Full Press Release Details
ProQR Achieves Successful Defense
of New Challenge to its Axiomer IP Portfolio
LEIDEN, Netherlands &
CAMBRIDGE, Mass., April 19, 2024 - ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through
transformative RNA therapies, today announced it has again successfully defended against opposition filed against a key patent for its
ADAR-mediated RNA editing platform Axiomer .
The opposition was filed in June 2021
with the European Patent Office (EPO) by a strawman against ProQR's granted patent EP 3 507 366 B1, which relates to editing
oligonucleotides that have certain chemical modifications in the base and/or ribose sugar to increase stability and recruit endogenous
ADAR to edit the target adenosine. This patent is part of ProQR's
intellectual property estate surrounding its Axiomer RNA editing platform and the current generation of its molecules. The Opposition
Division of the EPO held a public hearing on April 17, 2024, and ruled such that ProQR's strong IP position on chemically
modified editing oligonucleotides was maintained.
broad platform claims including: "An antisense oligonucleotide (AON) capable of forming a double stranded complex with a target
RNA sequence in a cell, preferably a human cell, for the deamination of a target adenosine in the target RNA sequence by an ADAR enzyme
present in the cell ", which protects ProQR's proprietary ADAR mediated RNA editing platform Axiomer.
further supports the confidence we have in the broad scope conferred by our IP estate protecting our proprietary Axiomer ADAR-mediated
RNA editing technology platform, and the use of chemically modified oligonucleotides to increase stability and recruit endogenous ADAR
to edit the target adenosine," said Ren Beukema, Chief Corporate Development Officer and General Counsel. "Given
the importance and value of our IP portfolio, we intend to continue to defend against such challenges and remain confident in our leading
position, as we progress our science, platform, and pipeline."
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 supports ProQR's ADAR-mediated RNA
editing platform Axiomer. Today ProQR has extensive patent protection related to the Axiomer RNA editing platform, including 15 published
patent families, that currently comprise a total of 28 patents. Beyond this, ProQR has several unpublished patent applications and continuously
invests in expanding its IP estate around ADAR-mediated RNA editing.
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.
Learn more about ProQR at www.proqr.com.
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. Such forward-looking statements include, but are not limited to, statements
regarding our patent estate, including its anticipated strength and our continued investment in it, as well as the potential of our Axiomer
RNA editing technology platform and product candidates. 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. Our actual results could differ materially from
those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other
factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F.
These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and other development activities
by us and our collaborative partners whose operations and activities may be slowed or halted shortage and pressure on supply and logistics
on the global market; our reliance on contract manufacturers or suppliers to supply materials for research and development and the risk
of supply interruption or delays from suppliers or contract manufacturers; the ability to secure, maintain and realize the intended benefits
of collaborations with partners, including the collaboration with Lilly; the possible impairment of, inability to obtain, and costs to
obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and
development; general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties;
and risks related to macroeconomic conditions and market volatility resulting from global economic developments, geopolitical instability
and conflicts.. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements,
and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except
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