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Aandeel ProQR Therapeutics NV OTC:PRQR.Q, NL0010872495

  • 1,880 18 apr 2024 22:00
  • -0,020 (-1,05%) Dagrange 1,810 - 2,050
  • 182.726 Gem. (3M) 226,1K

Nieuws media financieel ProQR Therapeutics NV

31 Posts
Pagina: 1 2 »» | Laatste | Omlaag ↓
  2. forum rang 4 harvester 12 oktober 2017 11:09
    quote:

    ivet schreef op 12 oktober 2017 00:21:

    zie: ir.proqr.com/phoenix.zhtml?c=253704&a...

    Filing Date Form Description Filing Group Downloads
    10/11/17 SC 13G A statement of beneficial ownership of common stock by certain persons
    Opmerkelijk zo een 10,5% belang door een Belgisch vrachtwagen bredrijf.
    Zeker door een grens gegaan, want er was geen emissie.
  4. Dar-win 12 oktober 2017 14:14
    quote:

    harvester schreef op 12 oktober 2017 11:09:

    [...]

    Opmerkelijk zo een 10,5% belang door een Belgisch vrachtwagen bredrijf.
    Zeker door een grens gegaan, want er was geen emissie.
    Dit betreft JDG BV een vehikel van:
    Jeroen Voskamp 700.000
    Daniël de Boer 1.012.028
    Gerard Platenburg 934.257
    JDG BV 2.646.285
    Betreft 10,46 % van 25.305.249 shares

    JDG zijn de Voornaam-letters van de heren.
  5. [verwijderd] 26 oktober 2017 13:11
    ProQR Announces Management Change and Key Promotions
    Key Updates

    Company announces departure of Noreen R. Henig, Chief Medical Officer, promotion of Peter Adamson to Senior Vice President Ophthalmology Franchise and Robert Friesen to Senior Vice President Science and Early Development.
    David M. Rodman, M.D., Chief Development Strategy Officer will assume leadership of clinical development at the Company.
    LEIDEN, the Netherlands, Oct. 26, 2017 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), (the “Company”), today announced that Noreen R. Henig, M.D., Chief Medical Officer, will leave ProQR effective November 4, 2017 to pursue a new opportunity, and will serve as a special advisor to the Company going forward. Chief Development Strategy Officer, David M. Rodman M.D, will assume leadership over clinical development. Additionally, the Company announced the promotion of Peter Adamson to Senior Vice President Ophthalmology Franchise and the promotion of Robert Friesen to Senior Vice President Science and Early Development.

    “In four years, ProQR has grown from a small company with big ideas to a RNA therapeutics company with two completed and positive clinical trials, a robust pipeline, and an incredibly talented team. I am truly proud of our achievements to date and believe the Company is positioned for continued growth,” said Noreen R. Henig M.D. “It’s been a privilege to work with the talent inside ProQR, as well as the investigators and scientific collaborators who helped us achieve our goals.”

    “During her four-year tenure, Noreen was instrumental in the successful completion of two global clinical trials for QR-010 for CF patients, bringing QR-110 for LCA10 patients into the clinic and advancing QR-313 towards clinical development. On behalf of all ProQRians, I thank Noreen for her dedication and leadership and wish her the very best in her future endeavors,” said Daniel A. de Boer, CEO of ProQR. “With several of our development programs having clinical data readouts next year, I feel confident that under Dave’s leadership we will meet our clinical milestones, continue to strengthen our portfolio and expand our innovative RNA therapeutic approach to treat patients that suffer from severe rare diseases.”

    David M. Rodman, M.D., Chief Development Strategy Officer at ProQR, will lead clinical development at the Company. Dr. Rodman joined ProQR in March 2017 with extensive experience in rare disease drug development, translational medicine and RNA therapeutics, having previously served in leadership roles with Novartis Institute for Biomedical Research (NIBR), Vertex Pharmaceuticals, miRagen Therapeutics and Nivalis Therapeutics.

