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uniQure« Terug naar discussie overzicht

Wat is de waarde van uniQure?

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  1. Vitavita 16 april 2017 09:50
    Wellicht berichtgeving over nieuwe ontwikkelingen tijdens de vele aanstaande conferenties/bijeenkomsten?

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, April 03, 2017 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced presentations at the following conferences taking place in April:

    World Orphan Drug Congress USA, April 19 - 21 2017, at the Washington Marriott Wardman Park in Washington D.C.

    Matt Kapusta, Chief Executive Officer, will be presenting a keynote address: Adeno-associated virus (AAV)-based gene therapies for rare, chronic and degenerative diseases, on Friday April 21st, 2017, 9.40 a.m. EDT.

    12th Annual HD Therapeutics Conference by CHDI, April 24 - 27 2017, at the Westin Dragonara Resort in St. Julian's, Malta.

    A presentation new preclinical data on huntingtin gene silencing in a mini-pig model of Huntington's Disease, using allele-specific and non-selective miRNAs, will be presented during the CHDI conference.

    Gene Therapy for Rare Disorders Conference, April 25 - 26 2017, at the Sheraton Boston Hotel, in Boston MA.

    Lance Weed, Vice President of US Operations, will be presenting: Optimizing the Scalability of Gene Therapy Manufacturing, on Tuesday April 25th, 2017, 2.00 p.m. EDT.
    Eileen Sawyer, Director of Global Medical Affairs, will be presenting: Bridging Gaps Through Early Integration of the Internal Medical Affairs Function, on Wednesday April 26th, 2017, 11.00 a.m. EDT.
    Daniel Leonard, Director of Global Patient Advocacy, will be presenting: The Intersection of Patient Advocacy and Gene Therapy: A Case Study in Hemophilia, on Wednesday April 26th, 2017, 2.00 p.m. EDT.

    Alliance for Regenerative Medicines (ARM)'s 5th Annual Cell & Gene Therapy Investor Day, April 27th 2017, at The State Room, in Boston MA.

    Matthew Kapusta, chief executive officer, will be participating in a fireside chat on Thursday, April 27th, 2017, 2.15 p.m. EDT.

    The Company also announced its participation in the following conferences taking place in April:

    Hemophilia Federation of America (HFA)'s Annual Symposium, April 6 - 9 2017, at the Rhode Island Convention Center in Providence, RI.

    The International Liver Congress 2017 by EASL, April 19 - 23 2017 at the RAI in Amsterdam, The Netherlands.

    Read more: www.nasdaq.com/press-release/uniqure-...
  2. Prof. Dollar 16 april 2017 11:11
    Ik heb er mijn bedenkingen bij; kwantiteit is geen kwaliteit. Nieuwe data Huntington kan mogelijk licht werpen op toegepaste vector. Waar blijven die 4DMT vectoren toch?! Want die nieuwe vectoren, promotors, indicaties en daadwerkelijk voordeel op time-to-market moeten ze echt snel een keer over de bühne brengen. Daarmee kunnen ze echt het verschil maken! Daarnaast hebben ze de verwachting uitgesproken om in de eerste helft van 2017 een update te geven over het Hemophilia A programma. Ook dat kan potentiële waarde genereren. Al die andere presentaties doen mijns inziens niet ter zake.
  3. Prof. Dollar 17 april 2017 10:16
    Alvast vooruit lopen op de een mogelijk Hemofilie A programma. Hoewel, ik zie QURE er voor aan het programma niet voort te zetten.

