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  1. Prof. Dollar 20 april 2017 19:41

    den haan schreef op 20 april 2017 18:55:

    Ik had wel een afstraffing verwacht, valt nog behoorlijk mee

    Dat heeft al eerder plaatsgevonden (-15%) toen QURE bekendmaakte dat ze Glybera niet in de US gaan introduceren. Het is vandaag heel simpel: Glybera is geen toegevoegde waarde.
  2. Prof. Dollar 24 april 2017 23:10

    uniQure Announces Presentations at the Upcoming Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT)

    Investor and Analyst Breakfast to be Held on Friday, May 12 at 7 a.m. EDT to Feature Data Presented at Meeting

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, April 24, 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 five abstracts have been accepted for presentation at the American Society of Gene and Cell Therapy (ASGCT) 20th Annual Meeting held in Washington, D.C., May 10-13, 2017. Together with their collaborators, members of uniQure's research and development team will present data focused on progress in re-administration protocols, gene therapy delivery systems and optimized vector distribution technologies in two of uniQure's key programs, hemophilia B and Huntington's disease. An investor and analyst breakfast meeting featuring senior members of uniQure's research and development team will be held on Friday, May 12 at 7 a.m.

    "The data to be presented at the ASGCT meeting demonstrate the development and validation of uniQure's technologies to improve gene therapy as a therapeutic approach, and supports our aim to rapidly bring new disease-modifying therapies to patients with severe genetic diseases," stated Matthew Kapusta, chief executive officer of uniQure.

    Harald Petry, Ph.D., chief scientific officer at uniQure, added, "We are making strong progress in advancing the preclinical development of AMT-130 in Huntington's disease, and further enhancing AAV vector technology and the potential for re-administration protocols, which continue to provide compelling evidence of our modular platform."

    Specific details on uniQure's presentations at ASGCT include:

    Title: Novel AAV Vector Reservoirs: peripheral Blood Cells and Hematopoietic Progenitors. (collaborator presentation)
    Oral Session Title: AAV Vector Biology
    Date and Time: Wednesday, May 10, 2017, 10:30 a.m. EDT
    Location: Marriott Salon 1

    Title: Circulating Anti-AAV5 Neutralizing Antibody Titers up to 1:1031 Do Not Affect Liver Transduction Efficacy of AAV5 Vectors in Non-Human Primates (Poster 198).
    Poster Session Title: Immunological Aspects of Gene Therapy and Vaccines I
    Session Date and Time: Wednesday, May 10, 2017, 5:30 - 7:30 p.m. EDT
    Location: Hall A&B South

    Title: Successful Repeated Hepatic Gene Delivery in Non-Human Primates Achieved with AAV5 by Use of Immune Adsorption (Poster 395).
    Poster Session Title: Immunological Aspects of Gene Therapy and Vaccines II
    Session Date and Time: Thursday, May 11, 2017, 5:15 - 7:15 p.m. EDT
    Location: Hall A&B South

    Title: AAV5-miHTT Gene Therapy Demonstrates Broad Vector Distribution and Strong Mutant Huntingtin Lowering in a Huntington's Disease Minipig Model.
    Oral Session Title: Preclinical Progress Towards Therapies for Neurologic Disorders
    Date and Time: Friday, May 12, 2017, 4:30 p.m. EDT
    Location: Delaware AB

    Title: Detection of AAV Vector DNA and Transgene RNA in Liver Tissue by Fluorescent In Situ Hybridization (Poster 567).
    Poster Session Title: AAV Vectors III
    Session Date and Time: Friday, May 12, 2017, 5:45 - 7:45 p.m. EDT
    Location: Hall A&B South

    Investor and Analyst Breakfast
    uniQure management will host an investor and analyst breakfast meeting featuring members of the research and development team to review the data presented during ASGCT.

    Date and Time: Friday, May 12, 2017 at 7:00 am EDT
    Location: Omni Shoreham Hotel, The Congressional Room, 2500 Calvert Street NW, Washington, DC.
    The Omni Shoreham hotel is located directly across the conference venue.
    To request attendance at the meeting, please RSVP to by May 5, 2017, as space is limited.
  3. forum rang 10 DeZwarteRidder 25 april 2017 08:26
    Biotech firm pulls pioneering gene therapy due to no demand
    ReutersApril 20, 2017

    By Ben Hirschler

    LONDON, April 20 (Reuters) - The biotech company behind the Western world's first gene therapy and the most expensive prescription medicine in history is giving up on the product because of lack of demand.

    Only one patient has been treated commercially since the drug was first approved in Europe nearly five years ago, a spokeswoman for Dutch-based UniQure said on Thursday.

    UniQure went into the record books when its gene therapy Glybera was approved by European regulators for an ultra-rare blood disorder in 2012, and the drug was finally launched two years later with a price tag of around $1 million per treatment.

    But the number of patients eligible for treatment was always tiny and, with no sign of demand improving, the company said it had decided not to renew Glybera's five-year European marketing authorisation, which is due to expire on Oct. 25.

    "Glybera's usage has been extremely limited and we do not envision patient demand increasing materially in the years ahead," said UniQure Chief Executive Matthew Kapusta.

    The group, which had already decided not to pursue a U.S. approval for the drug, said the decision was not related to any safety concerns.

    Glybera is given as a series of injections to fight lipoprotein lipase deficiency (LPLD), a disabling condition that clogs the blood with fat. The drug is sold in Europe on UniQure's behalf by Italian drugmaker Chiesi Farmaceutici.