    In his promotion to SVP Ophthalmology Franchise, Peter Adamson, Ph.D., will be responsible for development of drugs to treat severe inherited ophthalmic diseases. Dr. Adamson joined ProQR in April 2015 as the Head of Ophthalmology Research. Formerly, Dr. Adamson was Vice President and Head of Ophthalmology Research at GlaxoSmithKline. He obtained his Ph.D. in Biochemistry from the University of London (Institute of Psychiatry) and subsequently worked as a postdoctoral researcher at the Institute of Cancer Research and Vascular Biology Research Centre before taking up an academic position at the UCL Institute of Ophthalmology from 1994-2004 where he was a group leader and Reader in Molecular Pathology. Dr. Adamson has authored over 100 peer-reviewed scientific publications in the domains of inflammation, ophthalmology and neurology and retains an honorary appointment at UCL, Institute of Ophthalmology where he is Professor of Molecular Pathology (Ophthalmology).

    In Robert Friesen, Ph.D.’s new role as SVP Science and Early Development, he will be overseeing the scientific advancement of the Company and will be responsible for non-clinical development. Dr. Friesen has served as Head of Innovation at the Company since June 2016. He obtained a M.S. in Biology from the University of Utrecht, and a Ph.D. in Biochemistry, at the University of Texas Medical Branch. Previously, Dr. Friesen was Vice President and Head of Biologics Research within Janssen R&D, a Johnson & Johnson company, where he led a global team of over 200 scientists responsible for the discovery and early development of biotherapeutics for all therapeutic areas. Before joining Janssen R&D, he held senior R&D positions at AM-Pharma, MorphoSys and Crucell. Dr. Friesen has authored a number of publications in high impact scientific journals, and participated in numerous invited lectures. He has also been awarded multiple patents in the field of biotechnology.

    “As we continue to build our pipeline, the promotion of Pete and Robert further enforce our senior leadership team,” said Daniel A. de Boer, CEO of ProQR. “With their tremendous experience in science, early development and ophthalmology we are positioned for success.”

    ir.proqr-tx.com/phoenix.zhtml?c=25370...
  6. [verwijderd] 31 oktober 2017 12:13
    ir.proqr-tx.com/phoenix.zhtml?c=25370...

    ProQR to Present at EuroTIDES Conference
    LEIDEN, The Netherlands, Oct. 31, 2017 (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 genetic rare diseases including cystic fibrosis, Leber's congenital amaurosis 10 and dystrophic epidermolysis bullosa, today announced a presentation at the EuroTIDES conference taking place November 7 – 10, 2017 in Vienna, Austria.

    The presentation titled “IPRP UPLC Method Optimization for the Separation of Short- and Longmer Impurities in Oligonucleotide Therapeutics” by Hilde van Hattum, Ph.D., Sr. Scientist Characterisation & BioAnalytics at ProQR will take place on November 9, 2017 at 2:30pm CET. Additional information about the presentation and the EuroTIDES conference is available online.
  7. [verwijderd] 7 februari 2018 08:27
    ProQR to Present at Upcoming Scientific Meeting and Investor Conference

    LEIDEN, the Netherlands, Feb. 06, 2018 (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 genetic rare diseases including cystic fibrosis, Leber's congenital amaurosis 10 and dystrophic epidermolysis bullosa, today announced that the Company will present at an investor conference and scientific meeting during the month of February.
    LEERINK Partners 7th Annual Global Healthcare Conference
    On Wednesday, February 14, 20178 at 9:30am EST, Daniel de Boer, Chief Executive Officer of ProQR, will present at the LEERINK 7th Annual Global Healthcare Conference. The conference is being held at the Lotte New York Palace, New York, NY.
    The live and archived webcast of the presentation will be accessible from the ‘Investor Relations’ section of ProQR’s website (www.proqr.com) under ‘Events and Presentations’. The archived webcast will be available for 30 days following the presentation date.
    SMi RNA Therapeutics Annual Conference
    A presentation titled “Therapeutic RNA editing by Axiomer® editing oligonucleotides” by Janne Turunen, PhD, Director of RNA Editing Technologies at ProQR, will take place on February 21, 2018 at 11:00am GMT at the SMi RNA Therapeutics Annual Conference in London, UK. Additional information about the conference is available online.
    About ProQR
    ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such as cystic fibrosis, Leber’s congenital amaurosis 10 and dystrophic epidermolysis bullosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
    *Since 2012*
    FORWARD-LOOKING STATEMENTS
    This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.
  8. [verwijderd] 12 februari 2018 14:24
    Foundation Fighting Blindness will provide up to $7.5 million in funding to develop ProQR’s candidate QR-421a for Usher syndrome type 2A

