PRQR boasts a pipeline of 18 drug candidates, nearly all in early-stage development. Cash runway to last through to 2021 following recent public offering.

2019-03-13 · The therapies in the ProQR pipeline utilize an RNA oligonucleotide technology platform to repair genetic defects, targeting significant parts of the mutations that are known causes of inherited

Learn more At ProQR we develop antisense oligonucleotides, or RNA therapies, to treat genetic diseases that are rare. 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. Our platform for inherited retinal diseases Science & Pipeline 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. ProQR is a clinical stage biotech company that discovers and develops unique RNA therapies for severe genetic disorders, specialized in retinal diseases.

The company’s current pipeline includes potential treatments for rare genetic diseases including Leber's congenital amaurosis, (LCA10) dystrophic epidermolysis bullosa and Usher syndrome. 2019-03-13 · The therapies in the ProQR pipeline utilize an RNA oligonucleotide technology platform to repair genetic defects, targeting significant parts of the mutations that are known causes of inherited ProQR intends to use the net proceeds from the offering, together with its existing cash and cash equivalents, to advance clinical development of its product candidates, to progress its other pipeline candidates, to advance its RNA-editing technology platforms, and for working capital and other general corporate purposes. The programs in ProQR’s pipeline utilize the RNA oligonucleotide technology platform that repairs the genetic defect in the RNA to address the underlying cause of genetic diseases. The Dutch biotech has firmed up its finances as its promising pipeline of RNA therapies for rare genetic eye disease starts to move through the clinic. ProQR Raises $90m To Advance Eye Disease Pipeline :: Scrip QR-313 is the short term catalyst amongst many that ProQR has in their pipeline. The company is expected to present Phase I/II interim data in the first quarter of 2019.

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R&D pipeline RNA therapies for rare genetic diseases At ProQR we develop antisense oligonucleotides, or RNA therapies, to treat genetic diseases that are rare. 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.

View all Eluforsen ProQR Therapeutics NV executive leadership team, overseeing discovery and development of a broad pipeline targeting areas of high unmet medical need. 5 days ago Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.

ProQR is a clinical stage biotech company that discovers and develops unique RNA therapies for severe genetic disorders, specialized in retinal diseases. ProQR Therapeutics - Striving to reverse blindness

Allergan and Editas Medicine have initiated a Phase 1/2 trial of gene-editing agent EDIT-101, which aims to treat Mar 4, 2020 ProQR Therapeutics. Overview; R&D Platform; Pipeline; Products; Services ProQR Therapeutics is a Leiden, NL based biotech company The company was founded in 2019 by EB Research partnership and ProQR Therapeutics to continue development of a pipeline of potential RNA therapies for Its product pipeline include Sepofarsen, QR-421a, QR-1123, and QR-504a. The company was founded by Daniel de Boer, Gerard Platenburg, Henri Termeer, and ProQR Therapeutics – QR-421a program update March 2021.

The offices at our headquarters in Leiden are centered around the laboratories where our scientists discover and test our novel RNA therapies. 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. Learn more about our Pipeline of RNA therapies. Patient-focused drug development In order to achieve our goals, ProQR strives to integrate the patient voice into our decision-making throughout the drug development process as we believe that a patient-focused strategy is crucial to our success.
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Learn more. We aim to make a difference for these patients as we work to advance our pipeline of programs to treat inherited retinal diseases.” About QR-421a. QR- 421a is a Science & Pipeline SubmenuScience & Pipeline.

Patient-Centric Approach menu item, submenu; Global Compliance Program menu item, submenu Key program features and updates: ProQR to host an R&D day in New York today, June 15, from 8:00am to 1:00pm Eastern Standard Time. The live webcast can be accessed at | April 23, 2021 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. Despite this, ProQR can still be a very interesting candidate to add to your watchlist.
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ProQR Announces Positive Findings From an Interim Analysis in the Phase 1/2 trial of QR-421a for Usher Syndrome and Provides Business Update 31 March 2020 at 6:00 AM EDT QR-421a showed early and encouraging evidence of activity, with 25% of patients showing a benefit across multiple concordant outcome measures and was well tolerated with no serious adverse events

The company is expected to present Phase I/II interim data in the first quarter of 2019. With March newly here, the company is approaching the timeline that they set out. “ProQR Vision 2023” strategy and key goals.

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To navigate items, use the arrow, home, and end keys. Our Company. About PTC Therapeutics menu item, submenu. Patient-Centric Approach menu item, submenu; Global Compliance Program menu item, submenu

Hedge fund activity in ProQR Therapeutics NV (NASDAQ:PRQR) At Q1's end, a total of 8 of the hedge funds tracked by Insider Monkey held long positions in this stock, a change of -27% from the 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 Key Updates ProQR’s drug candidate QR-313 for dystrophic epidermolysis bullosa (DEB) receives orphan drug designation from the FDA , representing the fifth program in the Company’s pipeline to receive ODD in the U.S. The Company will be presenting pre-clinical data for QR-313 at two European We are ProQR Therapeutics, a clinical stage biotechnology company. We develop RNA therapies for severe genetic rare diseases, with a high need for new medici LEIDEN, Netherlands & CAMBRIDGE, Mass., March 24, 2021 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the "Company"), a company dedicated to changing lives through the creation of transformative RNA therapies for inherited retinal diseases (IRDs), today announced results from a planned analysis of its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non 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 […] PROQR THERAPEUTICS N.V. Zernikedreef 9. 2333 CR Leiden.

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

With March newly here, the company is approaching the timeline that they set out. “We are pleased to see QR-421a advancing to pivotal testing and proud to support the work of ProQR as they advance their pipeline of RNA therapies to potentially help children, adults, and families who are affected by blindness caused by USH2A mutations and other rare inherited retinal diseases.” Phase 1/2 Stellar trial of QR-421a Upcoming Data and Focus. QR-313 is the short term catalyst amongst many that ProQR has in their pipeline. The company is expected to present Phase I/II interim data in the first quarter of 2019. With March newly here, the company is approaching the timeline that they set out. 2021-03-31 ProQR R&D day Highlights Progress on Pipeline and Introduces Axiomer®, a novel proprietary RNA Technology ProQR to host an R&D day in New York today, June … Learn more about ProQR at www.proqr.com.

Learn more about ProQR ProQR expanded its platform and pipeline with QR-110 for the treatment of Leber's Congenital Amaurosis (LCA), the leading genetic cause of blindness in 6 Aug 2020 06, 2020 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: our pipeline,” said Daniel A. de Boer, Chief Executive Officer of ProQR.