ProQr Therapeutics
ProQR Therapeutics is a biotech company dedicated to creating RNA therapies for patients in need. Founded in 2012, it focuses on innovative RNA platform technologies like Axiomer to develop life-changing treatments for genetic disorders and diseases with high unmet medical needs. The company is headquartered in Leiden, Netherlands, with offices in Cambridge, Massachusetts, USA. ProQR's mission is to improve lives through science and technology, leveraging its proprietary RNA repair platform to address rare and prevalent diseases. The company emphasizes patient-focused drug development and integrates the patient voice into its decision-making process.
Industries
Nr. of Employees
medium (51-250)
ProQr Therapeutics
Products
Investigational RNA editing oligonucleotide targeting NTCP
An investigational editing oligonucleotide designed to modify hepatocyte NTCP RNA to produce a variant with reduced bile acid uptake, intended as a potential therapy for cholestatic liver diseases.
RNA base editing platform using ADAR recruitment
Platform for therapeutic single-nucleotide editing in RNA using chemically optimized oligonucleotides that recruit endogenous ADAR enzymes to modulate target transcripts.
Investigational RNA editing oligonucleotide targeting NTCP
An investigational editing oligonucleotide designed to modify hepatocyte NTCP RNA to produce a variant with reduced bile acid uptake, intended as a potential therapy for cholestatic liver diseases.
RNA base editing platform using ADAR recruitment
Platform for therapeutic single-nucleotide editing in RNA using chemically optimized oligonucleotides that recruit endogenous ADAR enzymes to modulate target transcripts.
Services
Collaborative research and licensing of RNA editing platforms
Partnering research and licensing arrangements to discover, develop and commercialize RNA editing-based therapeutic candidates.
Preclinical development and in vivo proof-of-concept studies
Design and conduct of preclinical rodent and non-human primate studies to evaluate editing efficiency, durability, biodistribution, PK/PD and safety.
Clinical development and regulatory submission support
Design and execution of early-phase clinical studies and preparation and submission of regulatory filings to support clinical trial initiation.
Collaborative research and licensing of RNA editing platforms
Partnering research and licensing arrangements to discover, develop and commercialize RNA editing-based therapeutic candidates.
Preclinical development and in vivo proof-of-concept studies
Design and conduct of preclinical rodent and non-human primate studies to evaluate editing efficiency, durability, biodistribution, PK/PD and safety.
Clinical development and regulatory submission support
Design and execution of early-phase clinical studies and preparation and submission of regulatory filings to support clinical trial initiation.
Expertise Areas
- RNA base editing therapeutics
- Oligonucleotide design and medicinal chemistry
- Hepatic delivery and biodistribution optimization
- Preclinical in vivo pharmacology and toxicology
Key Technologies
- ADAR-mediated RNA base editing
- Chemically modified oligonucleotides
- GalNAc-mediated hepatocyte targeting
- Structure-guided oligonucleotide design