ProQR's Drug Candidate QRX-411 for Usher Syndrome

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Vacancies; Contact; Events; Science & Pipeline. Pipeline. Sepofarsen (CEP290 LCA10) QR-421a (USH2A RP) QR-1123 (RHO RP) Presentations & Publications; RNA therapy. Axiomer technology; Patients & Community.

ProQR has programs in multiple therapeutic areas, such as cystic fibrosis and the rare skin condition Dystrophic epidermolysis bullosa, but ophthalmology assets make up the bulk of its pipeline. Key updates At ARVO ProQR will present additional positive pre-clinical proof-of-concept data for QR-110 targeting LCA 10. The company has recently received IND clearance to start clinical | January 11, 2021 ProQR plans to independently advance its pipeline of RNA medicines to establish a multi-product, platform company in IRD. By 2023, the company expects its pipeline to have at least two commercial products, and at least three late-stage and seven early-stage programs in development. Proven leader with deep worldwide ophthalmology experience, including multiple approvals globallyLEIDEN, Netherlands & CAMBRIDGE, Mass., Oct. 05, 2020 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V 2016-03-01 · ProQR to Highlight a Growing Pipeline During a Research & Development Day and Present at the Barclays Global Healthcare Conference March 01, 2016 07:00 ET | Source: ProQR Therapeutics N.V. ProQR LEIDEN, Netherlands & CAMBRIDGE, Mass., April 02, 2021 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA ProQR Therapeutics N.V. Investor Contact: Sarah Kiely ProQR Therapeutics N.V. T: +1 617 599 6228 skiely@proqr.com or Hans Vitzthum LifeSci Advisors T: +1 617 535 7743 hans@lifesciadvisors.com ProQR Therapeutics N.V. (Nasdaq: PRQR) shares rallied to close up 62% to $9.22, after initially only adding 5% Wednesday following the release of data from its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP). In an interview with PharmaShots, Daniel A. de Boer of ProQR shared his views on P-II/III pivotal trial of Sepofarsen to treat the root cause of a rare eye disease, Leber congenital amaurosis 10 (LCA10) Shots: The Illuminate P-II/III trial of sepofarsen is a double-masked, randomized, controlled, multiple-dose study to evaluate if sepofarsen is effective […] Jul 14, 2020 “Pontifax is pleased to enter into this strategic financing partnership with ProQR as it continues to advance its pipeline of RNA therapies for ophthalmology candidates in our pipeline, as we believe that RNA oligonucleotides hold great promise for the treatment of a variety of genetic eye diseases.”.

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About ProQR ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe orphan diseases such as cystic fibrosis and Leber’s congenital amaurosis. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.

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We have made significant progress in advancing our broad pipeline of investigational medicines through the various stages of drug development, from early discovery to late stage clinical trials. Science & Pipeline RNA technology to benefit patients. RNA technology to benefit patients.
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“Since last year we have made good progress on executing on our strategy to develop life-changing therapies for patients in need, through a diversified pipeline with a balanced risk profile,” said Daniel A. de Boer, CEO of ProQR. Key updates At ARVO ProQR will present additional positive pre-clinical proof-of-concept data for QR-110 targeting LCA 10. The company has recently received IND clearance to start clinical | January 11, 2021 Our Pipeline. Portfolio Pipeline menu item, submenu; Approved Medicines menu item, submenu; Clinical Trials menu item, submenu; News & Resources. Newsroom menu item, submenu; Press Releases menu item, submenu; Resources for Patients menu item, submenu; Resources for Health Care Professionals menu item, submenu; Careers.

ProQR has a U.S. campus in Cambridge, Mass., but is headquartered out of The Netherlands. This "Tier 4" developmental concern is focused on the development of RNA therapies for Our current pipeline includes treatments for Leber congenital amaurosis, Usher syndrome and retinitis pigmentosa. ProQR Therapeutics’s tracks ProQR Talks episode 3 "Empowering the IRD community with knowledge of their genetic diagnosis" by ProQR Therapeutics published on 2020-10-22T12:55:31Z ProQR Therapeutics N.V is primarely in the business of pharmaceutical preparations. For financial reporting, their fiscal year ends on December 31st. This page includes all SEC registration details as well as a list of all documents (S-1, Prospectus, Current Reports, 8-K, 10K, Annual Reports) filed by ProQR Therapeutics N.V..
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2017-06-15 · Bloomberg the Company & Its Products The Company & its Products Bloomberg Terminal Demo Request Bloomberg Anywhere Remote Login Bloomberg Anywhere Login Bloomberg Customer Support Customer Support We are ProQR. About us; Vision 2023; Leadership; Careers; Contact; Science & Pipeline. Research and development pipeline; Clinical Trials. QR-1123 Aurora phase 1/2 study for adRP; Sepofarsen ILLUMINATE phase 2/3 study for LCA10; QR-421a STELLAR phase 1/2 study for Usher syndrome; Sepofarsen INSIGHT – phase 1b/2 study for LCA10; Sepofarsen ProQR Therapeutics N.V.Investor Contact: Sarah Kiely ProQR Therapeutics N.V. T: +1 617 599 6228 skiely@proqr.com or Hans Vitzthum LifeSci Advisors T: +1 617 535 7743 hans@lifesciadvisors.comMedia ProQR’s pipeline now includes two clinical programs, one preclinical program and two programs ready to enter development. “Since last year we have made good progress on executing on our strategy to develop life-changing therapies for patients in need, through a diversified pipeline with a balanced risk profile,” said Daniel A. de Boer, CEO of ProQR. LEIDEN, Netherlands, Nov. 30, 2015 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe orphan diseases such as cystic fibrosis (CF) and Leber's congenital amaurosis (LCA), today presents an update on the innovation pipeline and announces an R&D day for investors.

2021-03-25 · ProQR Therapeutics shares reached a new 52-week high after the company reported highly positive results from its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP) due to USH2A exon 13 mutations. Key updates At ARVO ProQR will present additional positive pre-clinical proof-of-concept data for QR-110 targeting LCA 10. The company has recently received IND clearance to start clinical | January 11, 2021 2017-06-15 · Bloomberg the Company & Its Products The Company & its Products Bloomberg Terminal Demo Request Bloomberg Anywhere Remote Login Bloomberg Anywhere Login Bloomberg Customer Support Customer Support 2021-03-24 · Shares of ProQR Therapeutics (NASDAQ: PRQR) were trading higher on Wednesday after the company released positive results from a clinical trial for one of its leading pipeline candidates, QR-421a. The biotech is also benefiting from bullish commentary from a Wall Street analyst. ProQR plans to independently advance its pipeline of RNA medicines to establish a multi-product, platform company in IRD. By 2023, the company expects its pipeline to have at least two commercial products, and at least three late-stage and seven early-stage programs in development. ProQR has programs in multiple therapeutic areas, such as cystic fibrosis and the rare skin condition Dystrophic epidermolysis bullosa, but ophthalmology assets make up the bulk of its pipeline. ProQR’s pipeline now includes two clinical programs, one preclinical program and two programs ready to enter development.
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ProQR Announces Positive Top-Line Results from a Phase 1b

RNA technology to benefit patients. At ProQR science is literally at the heart of what we do. The offices at our headquarters in Leiden are centered around the laboratories where our scientists discover and test our novel RNA therapies.