ProQR Therapeutics N.V., a biopharmaceutical company, engages in the discovery and development of RNA-based therapeutics for the treatment of genetic disorders. It primarily develops sepofarsen that is in phase II/III clinical trial illuminate trial for treating leber congenital amaurosis 10 disease; and ultevursen, which is in phase II/III clinical trial to treat USH2A-mediated retinitis pigmentosa and usher syndrome. The company also engages in the developing of Axiomer RNA base-editing platform technology. It has a license agreement with Radboud University Medical Center, Inserm Transfert SA, Ionis Pharmaceuticals, Inc., and Leiden University Medical Center, as well as license and research collaboration with Eli Lilly and Company for the discovery, development, and commercialization of potential new medicines for genetic disorders in the liver and nervous system. ProQR Therapeutics N.V. was incorporated in 2012 and is headquartered in Leiden, the Netherlands.

ProQR Therapeutics N.V.

ProQR Therapeutics develops RNA therapies for patients with genetic disorders, utilizing its Axiomer technology for RNA base editing.

ProQR Therapeutics

ProQR Therapeutics | 13,848 followers on LinkedIn. Advancing our proprietary Axiomer™ RNA-editing platform technology | Changing lives is what drives us at ProQR Therapeutics. We are advancing our proprietary Axiomer™  RNA-editing platform technology. Our highly energetic team strives to combine innovative technologies and entrepreneurism to make a meaningful impact on the lives of individuals and the people around them.

Janne Turunen

Translational Drug Development Expert

Sjef De Kimpe

VP Business Development at ProQR Therapeutics NV

Andrew Morris

Workplace | Facilities | Project management | Bio science

Fred Jaspers

It was a pleasure to host @BiotechTVHQ at our office in Leiden. Thanks for visiting @BradLoncar – hope you enjoyed learning more about our Axiomer #RNAEditing technology, pipeline, and what its like being a #biotech in the Netherlands! #ItsInOurRNA #Axiomer #ADAR https://lnkd.in/g2nJ2eRZ

Great connecting with the field and sharing our progress at the 20th Annual Meeting of the Oligonucleotide Therapeutics Society #OTS2024 #oligomeeting . You can find our presentation under “Publications and Presentations” on our website: https://lnkd.in/dw38uu4 $PRQR #RNAEditing #ADAR #Axiomer

Last weekend, ProQRians participated in a boat parade along the Leiden canals as part of the #PrideLeiden celebration. Thanks to all who joined and to our DEI team for organizing! Learn more about our commitment to an inclusive culture: proqr.com/careers

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.”

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