PROQR THERAPEUTICS NV

Their core work

ProQR Therapeutics is a Dutch biotech company developing RNA-based therapies for genetic diseases, with a strong focus on rare and inherited conditions. Their core competence is designing oligonucleotide drugs that correct genetic defects at the RNA level, bypassing the need for gene replacement. They have advanced candidates from lab research through first-in-human clinical trials, notably for cystic fibrosis (F508del mutation) and Stargardt disease (inherited blindness). More recently, they contribute to large-scale rare disease screening and digital diagnostic initiatives across Europe.

What they specialise in

How they've shifted over time

ProQR's H2020 journey shows a clear shift from single-disease drug development toward broader rare disease platforms. Their early work (2015-2017) was tightly focused on cystic fibrosis — a specific oligonucleotide therapy targeting the F508del mutation, taken to clinical proof of concept. By 2018-2026, their involvement expanded to inherited blindness (Stargardt disease), genomics tools, genome editing, and large-scale rare disease screening using AI and digital health records — signaling a move from molecule-specific R&D toward platform-level rare disease infrastructure.

ProQR is broadening from single-indication drug development toward digital and genomic platforms for rare disease detection and treatment, making them increasingly relevant for multi-disease consortium initiatives.

How they like to work

ProQR operates as both a project leader and a specialist contributor. They coordinated their largest project (PRO-CF-MED, EUR 4.5M) but participate as partners in broader training networks and IMI-style consortia. With 60 unique partners across 16 countries from just 3 projects, they are comfortable in large, multi-national consortia — suggesting they bring focused biotech expertise into big collaborative frameworks rather than running small bilateral projects.

Despite only 3 H2020 projects, ProQR has built a remarkably wide network of 60 partners across 16 countries, driven by participation in large health consortia. Their network spans most of the EU, with natural concentration in Western European biotech and clinical research hubs.

What sets them apart

ProQR is one of very few European SMEs with hands-on experience taking an RNA-based therapeutic from lab bench to first-in-human clinical trial within an EU-funded project. Their combination of deep oligonucleotide chemistry know-how, clinical development capability, and growing involvement in digital rare disease screening makes them a rare bridge between drug discovery and patient-level diagnostics. For consortium builders, they offer what most academic partners cannot: actual drug development and regulatory pathway experience as an SME.

Highlights from their portfolio

• PRO-CF-MED
  ProQR's flagship — a EUR 4.5M coordinated project running a first-in-human clinical trial for an RNA-targeting cystic fibrosis drug, one of the largest SME-led clinical H2020 projects.
• SCREEN4CARE
  A large-scale rare disease initiative combining newborn genetic screening with machine-learning diagnostics, showing ProQR's expansion from therapeutics into digital health and early detection.
• StarT
  European training network tackling Stargardt disease through genome editing and stem cell biology — demonstrates ProQR's commitment to inherited blindness beyond their core pipeline.