    Foundation Fighting Blindness and ProQR enter into a partnership to develop QR-421a for Usher syndrome type 2A, targeting mutations in exon 13 of the causative USH2A gene.
    Foundation Fighting Blindness will provide milestone-based co-funding of up to $7.5 million to ProQR to advance the program into the clinic.
    QR-421a received orphan drug designation from the FDA.
    There are currently no therapies commercially available or in clinical development for the vision loss associated with Usher syndrome type 2A.
    QR-421a is part of ProQR’s growing ophthalmology pipeline which also includes lead candidate, QR-110 for Leber’s congenital amaurosis 10 currently in clinical trials, and three additional pipeline programs, QR-411 that addresses another genetic mutation resulting in Usher syndrome type 2A, QRX-1011 for Stargardt’s disease and QRX-504 for Fuchs endothelial corneal dystrophy.
    QR-421a is expected to advance towards the clinic in 2018, with clinical data expected in 2019.
    COLUMBIA, Md. and LEIDEN, the Netherlands, Feb. 12, 2018 (GLOBE NEWSWIRE) -- Foundation Fighting Blindness and ProQR Therapeutics N.V. (NASDAQ:PRQR), today announced that they have entered into a partnership to develop QR-421a for Usher syndrome 2A caused by an exon 13 mutation of the causative USH2A gene. Under the agreement, Foundation Fighting Blindness will provide up to $7.5 million in funding to ProQR for the preclinical and clinical development of QR-421a, which is expected to advance towards the clinic in 2018, and safety and efficacy results from the Phase 1/2 trial in Usher syndrome patients are expected in 2019.
    Usher syndrome is a devastating genetic disease in which patients first develop hearing loss and then progressive vision loss, thereby threatening their independence and quality of life. Currently there is no treatment for the ophthalmic manifestation of Usher syndrome type 2A. QR-421a is a first-in-class RNA oligonucleotide that is being developed for the treatment of vision loss associated with the disease. QR-421a is designed to modify the RNA such that functional usherin protein is produced in the retina with the goal of stopping the progression of the disease and potentially gaining peripheral vision. ProQR in-licensed the technology underlying QR-421a from Radboud University Medical Center in the Netherlands, where it was invented by lead investigator Dr. Erwin van Wyck.
    Foundation Fighting Blindness’ Clinical Research Institute (FFB-CRI) has also launched a natural history study in 120 people with USH2A mutations. The study — known as RUSH2A (“R” stands for “rate of progression”) — was launched in 2017 and is being conducted at about 20 clinical sites around the world. RUSH2A investigators will use a variety of technologies to monitor changes in vision and retinal structure to document and analyze disease progression. Knowledge and data obtained from this trial are intended to provide a better understanding of how USH2A mutations affect the severity and progression of vision loss and help to inform the development of QR-421a.
    “Teaming with corporate partners to help promising therapies move through preclinical and clinical development is central to FFB’s strategy so we are very pleased to enter into this partnership with ProQR,” said Benjamin R. Yerxa, PhD, CEO at Foundation Fighting Blindness. “The fact that there are currently no available treatments for Usher syndrome type 2A makes this work that much more exciting and critical.”
    QR-421a for Usher syndrome is the second program in ProQR’s growing ophthalmology pipeline scheduled to enter clinical trials. The lead program in the ophthalmology pipeline, QR-110, is currently in a Phase 1/2 safety and efficacy trial in adult and pediatric patients with Leber’s congenital amaurosis 10, due to the p.Cys998X mutation in the CEP290 gene. This pipeline also contains several other molecules for genetic eye diseases, including QR-411 for Usher syndrome type 2A due to the PE-40 mutation, QRX-1011 for Stargardt’s disease and QRX-504 for Fuchs endothelial corneal dystrophy.
    “We are excited to team up with the Foundation Fighting Blindness to develop QR-421a for patients that suffer from Usher syndrome due to exon 13 mutations," said Daniel A. de Boer, CEO of ProQR. “They are the leading private funder of retinal disease research with a very patient centric approach which is a core pillar of our strategy. Through this partnership with the Foundation we plan to gain access to important know-how to develop programs in retinal diseases. We expect that the additional funding will allow us to rapidly advance this novel therapy for this orphan disease with a severe unmet need.”
    About Foundation Fighting Blindness
    The Foundation Fighting Blindness was established in 1971 and has raised more than $725 million for research on preventing, treating and curing blindness caused by inherited retinal diseases. In excess of 10 million Americans, and millions more worldwide, experience or are at risk for vision loss due to retinal degenerations. Through its support of focused and innovative science, and by teaming with industry, the Foundation drives the research that has and will continue to provide treatments and cures for people affected by retinitis pigmentosa, macular degeneration, Usher syndrome and other inherited retinal diseases.
  10. [verwijderd] 3 april 2018 14:16
    ProQR to Present at Two Upcoming Conferences


