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  1. forum rang 4 ch@rter 7 juli 2017 18:03
    Het vertrouwen komt terug (of goklust,)

    Perceptive Advisors LLC Has $7,317,000 Position in uniQure N.V. (NASDAQ:QURE)
    Posted by Renata Jones on Jul 6th, 2017 // No Comments
    uniQure N.V. logoPerceptive Advisors LLC maintained its position in shares of uniQure N.V. (NASDAQ:QURE) during the first quarter, according to its most recent disclosure with the SEC. The institutional investor owned 1,265,963 shares of the biotechnology company’s stock at the end of the first quarter. Perceptive Advisors LLC owned approximately 5.02% of uniQure N.V. worth $7,317,000 as of its most recent SEC filing.
    Several other large investors have also made changes to their positions in the stock. JPMorgan Chase & Co. boosted its stake in shares of uniQure N.V. by 50.5% in the first quarter. JPMorgan Chase & Co. now owns 43,822 shares of the biotechnology company’s stock valued at $253,000 after buying an additional 14,702 shares during the last quarter. Renaissance Technologies LLC boosted its stake in shares of uniQure N.V. by 6.8% in the fourth quarter. Renaissance Technologies LLC now owns 251,300 shares of the biotechnology company’s stock valued at $1,407,000 after buying an additional 15,950 shares during the last quarter. Oxford Asset Management purchased a new stake in shares of uniQure N.V. during the first quarter valued at $100,000. Commonwealth of Pennsylvania Public School Empls Retrmt SYS purchased a new stake in shares of uniQure N.V. during the fourth quarter valued at $182,000. Finally, Weiss Multi Strategy Advisers LLC boosted its stake in shares of uniQure N.V. by 266.7% in the first quarter. Weiss Multi Strategy Advisers LLC now owns 55,000 shares of the biotechnology company’s stock valued at $318,000 after buying an additional 40,000 shares during the last quarter. 30.50% of the stock is owned by institutional investors
  2. Fartknock 10 juli 2017 18:54
    finance.yahoo.com/news/uniqure-announ...

    uniQure Announces Updated, Long-Term Clinical Data from Ongoing Phase I/II Trial of AMT-060 In Patients with Severe Hemophilia B
    GlobeNewswire•July 10, 2017

    Clinical Benefit Maintained in All Patients, with FIX Activity Persisting At Up To 18 Months of Follow-Up

    Second-dose Cohort Demonstrates Dose Response Up to One Year, with 84% Reduction in
    Spontaneous Bleeds and All Patients Free of Prophylactic FIX Replacement Therapy

    No Activation of T-Cell Responses or Loss of FIX Activity in Any Patient Up To 18 Months

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, July 10, 2017 (GLOBE NEWSWIRE) -- uniQure N.V. (QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, will today announce updated results from its ongoing, dose-ranging Phase I/II trial of AMT-060, its proprietary, investigational gene therapy in patients with severe hemophilia B. The data includes up to 18 months of follow-up from the low-dose cohort and up to one year of follow-up from the second dose cohort.

    The AAV5-based AMT-060 remains safe and well-tolerated with up to a year and a half of follow-up, with no serious adverse events and no development of inhibitors. All patients are now past one year of follow up with no loss of Factor IX (FIX) activity and no capsid-specific T-cell activation.

    One-year follow-up data from the second-dose cohort continue to show a dose response with substantial improvement in disease state in all five patients, including the discontinuation of routine prophylactic FIX infusions in all patients that previously required chronic replacement therapy. The annualized spontaneous bleeding rate for the second dose cohort declined 84% to a mean of 0.5 annual bleeds after gene transfer. During more than 1,700 cumulative patient days of observation, only one patient in the second cohort reported two unconfirmed spontaneous bleeds, and no such bleeds were reported by any patient during the last six months of observation.

    These clinical data will be presented today in an oral presentation at the 26th Biennial Congress of the International Society on Thrombosis and Haemostasis (ISTH), taking place this week in Berlin, Germany.

