UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 6-K
Report of Foreign Private Issuer
Pursuant to Rule 13a-16 or 15d-16 of
the Securities Exchange Act of 1934
For the month of January 2024
Commission File Number: 001-36622
PROQR THERAPEUTICS N.V.
Zernikedreef 9
2333 CK Leiden
The Netherlands
Tel: +31 88 166 7000
(Address, Including Zip Code, and Telephone
Number,
Including Area Code, of Registrant’s Principal Executive Offices)
Indicate by check mark whether the registrant files or will file annual
reports under cover of Form 20-F or Form 40-F.
Form 20-F x
Form 40-F ¨
Indicate
by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(1): ¨
Indicate
by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(7): ¨
On January 5, 2024, ProQR
Therapeutics N.V. (“ProQR”) issued a press release titled, “ProQR and Rett Syndrome Research Trust Join Forces with
Axiomer™ RNA Editing Collaboration.” A copy of the press release is attached hereto as Exhibit 99.1 and is incorporated
herein by reference.
ProQR hereby incorporates
by reference the information contained herein into ProQR’s registration statements on Form F-3 (File No. 333-270943, File
No. 333-263166, File No. 333-260775 and File No. 333-248740).
SIGNATURES
Pursuant to the requirements of the Securities
Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.
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PROQR THERAPEUTICS N.V. |
| |
|
| Date: January 5, 2024 |
By: |
/s/ René Beukema |
| |
|
René Beukema |
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|
Chief Corporate Development Officer and General Counsel |
INDEX TO EXHIBITS
Exhibit 99.1
ProQR and Rett Syndrome Research Trust Join Forces with Axiomer™
RNA Editing Collaboration
| · | Partnership
focused on utilizing Axiomer™ to develop editing oligonucleotides targeting
an underlying genetic variant that causes Rett syndrome |
| · | Collaboration
expands ProQR’s commitment to advance the development of therapies for central nervous
system diseases |
LEIDEN,
Netherlands & CAMBRIDGE, Mass., January 5, 2024 – ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated
to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform,
today announced a collaboration with the Rett Syndrome Research Trust (RSRT) that will focus on the design and development of editing
oligonucleotides (EONs) using ProQR’s Axiomer technology platform targeting the transcription factor Methyl CpG binding protein
2 (MECP2) and correcting mutations of interest.
“The
Rett Syndrome Research Trust is the leading patient advocacy group championing a cure for Rett syndrome and we look forward to collaborating
with them to further expand the broad applicability of our Axiomer RNA editing technology to Rett syndrome, a rare neurodevelopment disorder
with significant unmet medical need,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “Axiomer has the potential
to restore precise levels of the MECP2 protein, which is lacking in Rett syndrome. This collaboration marks an important step in accelerating
the development of Axiomer in CNS, one of our initial areas of strategic focus along with liver-originated diseases.”
“ProQR’s
leading RNA editing technology platform, coupled with their deep expertise in developing oligonucleotide therapies will offer an exciting
pathway to develop new therapies for Rett Syndrome. RNA approaches can offer several advantages, including, for example, that
because of their small size they do not require viral delivery,” said Bob Deans, RSRT’s Chief Technology Officer and Head
of Research. “Building on work RSRT has been funding in the laboratory of Dr. Peter Beal at UC Davis, we are excited
to now also work closely with ProQR to advance RNA editing as a potential therapeutic approach for Rett syndrome.”
RSRT awarded ProQR approximately $1 million as a research
grant for the initial phase of the project, which will encompass editing oligonucleotide design and optimization, including evaluation
in in vivo models for editing efficacy and MECP2 protein recovery. It is the intent of the partnership to be continued by an expanded
co-funding arrangement following the initial discovery work. The co-funding of the next phase of the collaboration would enable clinical
development of an Axiomer-based therapeutic for Rett syndrome MECP2.
About Axiomer™
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.
About Rett Syndrome
Rett syndrome is a progressive neurodevelopmental
disorder caused by genetic mutations in the Methyl CpG binding protein 2 (MECP2) and diagnosed primarily in females. It is characterized
by apparently normal psychomotor development during the first six to 18 months after birth, followed by a period of developmental stagnation,
then a regression in language and motor skills, followed by long-term relative stability. During the phase of regression, affected patients
develop repetitive, stereotypic hand movements that replace purposeful hand use. Additional symptoms include gait ataxia and apraxia,
seizures, tremors, episodic apnea and/or hyperpnea, gastrointestinal issues, scoliosis and musculoskeletal problems, anxiety and sleep
issues and bruxism.
About ProQR
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 for ProQR
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 “continue,” "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 collaboration with RSRT, including the plan of the collaboration
and the intended benefits thereof, our ability to complete the initial phase of the project and expand the collaboration, our business,
and the further development and advancement of our Axiomer™ platform, including our anticipated strength and
our continued investment in it, as well as the potential of our technologies 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 most recent annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing
and results of preclinical studies and clinical trials 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; the likelihood of our preclinical
and clinical programs being initiated and executed on timelines provided and reliance on our contract research organizations and predictability
of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; 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 potential for future data to alter initial and preliminary results of early-stage clinical trials; the
unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and
continue to advance and progress our clinical programs; the ability to secure, maintain and realize the intended benefits of collaborations
with partners, including the collaboration with Eli Lilly and Company; 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
as required by law.
ProQR Therapeutics N.V.
Investor
contact:
Sarah Kiely
ProQR Therapeutics N.V.
T: +1 617 599 6228
skiely@proqr.com
or
Peter Kelleher
LifeSci Advisors
T: +1 617 430 7579
pkelleher@lifesciadvisors.com
Media
contact:
Robert Stanislaro
FTI Consulting
T: +1 212 850 5657
robert.stanislaro@fticonsulting.com
ProQR Therapeutics NV (NASDAQ:PRQR)
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