ProQR Therapeutics

The fifth edition of the Axiomer Summit, our bi-annual internal event, brought ProQRians together for a day of science and collaboration! From thought-provoking discussions to a vibrant poster session, the event highlighted the remarkable progress and cutting-edge science driving our Axiomer platform forward. A huge thank you to our Scientific Advisory Board and Board members for their valuable contributions. #RNAEditing #Axiomer #Biotech #Innovation 

In connection with today's announcement, ProQR has made available a video presentation featuring it's Management Team discussing the Phase 1 AX-0810 data, the clinical validation of the Axiomer RNA editing platform, AX-0811 and next steps with NTCP. The video presentation is available at https://lnkd.in/eH4eKu3a #Axiomer #RNAEditing #Biotech

Last week, we welcomed ProQR co-founder Dinko Valerio back to ProQR to share reflections on the company’s journey since 2012. From the earliest days, when ProQR was still an ambitious idea to where we are today, advancing our Axiomer RNA editing platform in the clinic.    Dinko’s perspective on the company’s evolution, challenges along the way, and opportunities ahead made for an inspiring conversation with the team. We are grateful to Dinko for his contributions over the years and for sharing his passion and insights with us. #Axiomer #RNAEditing #Biotech

On 15 May, we recognize Mucopolysaccharidoses (MPS) Awareness Day.  MPS is a group of rare genetic lysosomal storage diseases that cause progressive, multisystem damage over time. Hurler syndrome, the most severe form of MPS I, begins in early life and is associated with significant disease burden, impacting both quality of life and life expectancy. Awareness days like this bring visibility to MPS and the need for continued progress in developing new treatment approaches. During our recent Investor and Analyst Event, we announced AX-0422, a potentially transformative #RNAEditing therapy designed to correct a specific mutation in the IDUA gene present in many people living with Hurler syndrome. Learn more here: https://lnkd.in/eaNV_cpV #ShineforMPS #HurlerSyndrome #RareDisease #Axiomer

We’re excited to share that ProQR will present data across multiple scientific conferences this May, highlighting the breadth of our Axiomer™ RNA editing platform. Across liver and CNS programs, these presentations reinforce the potential of RNA editing to address both rare and prevalent diseases. Presentations include:  • TIDES USA (Boston, MA) - Pipeline overview, highlighting AX-0422 targeting IDUA in Hurler syndrome  • ASGCT (Boston, MA) - AX-2402 for Rett syndrome, in vivo data  • ASGCT (Boston, MA) - AX-2911 targeting PNPLA3 in MASLD/MASH, in vivo data  • RNA Society Annual Meeting (Montreal, Canada) - RNA editing platform advancements  • EASL (Barcelona, Spain) - AX-2911 targeting PNPLA3 in MASLD/MASH, in vivo data   For more details, check out the PR: https://lnkd.in/eFWuUK-P   #RNAEditing #Biotech #TIDESUSA #ASGCT #RNASociety #EASL

Drug discovery can be slow and patients are waiting. We have partnered with Ginkgo Bioworks to advance our #Axiomer #RNAEditing platform, leveraging their autonomous lab for high throughput data generation to power our AI-driven drug discovery. Our CEO Daniel de Boer and Ginkgo CEO Jason Kelly sat together to discuss how our collaboration is accelerating timelines. #Biotech #AI

Following our partnership announcement with Ginkgo Bioworks, our CEO Daniel de Boer sat down with Ginkgo CEO Jason Kelly to discuss the opportunity ahead. Hear how our #Axiomer #RNAEditing platform, automation, and data can accelerate the path from target discovery towards delivering therapies to patients.  #Biotech #AI 

Today we highlighted key updates in our Investor and Analyst event titled, “Expanding the Axiomer™ RNA Editing Opportunity Beyond AX-0810.” With AX-0810 clinical target engagement data readout in healthy volunteers on track for this quarter, we are excited to share that biliary atresia has been selected as our initial Phase 2 indication. We also highlighted the following from our pipeline: • AX-0811, a next generation NTCP program for cholestatic diseases generated by our AI-enabled discovery engine • AX-0422 targeting IDUA for Hurler syndrome • AX-2911 targeting PNPLA3 for MASH advancing toward early clinical data generation with plans for a first-in-human (FIH) investigator-initiated trial (IIT) in China We continue to advance our Axiomer platform using AI-enabled discovery capabilities. Check out the PR for more details including upcoming milestones https://lnkd.in/ek_jKvAZ and watch the replay of the event: https://lnkd.in/eVU9wbre #Axiomer #RNAEditing

In one hour, at 10:00 ET, we are hosting a virtual Investor and Analyst event titled, “Expanding the Axiomer™ RNA Editing Opportunity Beyond AX-0810." We’ll highlight our broader #RNAEditing pipeline, introduce new programs with upcoming clinical readouts in the runway, and share the primary indication for AX-0810 ahead of the target engagement data, anticipated in H1 2026. For additional information about the event and to register, visit:https://lnkd.in/eVU9wbre #Axiomer #Biotech

We are pleased to share that we have partnered with Ginkgo Bioworks to leverage their autonomous lab for high throughput data generation. In addition, we have established an AI Advisory Board with leading experts to support the development of the ProQR’s AI strategy to accelerate drug discovery and optimization of Axiomer editing oligonucleotides. ProQR’s first AI-discovered Development Candidate is expected to enter the clinic in 2026. Learn more at our Virtual Investor & Analyst Event today at 10:00 ET. We’ll also share updates on our broader #RNAEditing pipeline, introduce new programs with upcoming clinical readouts, and unveil the primary indication for AX-0810 ahead of target engagement data anticipated in H1 2026. To register for the event, visit: https://lnkd.in/eVU9wbre #Axiomer #Biotech