    “We continue to observe a therapeutic benefit from AMT-060 that is clearly superior to their previous prophylactic FIX replacement therapy regimen, even in patients with advanced joint disease who experienced a high rate of bleeds prior to gene transfer,” stated Professor Wolfgang Miesbach, M.D., of the University Hospital Frankfurt, Germany.

    “Importantly, AMT-060 appears to be safe and well-tolerated, and quite differentiated, with no loss of FIX activity, no activation of T-cell response and no development of inhibitors for any of the ten patients in the study. The safety profile observed in this study suggests that the AAV5 vector offers long-term safety, efficacy and the potential for broad application in hemophilia B patients,” he added.

    Phase 1/2 Trial Overview

    The AMT-060 gene therapy consists of a codon-optimized wild type FIX gene cassette, the LP1 liver promoter and an AAV5 viral vector manufactured by uniQure using its proprietary insect cell-based technology platform. It is the only hemophilia gene therapy that combines a gene cassette with clinically proven multi-year durability, now out more than five years, and an AAV5 vector serotype that has demonstrated superior safety and broad applicability due to the low prevalence of clinically-relevant titers of neutralizing antibodies (NABs) as evaluated in more than 20 patients across clinical studies in three different diseases.

    The Phase I/II, open-label, multi-center study includes 10 patients each receiving a one-time, 30-minute, intravenous administration of AMT-060, without the prophylactic use of corticosteroids.

    The study includes two dose cohorts of five patients each, with the first cohort receiving 5x1012 gc/kg and the second cohort receiving 2x1013 gc/kg.

    Nine patients in the trial were classified as having severe (<1% FIX activity) hemophilia. One patient in the low-dose cohort had a moderate/severe (1.5% FIX activity) phenotype.

    Patients in the low-dose cohort were characterized by poorly controlled bleeding manifestations despite use of high-dose FIX replacement therapy during the year prior to study compared to the second-dose cohort.

    All but one patient in the study across both cohorts required chronic infusions of prophylactic FIX therapy at the time of enrollment. The remaining patient, who is in the second dose cohort, used FIX therapy on demand.

    Data Update from Phase I/II Clinical Trial of AMT-060 in Hemophilia B Patients

    Data as of May 12, 2017:

    All 10 patients in the study have demonstrated improvements in their disease state as measured by reduced FIX replacement therapy and bleeding frequency.

    In the second-dose cohort, no spontaneous bleeds were reported in the last six months of follow-up, with a reduction in the annualized spontaneous bleed rate of 84% compared to the one-year period prior to administration of AMT-060. Total bleeds were reduced by 64%.

    As previously announced, eight of the nine patients that required chronic FIX infusions prior to administration of AMT-060 have discontinued prophylaxis after treatment. All eight patients remained prophylaxis-free at the last follow up.

    Across both dose cohorts, cumulative annualized FIX consumption decreased by 79%, from 2.64 million to 544,741 IU.

    Through up to 12 months of follow-up among the five patients in the second-dose cohort, the mean steady-state FIX activity persisted at approximately 7% of normal. The mean FIX activity at the last follow-up (52 weeks) was 8.82%, ranging from 5.2% to 10.7%.

    AMT-060 continues to be well-tolerated, and there have been no severe adverse events.

    In both dose cohorts, FIX activity remained consistent and stable through up to 18 months of follow-up with no emergence of late immune response or loss of FIX activity in any of the patients.

    As previously announced, three patients experienced mild, asymptomatic elevations of alanine aminotransferase (ALT) soon after administration. For these patients, ALT levels returned to their baseline readings, no recurrence of ALT elevation has occurred, and no loss of FIX activity was observed.

    No patients across either cohort have developed inhibitory antibodies against FIX, or demonstrated sustained AAV5 capsid-specific T-cell activation.

