called ADAR (Adenosine Deaminase Acting on RNA). Axiomer EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G) – correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease.

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind. For more information, visit www.proqr.com.