Challenges and successes 2020 Recap by our CEO, Daniel de Boer

Our founder and CEO Daniel de Boer looks back at the challenges and successes of 2020 and discusses the milestones to look for in the upcoming year.

Daniel de Boer wearing a blue suit stands with arm crossed in a laboratory setting.

I view the year ahead with optimism in bringing us closer to our goal of stopping vision loss.

Daniel de Boer

During 2020 we have all faced significant challenges because of the COVID-19 pandemic. This was not different for ProQR. Despite these challenges and because of the dedication and hard work of many, we have been able to achieve important milestones for the company and with that for the communities we serve. I view the year ahead with optimism in terms of bringing us closer to our goal of stopping vision loss in people with genetic eye diseases.

For our lead program sepofarsen we have completed enrollment of Illuminate, our Phase 2/3 trial that is intended to support application for marketing approval. If approved, sepofarsen has the potential to be the first therapy to address this high unmet medical need for patients with CEP290 mediated Leber congenital amaurosis 10 who would otherwise face blindness.

We also completed enrollment of Stellar, a Phase 1/2 clinical trial of QR-421a for USH2A mediated Usher syndrome and retinitis pigmentosa. Our extensive pipeline beyond sepofarsen and QR-421a further includes additional RNA therapies for inherited retinal diseases and other genetic eye diseases ranging from discovery stage to early clinical development.

In 2020 we strengthened our management team and scientific advisory board with the appointment of several renowned experts in the field of RNA therapeutics and ophthalmology. We also bolstered our financial position extending our cash runway into 2023.

A close up of Daniel de Boer

I started ProQR to make a difference for those living with rare genetic conditions

Daniel de Boer

I started ProQR to make a difference for those living with rare genetic conditions and this is what I will continue to strive for in the work we do each day at ProQR. How we do our work changed dramatically in 2020, but why we do our work – to help people living with rare diseases that currently have no treatment options – remains our steadfast focus. I wish to thank everyone supporting this mission: our shareholders, employees, and collaborators, but especially the participants in our clinical trials and their caregivers.

Daniel A. de Boer