ProQR Partners with Ginkgo Bioworks to Accelerate AI-Driven RNA Editing Drug Discovery
Key Insights
ProQR Therapeutics announced a strategic partnership with Ginkgo Bioworks to leverage autonomous laboratory technology for high-throughput data generation supporting AI-enabled drug discovery for its Axiomer (search) RNA editing platform.
The company established an AI Advisory Board comprising industry leaders from organizations including NVIDIA (search), Owkin (search), and Hugging Face (search) to guide the development of AI strategies for optimizing Axiomer (search) editing oligonucleotides.
ProQR expects to advance its first AI-discovered development candidate into clinical trials with a CTA filing anticipated in mid-2026 and initial clinical data expected by year-end 2026.
ProQR Therapeutics announced a strategic partnership with Ginkgo Bioworks and the formation of an AI Advisory Board to accelerate drug discovery for its proprietary Axiomer (search) RNA editing technology platform. The collaboration aims to leverage artificial intelligence and autonomous laboratory capabilities to enhance the development of RNA-based therapeutics.
Strategic Partnership Enhances Data Generation Capabilities
The partnership with Ginkgo Bioworks provides ProQR access to Ginkgo's 50+ instrument autonomous laboratory, Nebula. According to the company, this autonomous lab removes a main bottleneck in AI-enabled drug discovery by increasing data generation throughput and speed. The capability is expected to increase the scale and speed of experimental data generation, enabling more efficient discovery timelines and improving the ability of ProQR's discovery AI models to predict drug candidates.
"Over the last 18 months we have developed an AI model that leads to significant acceleration and improvement of editing oligonucleotides in drug discovery," said Daniel A. de Boer, Founder and Chief Executive Officer of ProQR. "We're pleased to have announced the partnership with Ginkgo Bioworks, providing us access to their state-of-the-art autonomous lab enabling high throughput data generation to further scale up our AI-enabled drug discovery."
As part of the partnership agreement, Ginkgo made a strategic equity investment in ProQR.
AI Advisory Board Brings Industry Expertise
ProQR established an AI Advisory Board comprising leaders from the AI and techbio fields to support the development of the company's AI strategy for Axiomer (search). The Advisory Board will provide guidance on how ProQR applies AI in its discovery efforts, including best practices, emerging approaches, and insights from the field to help accelerate drug discovery and optimization of Axiomer editing oligonucleotides.
The AI Advisory Board includes:
- Eric Durand, PhD, Chief Data Science Officer, Owkin
- David Ruau, PhD, Head of BD EMEA, Healthcare & Life Sciences, NVIDIA
- Thomas Wolf, Co-Founder and Chief Science Officer of Hugging Face
- Gerard van Westen, Full Professor AI and Medicinal Chemistry at Leiden University
- Romain Lacombe, Venture Partner, HCVC
- Imran Haque, PhD, Chief Technology Officer, Kimia Therapeutics
Clinical Timeline for AI-Generated Programs
ProQR expects to advance its first programs generated using its AI-enabled discovery approach into the clinic, with a clinical trial application (CTA) anticipated in mid-2026 and initial clinical data anticipated by year-end 2026. The company plans to provide further details on its AI-enabled drug discovery and AI-generated development candidates at its investor and analyst event.
Axiomer Technology Platform
ProQR is pioneering Axiomer (search), a next-generation RNA base editing technology that could potentially yield a new class of medicines for diverse types of diseases. Axiomer "Editing Oligonucleotides," or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery present in human cells called ADAR (search) (Adenosine Deaminase Acting on RNA).
The technology works by recruiting and directing endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA. An Inosine is translated as a Guanosine (G), correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease.
Ongoing Development Programs
The company continues to advance its lead development pipeline programs, including the ongoing Phase 1 clinical study of AX-0810 in NTCP (search) for cholestatic diseases (search). ProQR plans to unveil the primary indication for AX-0810 at its upcoming investor event, along with showcasing its broader RNA editing pipeline and announcing new programs that will have clinical data readouts.