    Roche announces positive interim results for emicizumab in phase III study of children with haemophilia A www.roche.com/media/store/releases/me...
  4. [verwijderd] 17 april 2017 12:38
    quote:

    Prof. Dollar schreef op 16 april 2017 11:11:

    Ik heb er mijn bedenkingen bij
    kwantiteit is geen kwaliteit
    moeten ze echt snel een keer over de bühne brengen
    Daarmee kunnen ze echt het verschil maken!
    Al die andere presentaties doen mijns inziens niet ter zake.
    Vroeger was u zakelijk; het begint nu een b-tje brompotterig te worden.
  5. Prof. Dollar 17 april 2017 21:02
    quote:

    Beurs2020 schreef op 17 april 2017 12:38:

    [...]
    Vroeger was u zakelijk; het begint nu een b-tje brompotterig te worden.
    Waarvan akte; toegegeven, enig cynisme is er inderdaad ingeslopen. Maar hoop doet leven. Zo hield QURE vorig jaar geen R&D Day. Deze wordt mogelijk medio Q3/Q4 wel weer georganiseerd. Een mooi moment voor ze om de 'closed box' te openen.
  6. forum rang 10 DeZwarteRidder 18 april 2017 16:54
    quote:

    Eeuwige Rijkdom schreef op 18 april 2017 16:52:

    Hij heeft ze waarschijnlijk om niet gekregen! Dus dat verlies valt mee . Wat lastiger is dat hij blijkbaar geen potentie ziet op korte termijn voor veel hoger. Of hij had direct cash nodig.?????
    Hij had geld nodig om Galapagos te kopen.....
    lol
  7. Prof. Dollar 20 april 2017 13:56
    Persbericht

    uniQure Announces It Will Not Seek Marketing Authorization Renewal for Glybera in Europe

    -- Marketing Authorization for Glybera® to Expire on October 25, 2017 --

    -- Company Maintains Focus on Core Programs in Hemophilia B, Huntington's Disease and Congestive Heart Failure --

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, April 20, 2017 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will not pursue the renewal of the Glybera®(alipogene tiparvovec) marketing authorization in Europe when it is scheduled to expire on October 25, 2017.

    "The decision to not pursue marketing authorization renewal of Glybera in Europe involved a thoughtful and careful evaluation of patient needs and the clinical use of the therapy, and is not related to any risk-benefit concern," stated Matthew Kapusta, chief executive officer of uniQure. "Glybera's usage has been extremely limited and we do not envision patient demand increasing materially in the years ahead."

    Mr. Kapusta added, "In line with our previously announced strategy, we will focus our resources on advancing our hemophilia B program into a pivotal trial, moving our Huntington's disease program into a clinical proof-of-concept trial, and progressing our research and development collaboration with Bristol-Myers Squibb."

    In October 2012, the European Commission granted a five-year marketing authorization for Glybera under exceptional circumstances as a treatment for a small subset of patients with familial lipoprotein lipase deficiency (LPLD), an ultra-rare genetic disorder. As part of Glybera's approval, uniQure was required to establish a global registry for the long-term surveillance of patients, conduct a post-approval clinical study, submit for annual regulatory reassessments and implement additional risk management procedures. All of these activities required a significant infrastructure for uniQure that included the Company bearing the full costs of maintaining commercial manufacturing capabilities, managing development and validation of numerous assays and supporting regulatory interactions and inspections.

    uniQure has initiated discussions with the European Medicines Agency (EMA) to discuss steps to wind down these various activities and review plans for ongoing patient monitoring.

    Under the terms of the agreement between uniQure and Chiesi Group, which has exclusive rights for the commercialization of Glybera in Europe and other selected countries, uniQure will continue to make product available to Chiesi to treat any patients that are approved for treatment prior to October 25, 2017, and will also be responsible for terminating the Phase IV post-approval study.

    As a result of the withdrawal of Glybera, uniQure expects to reduce future expenses related to the product by approximately $2 million annually, beginning in 2018 and net of any payments to Chiesi. These cost savings will be in addition to those previously announced by the Company related to the consolidation of manufacturing into the Company's Lexington facility. uniQure continues to expect its existing cash resources will be sufficient to fund operations into 2019.
  8. forum rang 10 DeZwarteRidder 20 april 2017 14:00
    quote:

    Prof. Dollar schreef op 20 april 2017 13:56:

    Persbericht
    UniQure Announces It Will Not Seek Marketing Authorization Renewal for Glybera in Europe

    -- Marketing Authorization for Glybera® to Expire on October 25, 2017 --
    Dit debacle heb ik vele jaren geleden al voorspeld, toen de fans nog reclame maakten voor dit hopeloze en peperdure spul.
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