    LEIDEN, the Netherlands, April 03, 2018 (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 genetic rare diseases including Leber's congenital amaurosis 10, dystrophic epidermolysis bullosa and cystic fibrosis, today announced that the Company will present at two upcoming conferences in April.
    H.C. Wainwright Global Life Sciences Conference
    On Tuesday, April 10, 2018 at 9:25 am CET, Daniel de Boer, Chief Executive Officer of ProQR, will present at the H.C. Wainwright Global Life Science Conference. The conference is being held on April 8 - 10 in Monte Carlo, Monaco.
    World Orphan Drug Congress USA
    On Thursday, April 26, 2018 at 5:20 pm ET, Daniel de Boer, Chief Executive Officer of ProQR, will participate in a panel discussion titled ‘How rare disease companies are ramping up innovation to boost orphan drug development and patient centricity’ during the World Orphan Drug Congress. The congress is being held on April 25 - 27 in Oxon Hill, MD, USA.
  11. [verwijderd] 23 april 2018 13:36

    About the PQ-110-001 trial
    PQ-110-001 is an open-label trial that has been designed to enroll approximately six children (age 6 - 17 years) and six adults (= 18 years) who have LCA 10 due to one or two copies of the p.Cys998X mutation in the CEP290 gene. Patients are receiving four intravitreal injections of QR-110 into one eye; one every three months. The trial is being conducted at three specialized centers with significant expertise in genetic retinal disease: the University of Iowa, Iowa City, IA, US, the Scheie Eye Institute at the University of Pennsylvania, Philadelphia, PA, US and the Ghent University Hospital, Ghent, Belgium.
    The primary objectives of the trial are safety and tolerability. Secondary objectives include pharmacokinetics as well as restoration/improvement of visual function and retinal structure through ophthalmic endpoints such as visual acuity, full field stimulus testing (FST), optical coherence tomography (OCT), pupillary light reflex (PLR), mobility course and fixation stability. Changes in quality of life in the trial subjects are also being evaluated.

    About Leber’s Congenital Amaurosis 10
    Leber’s congenital amaurosis (LCA) is the most common cause of blindness due to genetic disease in children and consists of a group of diseases of which LCA 10 is one of the more severe forms. LCA 10 is caused by mutations in the CEP290 gene, of which the p.Cys998X mutation is the most common. LCA 10 leads to early loss of vision causing most people to lose their sight in the first few years of life. To date, there are no treatments approved or other products in clinical development that treat the underlying cause of the disease. Approximately 2,000 people in the Western world have LCA 10 because of this mutation.

    About QR-110
    QR-110 is a potential first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Leber’s congenital amaurosis 10 due to the p.Cys998X mutation in the CEP290 gene. The p.Cys998X mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional CEP290 mRNA. QR-110 is designed to restore normal (wild-type) CEP290 mRNA leading to the production of normal CEP290 protein by binding to the mutated location in the pre-mRNA causing normal splicing of the pre-mRNA. QR-110 is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the United States and the European Union and received fast-track designation by the FDA.

    About Fuchs Endothelial Corneal Dystrophy
    Fuchs endothelial corneal dystrophy (FECD) is a common inherited condition characterized by the dysfunction and degeneration of the corneal endothelium, a single cell layer of cells on the inside of the cornea. There are different types of this disease and we focus on age-related FECD (FECD3). Some patients with age-related FECD develop advanced disease with corneal edema and corneal clouding. These symptoms can lead to complete vision loss and the need for surgery and a corneal transplant.

    About QRX-504
    QRX-504 is a potential first-in-class investigational RNA-based oligonucleotide designed to prevent corneal dystrophy in patients that have FECD3 due to trinucleotide repeat expansions in the TCF4 gene. QRX-504 binds to the mutated TCF4 mRNA and thereby stops the formation of toxic aggregates that cause the disease. QRX-504 is intended to be administered through intraocular injection into the eye.