    “The data from our Phase I/II study demonstrate that AMT-060 continues to deliver sustained and significantly improved clinical benefits over the long term to patients suffering from severe hemophilia B,” stated Matt Kapusta, chief executive officer of uniQure. “Our AAV5-based gene therapy has been clinically demonstrated to be safe, effective and durable, with no loss of efficacy at up to 18 months of observation and no cellular immune responses in any patient. Moreover, our use of an AAV5 construct may enable us to offer the promise of gene therapy to nearly all patients suffering from hemophilia B. We continue to make significant progress preparing for our manufacturing campaign and regulatory interactions to support a potential pivotal study, and look forward to providing an update later this year.”
  3. forum rang 4 ch@rter 10 juli 2017 22:31
    Ik heb alles verkocht zie ik net na thuiskomst. (En nee helaas niet op de 6.98,)

    Wat een absurde spike is er geweest!
    Van de 6.4 naar de 6.98 van de 6.98 naar de 6.10 ..... Brrrr

    Zal qure blijven volgen met een half oog, wellicht om zo nu en dan te traden. LT in bio is verder niks voor mij.

    Good luck fellas
  4. Fartknock 11 juli 2017 13:22
    tools.eurolandir.com/tools/Pressrelea...

    UniQure Presents New Clinical Data in Hemophilia B Patients Demonstrating Therapeutic Efficacy of AAV5 Gene Therapy in the Presence of Pre-Existing Neutralizing Antibodies

    -- Findings Further Support Expanding the Eligibility of AAV5 Gene Therapies to Nearly All Patients with Hemophilia B --


    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, July 11, 2017 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today presented new clinical data demonstrating that the presence of pre-existing anti-AAV5 neutralizing antibodies (NABs) does not predict the potential efficacy of AAV5-mediated gene transfer in patients with hemophilia B. Clinically meaningful factor IX (FIX) activity levels from the ongoing Phase I-II trial of AMT-060 were observed at NAB titers up to 1:341, determined as corresponding up to the 90th percentile of a healthy control population. NABs were quantified in the blood sera of these patients using a highly sensitive assay. These clinical data were presented today in a poster presentation at the 26th Biennial Congress of the International Society on Thrombosis and Hemostasis (ISTH), taking place this week in Berlin, Germany.

    The presence of pre-existing NABs to adeno-associated virus (AAV) vectors has long posed a critical challenge for the clinical application of gene therapies, as patients who currently screen positive for NABs are generally excluded from treatment. Researchers from uniQure recently presented data in non-human primates suggesting that AAV5 could successfully mediate gene transfer in the presence of NABs at levels as high as 1:1031.

    In a poster presentation at the ISTH meeting, a re-analysis was described of pre-gene transfer screening samples from the 10 patients who have been treated in the ongoing Phase I/II trial of AMT-060 for hemophilia B. The patients had tested negative for pre-existing anti-AAV5 NAbs using a green fluorescent protein-based (GFP) assay before receiving treatment. These samples were later re-assessed using a highly sensitive luciferase-based (LUC) NAB assay. Anti-AAV5 NABs were detected retrospectively in three patients who had been treated with the low dose (5x1012 gc/kg) of AMT-060. However, all three patients presented increases in FIX expression and, especially, the patient with the highest NAB level (titer 1:341) had the highest FIX-activity (steady-state FIX 6.8% of normal; latest FIX measurement 10.7% of normal) among all five patients treated in the low-dose cohort. None of the three patients who tested positive for NAB titers, experienced over time elevations in liver enzymes post gene transfer, FIX activity loss, or clinically relevant T-cell responses to the capsid.

    "These clinical data show that hemophilia B patients presenting with neutralizing antibodies may be considered eligible for AAV5-mediated gene transfer," stated Matthew Kapusta, chief executive officer at uniQure. "This development potentially expands the applicability of AAV5 gene therapies to nearly all hemophilia B patients. We believe these factors contribute to making AAV5 a potential best-in-class vector for delivering gene therapies more effectively and safely to a greater portion of patients in need of treatment."
  5. Prof. Dollar 11 juli 2017 15:19
    'The patients had tested negative for pre-existing anti-AAV5 NAbs using a green fluorescent protein-based (GFP) assay before receiving treatment. These samples were later re-assessed using a highly sensitive luciferase-based (LUC) NAB assay. Anti-AAV5 NABs were detected retrospectively in three patients who had been treated with the low dose (5x1012 gc/kg) of AMT-060.'

