ProQR Therapeutics Shifts Focus Away from Inherited Retinal Diseases
For many in the inherited retinal disease (IRD) community, news of advancements or setbacks in therapeutic development can profoundly impact hope and future planning. A recent development from ProQR Therapeutics, a company that previously focused on RNA therapies for IRDs, marks a significant shift in their research priorities. This change, while not directly impacting current patients, highlights the challenging and often unpredictable nature of drug development for rare diseases.
According to a report by MedCity News, ProQR Therapeutics has decided to pivot its research and development efforts away from ophthalmology, including inherited retinal diseases, to concentrate on liver and central nervous system (CNS) applications for its RNA-editing technology. This strategic change comes after feedback from the European Medicines Agency (EMA) regarding their lead program, sepofarsen, for Leber congenital amaurosis 10 (LCA10). The EMA recommended an additional clinical trial for sepofarsen before a marketing authorization application could be submitted.
Rather than conducting a new trial, ProQR announced in August 2022 its intention to stop ophthalmology research and seek a strategic partner for all its eye drug assets. This includes sepofarsen, which had reached pivotal testing in LCA10, and ultevursen (QR-421a), previously investigated for Usher syndrome type 2A and non-syndromic retinitis pigmentosa caused by USH2A exon 13 mutations. ProQR estimates that winding down clinical trial work for these two advanced RNA drug candidates will help preserve capital.
The company will now focus exclusively on its Axiomer® RNA-editing technology platform, with initial therapeutic areas including the liver and CNS. This platform aims to make specific single nucleotide edits in RNA to reverse mutations or modulate protein expression. ProQR has already entered into a partnership with Eli Lilly in 2021, licensing five targets in the liver and nervous system areas. The company expects to announce new pipeline targets and provide updates on its partnership with Eli Lilly. In March 2023, ProQR announced initial pipeline programs focused on diseases originating in the liver, with clinical trials anticipated to begin in late 2024 or early 2025. More recently, in June 2026, ProQR announced positive target engagement data from a Phase 1 study of AX-0810, their first investigational Axiomer RNA editing oligonucleotide, in healthy volunteers for cholestatic diseases.
For patients and families affected by IRDs, this news means that ProQR's previously promising ophthalmic programs, such as those for LCA10 and Usher syndrome, will not be advanced by ProQR itself. While ProQR is seeking a strategic partner to continue the development of these ophthalmic assets, their future remains uncertain without a new entity taking them forward. This underscores the critical need for ongoing research and development from a diverse range of companies and institutions to address the many forms of inherited retinal diseases.
ProQR's shift highlights the dynamic landscape of biotech research. While their focus has moved, the broader scientific community continues to explore various avenues for treating IRDs. The search for new treatments remains a vital endeavor, with many other organizations and researchers dedicated to finding solutions for inherited retinal diseases.