    About Stargardt’s Disease
    Stargardt’s disease (fundus flavimaculatus) is a form of juvenile macular degeneration which causes progressive central vision loss, reduced visual acuity, impaired color vision and problems in night vision. The signs and symptoms typically appear in late childhood to early adulthood and worsen over time with loss of peripheral vision.

    About QRX-1011
    QRX-1011 is a potential first-in-class investigational RNA-based oligonucleotide that aims to treat vision loss caused by the specific c.5461-10T>C mutation in ABCA4 gene which leads to an important part of the protein being deleted. QRX-1011 modulates splicing of the mRNA and leads to inclusion of the deleted sequence and formation of functional, wild-type ABCA4 protein which will potentially stop and perhaps reverse the progression of the disease.
  12. [verwijderd] 23 april 2018 13:36
    ProQR Provides Enrollment Update on QR-110 Clinical Trial and Highlights Ophthalmology Presentations at ARVO

    Key Updates
    • Enrollment is on track in the ongoing Phase 1/2 clinical trial of QR-110 with eight out of twelve patients with LCA 10 enrolled.
    • The trial is on track to announce interim six-month data on safety, effects on vision and retinal structure in the second half of the year.
    • ProQR to present two abstracts on programs for Fuchs endothelial corneal dystrophy and Stargardt’s disease at the ARVO annual meeting. The company will also deliver a presentation during the Foundation Fighting Blindness/Casey Innovation summit prior to ARVO.
    LEIDEN, the Netherlands, April 23, 2018 (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 genetic rare diseases, today announced that eight out of twelve patients have been enrolled in PQ-110-001, a Phase 1/2 open-label trial assessing the safety, tolerability, pharmacokinetics and efficacy of QR-110 for the treatment of Leber’s congenital amaurosis 10 (LCA 10). The trial has been designed to enroll approximately six adults and six children who have LCA 10 due to the p.Cys998X mutation in the CEP290 gene. Interim safety and efficacy trial results are expected to be announced in the second half of 2018 when six or more patients have received at least six months of treatment, with full twelve-month treatment data from all patients expected in 2019.
    “We are very pleased with the enrollment in our trial and the continued enthusiasm from our investigators in helping bring a novel and first-in-class treatment to LCA 10 patients that have no other treatment options,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “We look forward to the clinical data for the QR-110 trial later this year as it will to help guide and accelerate the development of other ophthalmology candidates in our pipeline, as we believe that RNA oligonucleotides hold great promise for the treatment of a variety of genetic eye diseases.”
    Presentations at ARVO conference
    ProQR will present data from two ophthalmology programs at the 2018 annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) to be held April 29 – May 3, 2018 in Honolulu, HI, USA. Abstracts for the presentations are or will be made available on the ARVO 2018 annual meeting website.

    Oral Presentation: RNA toxicity induced by TCF4 CTG expansions is ameliorated by antisense therapeutics in a patient-derived cell model of Fuchs corneal endothelial dystrophy (FECD)
    Presenter: Dr. Alice Davidson of UCL Institute of Ophthalmology, London
    Date: Tuesday, May 1
    Time: 12:45 - 1:00pm HAST

    Poster Presentation: Oligonucleotide-based splice correction of the ABCA4 c.5461-10T>C mutation in Stargardt’s disease type 1
    Presenter: Kalyan Dulla, Ph.D., director of non-clinical research of ProQR
    Poster #: 5315 - C0248
    Date: Wednesday, May 2
    Time: 3:30 – 5:15pm HAST

    Oral Presentation: Retinal organoids from IRDs resulting from intronic mutations provide powerful models to both validate pharmacological treatment approaches and determine initial dose-ranging in clinical studies
    Presenter: Dr. Peter Adamson, Senior Vice President, Ophthalmology Franchise of ProQR
    Session: FFB/Casey Innovation summit prior to ARVO: Retinal Cell and Gene Therapy
    Date: Friday, April 27
    Time: 10:45 – 11:00am HAST
  13. [verwijderd] 30 april 2018 13:05
    ProQR Announces Conference Presentations for Axiomer® RNA Editing Technology and QR-313 for DEB

