Drugs / QR-313
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT03605069 | Jul → Dec 2018 | recessive dystrophic epidermolysis bullosa | Phoenicis Therapeutics | Terminated | No outcome recorded Stop: Enrollment |
News releases announcing trial results or a regulatory action · 2
| Date | Issuer | Release |
|---|---|---|
| 2017-11-29 | ProQR Therapeutics | Regulatory ProQR Receives Orphan Drug Designation from EMA for Drug Candidate QR-313 for Dystrophic Epidermolysis Bullosa proqr.com ↗
today announced that investigational drug QR-313 for dystrophic epidermolysis bullosa (DEB) has received orphan drug designation (ODD) from the European Medicines Agency (EMA). |
| 2017-09-19 | ProQR Therapeutics | Regulatory ProQR Receives Orphan Drug Designation from FDA for Drug Candidate QR-313 for Dystrophic Epidermolysis Bullosa and will Present Data at two Scientific Conferences proqr.com ↗
ProQR Therapeutics N.V. (Nasdaq:PRQR) today announced that investigational drug QR-313 for dystrophic epidermolysis bullosa (DEB) has received orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA). |
All press releases naming this drug 5 releases
| Date | Issuer | Release |
|---|---|---|
| 2018-06-28 | ProQR Therapeutics | ProQR Initiates Phase 1/2 Clinical Trial of QR-313 for Dystrophic Epidermolysis Bullosa proqr.com ↗ |
| 2018-06-12 | ProQR Therapeutics | ProQR Receives up to $5 Million in Partnership with EB Research Partnership and EB Medical Research Foundation to Develop QR-313 for the Treatment of Dystrophic Epidermolysis Bullosa proqr.com ↗ |
| 2018-04-30 | ProQR Therapeutics | ProQR Announces Conference Presentations for Axiomer® RNA Editing Technology and QR-313 for DEB proqr.com ↗ |
| 2017-11-29 | ProQR Therapeutics | ProQR Receives Orphan Drug Designation from EMA for Drug Candidate QR-313 for Dystrophic Epidermolysis Bullosa proqr.com ↗ |
| 2017-09-19 | ProQR Therapeutics | ProQR Receives Orphan Drug Designation from FDA for Drug Candidate QR-313 for Dystrophic Epidermolysis Bullosa and will Present Data at two Scientific Conferences proqr.com ↗ |
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | QR-313 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03605069 ↗ |
| Modality | Antisense oligonucleotide | “QR-313 is a 21-nucleotide antisense oligonucleotide (AON)” NCT03605069 ↗ |
| Route | Topical | “QR-313 will be applied topically once daily for 8 weeks of treatment.” NCT03605069 ↗ |
| Target | COL7A1 | “QR-313 is a 21-nucleotide antisense oligonucleotide (AON) designed to hybridize to a specific sequence in the COL7A1 pre-messengerRNA (pre-mRNA).” NCT03605069 ↗ |