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Biotechnologie.

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  5. PlayBall10 16 augustus 2017 13:15

    • PlayBall10

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    ProQR Announces Results for the Second Quarter of 2017
    Key updates

    •Data from two clinical trials of QR-010 presented at the European Cystic Fibrosis Conference. Enrollment completed in the Phase 1b clinical trial in cystic fibrosis (CF) and top-line data expected to be announced in September.
    •The Investigational New Drug Application (IND) and the Clinical Trial Authorization (CTA) cleared and Fast Track designation received from US Food and Drug Administration (FDA) for QR-110 in patients with Leber’s congenital amaurosis 10 (LCA 10).
    •Two key patents granted protecting QR-010 for cystic fibrosis in the US and EU.
    •Pre-clinical data for three programs in the ophthalmology pipeline targeting LCA 10 and Usher syndrome presented at the ARVO annual meeting.
    •Second annual Research & Development Day featured presentations by ProQR senior management and key opinion leaders and introduced next-generation Axiomer® RNA technology platform. www.ir.proqr.com/phoenix.zhtml?c=2537...
  6. Hans Igor 16 augustus 2017 14:21

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    Financial Highlights

    At June 30, 2017, ProQR held cash and cash equivalents of €42.3 million, compared to €59.2 million at December 31, 2016. Net cash used in operating activities during the three month period ended June 30, 2017 was €9.8 million, compared to €8.3 million for the same period last year.

    Research and development costs totaled €7.6 million for the quarter ended June 30, 2017 compared to €8.6 million for the same period last year and comprised of allocated employee costs including share-based payments, the costs of materials and laboratory consumables, outsourced activities, license and intellectual property costs and other allocated costs. The decrease in expenses was primarily due to the completion of the nasal potential difference (NPD) study for QR-010.

    General and administrative costs increased to €2.9 million for the quarter ended June 30, 2017 compared to €2.6 million for the quarter ended June 30, 2016.

    Net result for the three month period ended March 31, 2017 was a €11.3 million loss or €0.47 per share, compared to a €10.0 million loss or €0.43 per share for the same period last year. For further financial information for the period ending June 30, 2017, please refer to the financial statements appearing at the end of this release.

  8. PlayBall10 18 augustus 2017 08:56

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    JMP handhaafd koersdoel op $14.
    Second quarter results in line as CF data of QR-010 are on track for September;
    we reiterate our Market Outperform rating and $14 risk-adjusted, DCF-derived
    price target on ProQR Therapeutics. We anticipate top-line data in September from
    the Phase 1b trial of lead RNA therapy QR-010 in 64 cystic fibrosis (CF) patients
    with F508del homozygous mutation.
    Bijlage:
  10. PlayBall10 28 augustus 2017 13:20

    • PlayBall10

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    ProQR Strengthens its Scientific Advisory Board with the Appointment of Dr. Phil Zamore, Dr. Cy Stein and Dr. Scott Armstrong

    ProQR appoints three new members to the Company’s Scientific Advisory Board: Dr. Phil Zamore, Dr. Cy Stein and Dr. Scott Armstrong, who bring a tremendous amount of experience in the fields of RNA-based therapeutics and genetic pediatric diseases.
    The new members will join Dr. Art Levin and Dr. Annemieke Aartsma-Rus on the Scientific Advisory Board that will play a strategic role in the development of the Company’s pipeline.
    The Scientific Advisory Board will also play a key role in the advancement of the Company’s novel and proprietary next generation RNA editing technology, Axiomer®, that utilizes endogenous RNA editing processes to target a number of genetic disorders.
    www.ir.proqr.com/phoenix.zhtml?c=2537...
  14. [verwijderd] 31 augustus 2017 19:15

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    quote:

    RW1963 schreef op 1 augustus 2017 17:11:

    Wanneer gaat hier eens iets gebeuren? Aandeel is bijna niet vooruit te branden.
    Laatste dagen gaat het als de brandweer. Gisteren 8% vandaag al weer 3%.
  15. RW1963 31 augustus 2017 19:28

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    quote:

    PaxetBonum schreef op 31 augustus 2017 19:15:

    [...]