    LEIDEN, the Netherlands, April 30, 2018 (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 genetic rare diseases, today announced that the Company will present at two upcoming conferences in May.
    Presentation at the TIDES 2018 Conference
    ProQR will deliver a presentation on the Axiomer® RNA editing technology during the TIDES: Oligonucleotide and Peptide Therapeutics Conference to be held on May 7 – 10, 2018 in Boston, MA, USA.
    Abstract title: Axiomer® technology: Therapeutic oligonucleotides for directing site-specific A-to-I editing by endogenous ADAR enzymes
    Presenter: Antti Aalto, Ph.D., senior scientist protein interactions of ProQR
    Oral Presentation: Thursday, May 10, 11:45am – 12:15pm ET
    Session: Track 5: mRNA, CRISPR and Hot Topics in Oligonucleotides
    Presentation at the IID 2018 Meeting
    ProQR will present data from the QR-313 program for dystrophic epidermolysis bullosa at the International Investigative Dermatology (IID) 2018 meeting to be held on May 16 – 19, 2018 in Orlando, FL, USA.
    Abstract title: QR-313, an antisense oligonucleotide, restores expression of functional type VII collagen in DEB patient cells
    Presenter: Ilse Haisma, PH.D., senior scientist dermatology of ProQR
    Poster Presentation: Saturday, May 19, 11:45 am – 1:45 pm ET
    Poster #: 984 – 1474 EVEN/ 493 – 981 ODD
    Oral Presentation: Saturday, May 19, 3:15 - 5:45pm ET
    About Axiomer® platform technology
    ProQR is pioneering a next-generation RNA technology called Axiomer®, which could potentially yield a new class of medicines for genetic diseases. Axiomer® “Editing Oligonucleotides”, or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells. The Axiomer® EONs are designed to recruit an endogenously expressed RNA editing system called ADAR, which can direct the change of an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G).
    About QR-313
    QR-313 is a potential first-in-class RNA-based oligonucleotide designed to address the underlying cause of dystrophic epidermolysis bullosa (DEB) due to mutations in exon 73 of the COL7A1 gene. Mutations in this exon can cause loss of functional collagen type VII (C7) protein. Absence of C7 results in the loss of anchoring fibrils that normally link the dermal and epidermal layers of the skin together. QR-313 is designed to exclude exon 73 from the mRNA (exon skipping) and produce a functional C7 protein, thereby restoring functionality of the anchoring fibrils. QR-313 has been granted Orphan Drug Designation in the United States and the European Union.
    About ProQR
    ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such as Leber’s congenital amaurosis 10, dystrophic epidermolysis bullosa and cystic fibrosis. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
    *Since 2012*
    FORWARD-LOOKING STATEMENTS
    This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such statements include those relating to our participation at the TIDES: Oligonucleotide and Peptide Therapeutics Conference and the International Investigative Dermatology meeting. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.
    ProQR Therapeutics N.V.:
    Smital Shah
    Chief Financial Officer
    T: +1 415 231 6431
    ir@proqr.com
  15. PlayBall10 4 juni 2018 13:28

    ProQR Announces Presentations at ECFS on Eluforsen for F508del Cystic Fibrosis and at CFF Research Conference

    4 June 2018 at 7:00 AM EDT

    LEIDEN, The Netherlands, June 04, 2018 (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 genetic rare diseases, today announced upcoming presentations at the European Cystic Fibrosis Society (ECFS) conference and the CFF Research Conference.

    Presentation at ECFS Conference

    ProQR will deliver a short talk (ePoster) and a poster presentation on eluforsen during the ECFS: European Cystic Fibrosis Society Conference to be held on June 6 – 9, 2018 in Belgrade, Serbia.

    Abstract title: Exploratory immune assays distinguish healthy volunteer from CF patient cohorts and were validated in a dose escalation study of QR-010 in subjects with cystic fibrosis homozygous for the F508del CFTR mutation
    Presenter: Miriam Bujny, Ph.D., director biomarkers of ProQR
    Poster Presentation: Friday, June 9 14:00 – 15:00 CET
    Poster #: EPS3.02
    ePoster Presentation: Session: New treatments: from genes to protein. Thursday, June 7, 14:00-15:00 CET

    Presentation at 2018 CFF Research Conference – New Technologies Advancing Toward a One-Time Cure

    ProQR’s scientific advisory board (SAB) member, Prof. Yi-Tao Yu from the University of Rochester Medical Center (URMC), will give a presentation at the upcoming CFF Research Conference – New technologies Advancing Towards a One-Time Cure. The conference will be held on June 18 -21, 2018 at the Snow King Resort in Jackson, WY USA.

