Full Press Release Details
Announces Japanese Axiomer Patent Upheld Following Opposition Against Its Leading IP Estate
for ADAR-mediated RNA Editing
Netherlands & CAMBRIDGE, Mass., February 15, 2024 - ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to
changing lives through transformative RNA therapies, today announced it has successfully defended against an opposition filed in Japan
against a patent directed to its ADAR-mediated RNA editing platform Axiomer .
opposition was filed in September 2023 with the Japanese Patent Office by a strawman against ProQR's granted patent JP 7244922,
which is related to the broad concept of using chemically modified oligonucleotides to target specific adenosines within target RNA using
endogenous enzymes and is part of ProQR's intellectual property estate surrounding its Axiomer
ADAR-mediated RNA editing technology platform. The Japanese Patent Office rejected the strawman's opposition and indicated that
all claims were to be maintained as granted to ProQR.
been consistently upheld when challenged by multiple parties in various jurisdictions, further supporting the strength of our leading
IP position in the field of RNA editing using oligonucleotides and endogenous ADAR," said Ren Beukema, Chief Corporate
Development Officer and General Counsel. "The decision by the Japanese Patent Office shows that our granted claims are considered
novel, inventive, and the patent remains in force. 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."
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 13 published patent families, that currently comprise
a total of 27 patents. Beyond this, ProQR has several unpublished patent applications and continuously invests in expanding its IP estate
around ADAR-mediated RNA editing.
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.
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.
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; and general business, operational, financial and accounting risks, and risks related
to litigation and disputes with third parties. 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 as required by law.
For ProQR Therapeutics
ProQR Therapeutics N.V.