    Laatste dagen gaat het als de brandweer. Gisteren 8% vandaag al weer 3%.
    Ja, nu zit er eindelijk weer schot in de zaak. En nu die opgaande lijn blijven volhouden. Sommigen zien hierin dezelfde toekomst als bij Galapagos. Dat zou niet verkeerd zijn.
  16. NielsjeB 31 augustus 2017 20:29

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    Lekker druk hier, zal toch niet aan de gestegen koers liggen?
    De volumes gaan ook omhoog, vandaag 47k. Kan me herinneren dat dat soms maar 1000 stukken per dag was.
  17. Cees01 5 september 2017 13:28

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    Printer Friendly Version View printer-friendly version << Back
    ProQR’s Drug Candidate QRX-421 for Usher Syndrome Receives Orphan Drug Designation from FDA and EMA
    Key Updates

    ProQR’s drug candidate QRX-421 for Usher syndrome receives orphan drug designation from the FDA and EMA, representing the third candidate in the company’s ophthalmology pipeline and the fourth in the broader pipeline to receive ODD in the U.S. and EU.
    There are currently no therapies commercially available or in clinical development for the vision loss associated with Usher syndrome type 2.
    QR-421 is part of the company’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, QRX-411 that addresses another genetic mutation resulting in Usher syndrome, QRX-1011 for Stargardt’s disease, and QRX-504 for Fuchs endothelial corneal dystrophy.
    Promising QRX-421 pre-clinical data in both patient fibroblasts and the optic cup model for mRNA restoration were presented at the May 2017 Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) in Baltimore, MD.
    In July 2017, QRX-411, ProQR’s second candidate for Usher syndrome received ODD from the FDA and EMA.
    LEIDEN, the Netherlands, Sept. 05, 2017 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR) today announced that investigational drug QRX-421 for Usher syndrome has received orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA). This marks the third drug candidate in the company’s ophthalmology pipeline and the fourth drug in the broader pipeline to receive ODD from the FDA and EMA. QR-421 is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Usher syndrome due to mutations in exon 13 of the USH2A gene. Usher syndrome is the leading cause of combined deafness and blindness.

    ODD in the U.S. and European Union provides a special status for investigational drugs being developed for rare diseases. The ODD programs offer development program tax benefits and a waiver of the NDA application user fee, as well as market exclusivity for up to seven years in the U.S. and ten years in the European Union following market approval.

    “We are pleased to have ODD designation for both our programs targeting Usher syndrome in the U.S. and EU, representing yet another important milestone for our company and highlighting the unmet need for patients in this disease,” said David M. Rodman, MD, Chief Development Strategy Officer of ProQR. “At ProQR, we are focused on designing accelerated development strategies that capitalize on our oligonucleotide approach to potentially bring our novel medicines to patients quicker and receiving ODD designations for these is an important step towards this goal.”

    ProQR’s ophthalmology pipeline includes the following:

    QR-110 for Leber’s congenital amaurosis 10 (LCA 10) due to the p.Cys998X mutation, which received IND and CTA clearance and is in clinical development (PQ-110-001 Phase 1/2 safety and efficacy study). QR-110 was also granted Fast Track designation by the FDA and ODD designation by the FDA and EMA.
    QRX-421 for Usher syndrome type 2 due to exon 13 mutations in the USH2A gene, for which a clinical candidate has been selected and is ready for IND enabling development studies.
    QRX-411 for Usher syndrome type 2 due to the PE-40 mutation in the USH2A gene, for which a clinical candidate has been selected and is ready for IND enabling development studies. QRX-411 also received ODD designation by the FDA and EMA.
    QRX-1011 for Stargardt’s disease due to c.5461-10T>C mutations in the ABCA4 gene, which is in optimization phase.
    QRX-504 for Fuchs endothelial corneal dystrophy (FECD), for which a clinical candidate has been selected and is ready for IND enabling development studies.
    About QRX-421

    QRX-421 is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Usher syndrome due to mutations in exon 13 of the USH2A gene. Mutations in this exon can cause loss of functional USH2A protein that causes the disease. QRX-421 is designed to exclude exon 13 from the mRNA (exon skipping) and produce truncated but functional USH2A protein, thereby modifying the underlying disease.

    About Usher Syndrome

    Usher syndrome is the leading cause of combined deafness and blindness. Patients with this syndrome generally progress to a stage in which they have very limited central vision and moderate to severe deafness. To date, there are no treatments approved or products in clinical development that treat the vision loss associated with Usher syndrome type 2. Usher syndrome type 2 is one of the most common forms of Usher syndrome and is caused by mutations in the USH2A gene.

    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*
  18. [verwijderd] 10 september 2017 20:13

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    Uit NRC 09-09-2017
    Hoop voor zeldzame ziektes
    Daniël de Boer is CEO van ProQR. Dit biotechbedrijf
    ontwikkelt onder andere een medicijn tegen taaislijmziekte.

    Bijlage:
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