    Presentation title: Suppression of nonsense mutations in the CFTR gene by targeted RNA pseudouridylation
    Presenter: Yi-Tao Yu, Prof. University of Rochester Medical Center
    Session: Session 2: Approaches for Targeting Nonsense Mutations
    Schedule: Tuesday, June 19 10:55 – 11:20 MT

    About eluforsen

    Eluforsen, formerly known as QR-010, is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of the disease by targeting the mRNA in CF patients that have the F508del mutation. The technology was exclusively licensed from Massachusetts General Hospital. The F508del mutation results in the production of a misfolded CFTR protein that does not function normally. Eluforsen is a single agent designed to bind to the defective CFTR mRNA and to restore CFTR function. Eluforsen is designed to be self-administered via an optimized eFlow® Nebulizer (PARI Pharma GmbH). eFlow® is a small, handheld aerosol delivery device which nebulizes eluforsen into a mist inhaled directly into the lungs. Eluforsen has been granted orphan drug designation in the United States and the European Union and fast-track status by the FDA. The eluforsen project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 633545.
    ir.proqr.com/news-releases/news-relea...
  17. [verwijderd] 28 juni 2018 13:41

    ProQR Initiates Phase 1/2 Clinical Trial of QR-313 for Dystrophic Epidermolysis Bullosa

    The trial, called WINGS, will evaluate the safety and efficacy of QR-313 in subjects with recessive dystrophic epidermolysis bullosa due to mutations in exon 73 of the COL7A1 gene.
    QR-313 is a first-in-class potential therapeutic RNA molecule designed to improve the healing and integrity of skin in DEB patients

    Interim results are expected in late 2018; full data are expected in 2019

    LEIDEN, the Netherlands, June 28, 2018 (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 genetic rare diseases, today announced that a Phase 1/2 clinical trial called “WINGS”, to evaluate the safety and efficacy of QR-313 in patients with recessive dystrophic epidermolysis bullosa (RDEB) is open for enrollment.

    “The initiation of our first human clinical trial for QR-313 is an exciting next step in the development of this novel investigational therapy for DEB. WINGS is designed to initially provide molecular proof of mechanism and subsequently clinical proof of concept for QR-313,” said David M. Rodman, MD, Executive Vice President of Research and Development at ProQR. “Now that the study is initiated we expect to dose the first set of adult and pediatric patients over the next few months and provide interim proof of mechanism results late this year.”

    Dystrophic epidermolysis bullosa is a severe blistering disease that causes fragile skin. People with DEB live with constant pain and have a high risk of malnutrition and infections. Symptoms of the disease include poorly healing wounds, skin infections, fusion of fingers and toes, anemia and gastrointestinal tract problems. Some patients develop very aggressive forms of skin cancer in adulthood. There are currently no approved treatment options available that target the underlying cause of DEB.

    David M. Rodman, MD continued: “The WINGS study marks the second of three clinical trials that we are conducting at ProQR this year. Beyond WINGS for DEB we have an ongoing QR-110 study in LCA 10 patients and plan to start a clinical trial of QR-421a in patients that suffer from Usher syndrome type 2.”

    About the WINGS Trial

    WINGS is a first-in-human Phase 1/2 double-blind, randomized, intra-subject placebo controlled clinical trial of QR-313 in approximately eight subjects (at least six years of age) that have RDEB due to mutation(s) in exon 73 of the COL7A1 gene.

    The primary objectives for the study are to evaluate the safety and tolerability of topically applied QR-313 and assess proof of mechanism (exclusion, or skipping, of exon 73 from COL7A1 mRNA assessed by polymerase chain reaction). The secondary objectives are to quantify blood levels of QR-313, assess effects on wound healing, skin strength, the presence of collagen type 7 protein and anchoring fibrils in the skin.

    During the study QR-313 or placebo formulated in a gel will be topically applied to a patient’s wounds approximately every-other-day for up to four weeks with a subsequent eight week observation period. Up to four small skin biopsies will be performed and tissue analyzed for molecular endpoints. The trial will be conducted at specialized centers in the U.S. and selected European countries.

