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QR-1123

Autosomal Dominant Retinitis Pigmentosa (adRP) caused by P23H mutation in RHO gene

Phase 1/2Active

Key Facts

Indication
Autosomal Dominant Retinitis Pigmentosa (adRP) caused by P23H mutation in RHO gene
Phase
Phase 1/2
Status
Active
Company

About ProQR

ProQR Therapeutics is a clinical-stage biotech company developing transformative RNA therapies for genetic diseases, particularly in ophthalmology. The company leverages its innovative Axiomer® RNA editing platform to create oligonucleotide-based treatments that can correct single nucleotides in RNA, offering a potentially safer and more versatile alternative to DNA editing. While facing clinical setbacks with its earlier candidate sepofarsen, ProQR is advancing a new pipeline of RNA editing candidates and maintains a strategic collaboration with Eli Lilly. The company is publicly traded on the NASDAQ.

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