ProQR’s Drug Candidate QRX-411 for Usher Syndrome Receives Orphan Drug Designation from FDA and EMA
ProQR Therapeutics N.V. - Ordinary Shares (PRQR)
Last proqr therapeutics n.v. - ordinary shares earnings: 2/26 07:00 am
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Source: GlobeNewswire
Key Updates ProQR’s QRX-411 receives Orphan Drug Designation by the FDA and EMA for the treatment of retinitis pigmentosa, including Usher syndrome, the subtype targeted by QRX-411. Usher syndrome is an inherited condition that is characterized by combined deafness and blindness.QRX-411 targets the pseudo-exon 40 (PE-40) mutation in the USH2A gene and currently there are no therapies commercially available or in clinical development for the vision loss associated with this disease.QRX-411 has shown promising preclinical data in both patient fibroblasts and the optic cup model for mRNA restoration, which was presented at the Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) in May 2017.A lead candidate has been selected for this program and is currently ready for IND-enabling studies.QRX-411 is part of ProQR’s ophthalmology pipeline that currently also includes one clinical compound, QR-110 for Leber’s Congenital Amaurosis Type 10, and three preclinical
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News
- ProQR Nominates Martin Maier, PhD to Board and Announces Annual Meeting of Shareholders to be Held May 22, 2024GlobeNewswire
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- ProQR Achieves Successful Defense of New Challenge to its Axiomer™ IP PortfolioGlobeNewswire
- ProQR Therapeutics (NASDAQ: PRQR) had its price target raised by analysts at Citigroup Inc. from $1.80 to $2.00. They now have a "buy" rating on the stock.MarketBeat
PRQR
Earnings
- 11/7/23 - Miss
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Sec Filings
- 4/23/24 - Form 6-K
- 4/19/24 - Form 6-K
- 3/13/24 - Form 20-F
- PRQR's page on the SEC website