    Interim proof of mechanism results from the trial are expected in 2018. Full clinical proof of concept results from the study are expected in 2019. Depending on results of the interim analysis, the trial may be adapted to adjust the frequency and/or method of topical delivery to further enhance uptake and activity. It is planned that eight subjects will receive either active gel or placebo on two separate wounds. Clinical proof of concept will compare the rate, strength and stability of wounds treated with active gel to those treated with placebo.

    About QR-313

    QR-313 is a potential first-in-class RNA-based oligonucleotide designed to address the underlying cause of dystrophic epidermolysis bullosa (DEB) due to mutations in exon 73 of the COL7A1 gene. Mutations in this exon can cause loss of functional collagen type VII (C7) protein. Absence of C7 results in the loss of anchoring fibrils that normally link the dermal and epidermal layers of the skin together. QR-313 is designed to exclude exon 73 from the mRNA (exon skipping) and produce a functional C7 protein, thereby restoring functionality of the anchoring fibrils. The clinical development of QR-313 is supported with funding from EB Research Partnership and EB medical Research Foundation. QR-313 has been granted orphan drug designation in the United States and the European Union.

    About Dystrophic Epidermolysis Bullosa

    Epidermolysis bullosa (EB) is a group of rare genetic skin diseases of which dystrophic EB (DEB) is one of the most severe forms. DEB is caused by a mutation in the COL7A1 gene which is responsible for the formation of the collagen type C7 protein that anchors fibrils that bind the inner and outer skin layers together. This mutation causes a loss of the anchoring fibrils resulting in fragile skin. People with DEB live with constant pain and have a high risk of malnutrition and infections. Symptoms include poorly healing wounds, skin infections, fusion of fingers and toes, anemia, gastrointestinal tract problems and with adulthood some develop very aggressive forms of skin cancer. There are no approved treatment options available that target the underlying cause of DEB.
  20. [verwijderd] 10 december 2018 13:19
    ProQR Receives € 4.7 million in Innovation Credit from Dutch Government for QR-110 for LCA10

    LEIDEN, Netherlands and CAMBRIDGE, Mass., Dec. 10, 2018 (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 genetic rare diseases, today announced that it was awarded an innovation credit by the Dutch government for the clinical development and efforts to obtain marketing approval (NDA/MAA) for the QR-110 program that is being developed for patients with Leber’s congenital amaurosis 10 (LCA10), a genetic eye disease that is the leading cause of childhood blindness. Repayment of the credit is triggered only by market approval of QR-110.

    Innovation credit (“Innovatiekrediet”)

    The Innovation Credit is awarded by the Dutch government through its agency RVO of the Ministry of Economic Affairs, and is aimed at the development of promising innovations. This can include the technical development of a new product or process or the clinical development of a medicine or device.

    ProQR was awarded an Innovation credit for the QR-110 program. Amounts will be drawn under this facility from 2018 thru 2021. The credit of € 4.7 million through December 31, 2021 will be used to conduct the Phase 2/3 clinical study and efforts to obtain regulatory and ethical market approval (NDA/MAA) of QR-110 for LCA10.

    The credit, including accrued interest, is repayable depending on obtaining market approval.

    About QR-110

    QR-110 is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Leber’s congenital amaurosis 10 due to the p.Cys998X mutation (also known as the c.2991+1655A>G mutation) in the CEP290 gene. The p.Cys998X mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional CEP290 protein. QR-110 is designed to restore normal (wild-type) CEP290 mRNA leading to the production of normal CEP290 protein by binding to the mutated location in the pre-mRNA causing normal splicing of the pre-mRNA. QR-110 is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the United States and the European Union and received fast-track designation by the FDA.

    About Leber’s Congenital Amaurosis 10

    Leber’s congenital amaurosis (LCA) is the most common cause of blindness due to genetic disease in children and consists of a group of diseases of which LCA10 is the most frequent and one of the more severe forms. LCA10 is caused by mutations in the CEP290 gene, of which the p.Cys998X mutation is the most common. LCA10 leads to early loss of vision causing most people to lose their sight in the first few years of life. To date, there are no treatments approved or other products in clinical development that treat the underlying cause of the disease. Approximately 2,000 people in the Western world have LCA10 because of this mutation.

    About ProQR

    ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such as Leber’s congenital amaurosis 10, dystrophic epidermolysis bullosa and cystic fibrosis. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
    *Since 2012*
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