ProQR to Present Axiomer RNA Editing Platform Technology at RNA Editing Summit

Rhea-AI Summary

On April 4, 2022, ProQR Therapeutics (Nasdaq: PRQR) announced its participation in the 3rd RNA Editing Summit in Boston, MA. Co-founder Gerard Platenburg will present Axiomer^®, a proprietary RNA base editing platform, on April 6 at 11:00am ET. This innovative technology utilizes the body's ADAR editing machinery to reverse disease-causing RNA mutations. Axiomer^® has broad potential, targeting genetic diseases like Leber congenital amaurosis and Usher syndrome, with a strong patent portfolio reinforcing its promise in treating previously untreatable conditions.

LEIDEN, Netherlands & CAMBRIDGE, Mass., April 04, 2022 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA therapies for genetic eye diseases, today announced a presentation at the 3^rd RNA Editing Summit held April 5-7 in Boston, MA, US.

On Wednesday, April 6 at 11:00am ET Gerard Platenburg, co-founder and Chief Innovation Officer of ProQR will present our proprietary RNA Base Editing Axiomer^® Technology including editing efficiency in various models.

“Our Axiomer^® RNA base editing platform uses the body’s own editing machinery called ADAR to make single nucleotide edits in RNA,” said Gerard Platenburg, co-founder and Chief Innovation Officer of ProQR. “With this approach disease-causing mutations can be reversed, and specific proteins can be altered. The platform utilizes oligonucleotides that can be delivered to the eye as well as various other tissues like the liver and nervous system which are being explored as targets in our collaboration with Eli Lilly. With broad applicability, and a leading patent portfolio in the ADAR editing space, Axiomer^® has significant potential to target genetic diseases otherwise thought untreatable.”

About Axiomer^®

ProQR is pioneering a next-generation RNA base editing technology called Axiomer^®, which could potentially yield a new class of medicines for genetic 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. The Axiomer^® EONs are designed to recruit an endogenously expressed RNA editing system called ADAR (Adenosine Deaminase Acting on RNA), which can direct the change of an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G).

About ProQR

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA
therapies for the treatment of severe genetic rare diseases such as Leber congenital
amaurosis 10, Usher syndrome and retinitis pigmentosa. 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 Axiomer and our participation in the RNA Editing Summit. 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. 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
Hans Vitzthum
LifeSci Advisors
T: +1 617 430 7578
hans@lifesciadvisors.com

Media Contact:
Robert Stanislaro
FTI Consulting
T: +1 212 850 5657
robert.stanislaro@fticonsulting.com

FAQ

What is the Axiomer® technology presented by ProQR at the RNA Editing Summit?

Axiomer® is ProQR's proprietary RNA base editing platform that utilizes the body's ADAR machinery to make precise edits in RNA, reversing mutations linked to genetic diseases.

When is ProQR's presentation at the RNA Editing Summit?

ProQR's presentation will take place on April 6, 2022, at 11:00am ET during the RNA Editing Summit in Boston, MA.

What genetic diseases can Axiomer® potentially target?

Axiomer® has the potential to target severe genetic diseases such as Leber congenital amaurosis, Usher syndrome, and retinitis pigmentosa.

Who is presenting on behalf of ProQR at the RNA Editing Summit?

Gerard Platenburg, the co-founder and Chief Innovation Officer of ProQR, will present at the summit.

What date did ProQR announce its participation in the RNA Editing Summit?

ProQR announced its participation on April 4, 2022.