    Dit zet wel te denken: Eerst zegt men dat er geen pre-existing anti-AAV5 NAbs zijn. En nu - met een andere meetmethode - liefst bij drie patiënten. Nota bene allen uit het eerste cohort; dus meer dan de helft.
  6. Frenky_Tornado 11 juli 2017 23:11
    In the second-dose cohort, no spontaneous bleeds were reported in the last six months of follow-up, with a reduction in the annualized spontaneous bleed rate of 84% compared to the one-year period prior to administration of AMT-060. Total bleeds were reduced by 64%.

    As previously announced, eight of the nine patients that required chronic FIX infusions prior to administration of AMT-060 have discontinued prophylaxis after treatment. All eight patients remained prophylaxis-free at the last follow up.

    Dat zijn heel mooie resulaten mensen. Denk je eens in: niet meer naar het ziekenhuis te hoeven, elke week. Geweldig toch!

  7. forum rang 10 DeZwarteRidder 11 juli 2017 23:26
    quote:

    Frenky_Tornado schreef op 11 juli 2017 23:11:


    In the second-dose cohort, no spontaneous bleeds were reported in the last six months of follow-up, with a reduction in the annualized spontaneous bleed rate of 84% compared to the one-year period prior to administration of AMT-060. Total bleeds were reduced by 64%.
    As previously announced, eight of the nine patients that required chronic FIX infusions prior to administration of AMT-060 have discontinued prophylaxis after treatment. All eight patients remained prophylaxis-free at the last follow up.
    Dat zijn heel mooie resulaten mensen. Denk je eens in: niet meer naar het ziekenhuis te hoeven, elke week. Geweldig toch!

    En wanneer gaat UniQure nu winst maken..?? Over 10 jaar...??? Of nooit..??
  8. Joey R 12 juli 2017 16:06
    quote:

    Prof. Dollar schreef op 29 juni 2017 11:22:


    [...]
    Dat is beslist geen humor, maar wederom een gedraging in het systematisch verkrachten van een ooit inhoudelijk forum. Spijtig.


    Het is bijzonder dat iemand die (blijkbaar?) geen aandelen in een bepaald fonds heeft toch het forum daarvan jarenlang blijft verzieken. Dit stalkgedrag wijst op een psychopathologisch probleem. En je ziet de 1 na de ander (daardoor?) op dit forum af haken.

    Respect voor Prof en de anderen die ondanks dit obsessieve chagrijn hun lezenswaardige bijdragen blijven posten. Ik lees en waardeer ze.
  9. forum rang 10 DeZwarteRidder 12 juli 2017 17:47
    quote:

    Beurs2020 schreef op 12 juli 2017 17:44:


    [...]7 vraagtekens bij suggestieve en negatieve vragen. Wat vind je van het resultaat, dat gepresenteerd werd DZR?

    Ze zijn nog net zover als ca 10 jaar geleden bij AMT.

    Voor het einde van het jaar weer een emissie tegen 4$...???
  10. T. Montana 12 juli 2017 20:32
    quote:

    Joey R schreef op 12 juli 2017 16:06:


    [...]

    Het is bijzonder dat iemand die (blijkbaar?) geen aandelen in een bepaald fonds heeft toch het forum daarvan jarenlang blijft verzieken. Dit stalkgedrag wijst op een psychopathologisch probleem. En je ziet de 1 na de ander (daardoor?) op dit forum af haken.

    Respect voor Prof en de anderen die ondanks dit obsessieve chagrijn hun lezenswaardige bijdragen blijven posten. Ik lees en waardeer ze.


    Helemaal mee eens. Een plaagstoot op zijn tijd is best en zelfs gezond, maar dit is zo beneden ieder peil dat de iex moderator iets aan deze melige Qurepruim zou moeten doen, reglementen of niet.
  11. Joey R 12 juli 2017 21:48
    quote:

    T. Montana schreef op 12 juli 2017 20:32:


    [...]

    Helemaal mee eens. Een plaagstoot op zijn tijd is best en zelfs gezond, maar dit is zo beneden ieder peil dat de iex moderator iets aan deze melige Qurepruim zou moeten doen, reglementen of niet.


    Als iemand in mijn omgeving dit soort gedrag vertoonde zou ik de hulpverlening attenderen. Wat een verknipte mafkees.
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