QR-4210 (sepofarsen)
Usher syndrome type 2A / Retinitis Pigmentosa (USH2A exon 13 mutations)
Key Facts
Indication
Usher syndrome type 2A / Retinitis Pigmentosa (USH2A exon 13 mutations)
Phase
Phase 2/3
Status
Discontinued
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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