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ProQR Therapeutics

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.


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Company: ProQR Therapeutics
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