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Main focus: RNA therapies for the treatment of genetic diseases
Company stage: Pre-clinical
Diseases (gene editing): Genetic disorders in the liver and nervous system
Genome editing tool: ADAR RNA editing
Funding stage: Private
Location: Leiden, Netherlands and Cambridge, MA, USA
Gene editing partnerships: Eli Lilly
ProQR Therapeutics develops transformative RNA therapies for the treatment of severe genetic rare diseases. Its has developed its own proprietary RNA editing technology tha involves the recruitment of the ADAR enzyme for RNA editing. The company is currently developing the technology at pre-clinical level, and has also established a collaboration with Eli Lilly, which has been granted access to use the technology.