ProQR Therapeutics

ProQR Announces Second Quarter 2024 Operating and Financial Results   Read the press release here: https://lnkd.in/epb6d63c   $PRQR #RNAediting  

Pleased to have attended the 4th Annual Oligonucleotides for CNS Summit this week, learning from and engaging with the field. #RNAEditing

As pride month comes to a close, we caught up with our Lead Talent Acquisition and DEI team member, Silvia Catellani, to talk about ProQR, Pride, and driving an inclusive culture for present and future ProQRians. Learn more at the blog post at the link below. #PrideMonth #BeOpenBeCuriousBeYourself https://lnkd.in/eVtR8JKS

Embracing Diversity: Why it Matters As European Diversity Month comes to a close, the ProQR Diversity, Equity, and Inclusion (DEI) team have posted their latest blog: https://bit.ly/4dQ8BzD This Diversity Month let's recommit ourselves to fostering an inclusive culture where every voice is heard, and every perspective is valued. Let's continue to learn from one another, challenge our assumptions, and build a more equitable future, both within our organization and beyond. #DiversityMonth #Inclusion #EmbraceDiversity

ProQR’s CEO Daniel de Boer spoke to BiotechTV at #ASGCT2024 about the new preclinical data we presented at the conference on our Axiomer AX-0810 #RNAediting pipeline program targeting NTCP for Cholestatic Diseases. In the interview, Daniel shared more about the exciting potential of our technology and the significance of ProQR reporting proof of target engagement leading to meaningful changes in biomarkers in NHPs. Tune into the clip to learn more about why we these preclinical data are an important derisking step as we progress to enter the clinic in late 2024/early 2025. https://lnkd.in/geg5w6UN

Starting in 30 minutes we will host an investor webcast with members of the ProQR Management Team to highlight the data being presented in the ASGCT poster session and will also conduct an analyst Q&A session. To join, follow this link: https://bit.ly/44GyYDW #RNAediting

ProQR Announces First Quarter 2024 Operating and Financial Results Today we have reported our financial and operating results for the first quarter ended March 31, 2024, and provided a business update. Read the full press release here: https://bit.ly/46TZf0S Speaking on the announcement, Daniel A. de Boer, Founder and CEO of ProQR, said: “The preclinical proof of concept data for our NTCP program at ASGCT, demonstrating for the first time in the ADAR RNA editing field in vivo proof of target engagement with meaningful changes in biomarkers in NHPs marks an exciting milestone for ProQR and further supports the confidence we have in our Axiomer technology. Moving forward, we expect multiple additional milestones this year as we progress our first editing oligonucleotide pipeline programs to the clinic in late 2024/early 2025. In the second half of the year, we will share translational data and more about our clinical plans for AX-0810, as well preclinical proof of concept and translational data for AX-1412 targeting B4GALT1 for cardiovascular diseases. As a company founded to make a difference for patients, we are eager to embark on this next stage in our evolution as the leading ADAR RNA editing company equipped with strong science driving a biomarker effect in preclinical models, a robust IP estate, a fruitful partnership with Eli Lilly, and cash runway into mid-2026.”

We are pleased to announce that today we have published new preclinical data for our proprietary Axiomer™ RNA editing technology platform, including the first preclinical proof of concept data for its AX-0810 pipeline program for cholestatic diseases targeting NTCP. The data are being presented at a poster session today at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, May 7-11, 2024, in Baltimore, Maryland. Read the full PR here: https://bit.ly/3JV2pbL Speaking on the news today, Gerard Platenburg, Chief Scientific Officer and co-founder, ProQR, said: “Our Axiomer ADAR RNA editing data at ASGCT show the exciting potential of our technology and for the first time in the field we report proof of target engagement leading to meaningful changes in biomarkers in NHPs. In our AX-0810 program targeting NTCP for cholestatic diseases, we have demonstrated preclinical proof of concept showing that Axiomer RNA editing oligonucleotides can have a meaningful effect on levels of serum biomarker bile acid. We believe this is a significant derisking step as we approach the clinic as our NHP studies closely resemble the design of the first in human trial that will focus on target engagement and biomarkers. We look forward to sharing translational data for our AX-0810 program and to announcing our clinical program candidate later this year, as we progress to enter the clinic in late 2024/early 2025.” Check out the latest scientific posters from ASGCT here: https://bit.ly/3O5KHEX #RNAediting #ASGCT2024 #Axiomer

A nice weekend read. We are pleased to share the recent Nature article: “RNA-editing drugs advance into clinical trials” https://lnkd.in/e65CnBBD. Published this week reviewing companies working on the next generation of ADAR-based medicines. #ADAR #RNAediting

Today we announced that ProQR has again successfully defended against opposition filed against a key patent for its ADAR-mediated #RNAediting platform Axiomer™. The opposition was filed in June 2021 with the European Patent Office (EPO) and 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. Read the full release here: https://bit.ly/3vXeo5g Speaking on the decision, René Beukema, ProQR’s CCDO, outlined: “This decision 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. 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 its 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. #RNA #biotech #ADAR