PRQR - ProQR Therapeutics N.V. (PRQR) ASGCT Investor Webcast (Transcript)
2024-05-11 17:57:09 ET
ProQR Therapeutics N.V. (PRQR)
ASGCT Investor Webcast
May 09, 2024 08:00 AM ET
Company Participants
Sarah Kiely - IR
Daniel de Boer - Founder and CEO
Gerard Platenburg - Chief Scientific Officer
Conference Call Participants
Jon Wolleben - Citizens JMP
Steve Seedhouse - Raymond James
Presentation
Sarah Kiely
Thank you, and good day, everyone. We appreciate you joining our event today. Today, we are pleased to highlight new preclinical proof-of-concept data for our AX-0810 program targeting NTCP, which was presented yesterday at the ASGCT Annual Meeting in Baltimore. On Slide 2, you'll find the agenda for our call and our speakers. From the management team are Daniel de Boer, our Founder and CEO, who will provide a brief introduction; and then Gerard Platenburg, our Chief Scientific Officer, who will take us through the data presented at ASGCT. Following the presentation, we will have a Q&A session with covering analysts before we conclude the call. Today's event is being recorded, and we will have a replay available on our Web site following the event. You can also find our ASGCT poster under the Publications and Presentations section of our Web site. On Slide 3 is our forward-looking statements. During the call today, we will make forward-looking statements. There are risks and uncertainties associated with an investment in ProQR, which are described in detail in our SEC filings.
I will now turn the call over to Daniel. Daniel?
Daniel de Boer
Thank you, Sarah, and good morning, and good afternoon, everyone. Thank you for joining us today. We are pleased to share an update with you on the data that we shared at the ASGTC Annual Meeting. But first, I'll provide a brief overview of the company's strategy and our proprietary Axiomer RNA editing platform. Axiomer was invented at the ProQR Labs in 2014 and uses the well-proven modality of oligonucleotides to recruit a novel mechanism of action. Axiomer uses editing oligonucleotides or EONs, to recruit endogenous ADAR to edit individual basis in RNA. ADAR is present in all human cells, and RNA editing is a naturally recurring process. In fact, it's happening in all of us right now. Our proprietary Axiomer platform makes use of the ADAR mechanism that nature has developed and recruits it to edit specific nucleotides in a targeted way. Preclinical platform data demonstrate that Axiomer is broadly validated across multiple genes. And today, we'll focus on the preclinical proof of concept for our AX-0810 program, starting NTCP for cholestatic diseases. Our strategy includes both in-house development of pipeline programs, initially including AX-0810 as well as AX-1412, targeting the B4GALT1 gene for cardiovascular disease and using the Axiomer technology for selective partnering and expertise in pathology, like in our partnership with Eli Lilly, allowing us to capture the full value of this platform technology....
ProQR Therapeutics N.V. (PRQR) ASGCT Investor Webcast (Transcript)