    The commercial flop is a reminder of the economic challenges facing the emerging field of gene therapy, which seeks to cure rare genetic diseases by offering a one-time fix of a faulty DNA but inevitably comes at a very high price.

    However, the setback is unlikely to derail rising investor interest in gene therapy, which has been triggered recently by a number of advances in treating a range of genetic diseases, most of which affect far more patients than LPLD.

    Industry analysts said the decision to pull the plug on Glybera would make little difference to UniQure's financial outlook. In fact, the move will save some $2 million in annual costs and help UniQure focus on other gene medicines.

    The Nasdaq-listed firm has high hopes for its next wave of gene therapies against haemophilia, Huntington's disease and congestive heart failure, where it has a collaboration with Bristol-Myers Squibb.

    UniQure, whose shares slipped 2 percent in early trading, said it would continue to make Glybera available to Chiesi to treat any patients approved for therapy in Europe before Oct. 25.

    Scientists have been working on gene therapies for more than a quarter of a century but it is only recently that the approach has started to become a commercial reality, although the U.S. Food and Drug Administration has yet to approve any. (Editing by Adrian Croft)
  4. forum rang 10 DeZwarteRidder 25 april 2017 10:49
    Op minder gebied actief

    UniQure denkt nog steeds een rol van betekenis te kunnen spelen. Maar dan moet het bedrijf volgens Kapusta zijn inspanningen wel richten op een beperkt aantal onderzoeksterreinen, 'waar we echt verschil kunnen maken'. De toegenomen focus gaat ook gepaard met verlies aan arbeidsplaatsen. Begin dit jaar kondigde UniQure al aan dat vijftig tot zestig medewerkers hun baan zullen verliezen, vooral in Amsterdam.

    Eén van de door Kapusta geselecteerde onderzoeksterreinen is de Ziekte van Huntington, een erfelijk aandoening die delen van de hersenen aantast. Het is een terrein waar de concurrentie niet erg hevig is. UniQure is waarschijnlijk het eerste bedrijf dat een gentherapie op patiënten met Huntington gaat testen.

    Gentherapie tegen hartfalen

    Het belangrijkste onderzoeksterrein blijft zonder twijfel de ontwikkeling van een gentherapie tegen hartfalen - in potentie een markt van miljarden euro's. Dit onderzoek is ook de kurk waarop UniQure drijft: twee jaar geleden sloot het Amsterdamse biotechbedrijf een lucratieve deal met Bristol-Myers Squibb (BMS), een groot Amerikaanse farmaconcern, over de gezamenlijk ontwikkeling van nieuwe medicijnen tegen hartfalen.

    In ruil voor de kennis van UniQure maakte BMS honderden miljoenen dollars over naar Amsterdam, waarop de beurskoers van UniQure tot ongekende hoogtes omhoog schoot. Inmiddels is de koers sterk teruggevallen - van meer dan $30 tot minder dan $6 - maar UniQure beschikt nog altijd over een sterke kaspositie waardoor het biotechbedrijf voorlopig vooruit kan. In de loop van dit jaar komen BMS en UniQure met nadere mededelingen over de vorderingen van hun onderzoek naar hartfalen.

    Positieve gegevens over hemofilie

    Hoge verwachtingen heeft Kapusta ook van een gentherapie voor patiënten met de bloederziekte hemofilie-B. Het biotechbedrijf publiceerde vorig jaar positieve onderzoeksgegevens over de behandeling van tien patiënten met deze ziekte. Bij veruit de meeste patiënten was het aantal bloedingen na een behandeling met de experimentele gentherapie sterk afgenomen.

    Ondanks de positieve uitkomst was de ontvangst van het onderzoek sceptisch, vooral omdat UniQures belangrijkste concurrent op het zelfde moment onderzoeksresultaten publiceerde die nog beter lijken te zijn. Kapusta wijst er echter op dat de data een gecompliceerd beeld geven en voor meerdere uitleg vatbaar zijn. Duidelijk is in ieder geval dat de therapie van UniQure veilig is - wat een belangrijk voordeel kan zijn. Het wachten is nu op de resultaten van vervolgonderzoek. Daaruit moet vooral blijken of de positieve effecten werkelijk langere tijd aanhouden.
  5. T. Montana 25 april 2017 13:38
    uniQure Receives European Medicines Agency Priority Medicines (PRIME) Designation for AMT-060 in Hemophilia B

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, April 25, 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 AMT-060, its proprietary, investigational gene therapy in patients with severe hemophilia B, has received PRIME designation by the European Medicines Agency (EMA). This designation is based on results from the ongoing, dose-ranging Phase 1-2 study that show a near cessation of spontaneous bleeding in patients with severe disease at up to 12 months follow-up, clinically significant and sustained increases in Factor IX (FIX) and substantial reductions in FIX replacement usage.

    “We are very pleased to have AMT-060 for hemophilia B accepted into the PRIME program,” stated Matthew Kapusta, chief executive officer of uniQure. “Similar to the Breakthrough Therapy designation that AMT-060 received from the U.S. Food and Drug Administration earlier this year, we look forward to this enhanced collaboration with the EMA to advance the clinical development of this potentially transformative therapy for hemophilia B patients.”

    Phase 1-2 Data

    Updated clinical data from the ongoing, two-cohort Phase 1-2 trial of AMT-060 were presented last December at the 58th American Society of Hematology (ASH) Annual Meeting. The data included up to 52 weeks of follow-up from the low-dose cohort and up to 31 weeks of follow-up from the second dose cohort.

    Data from the second-dose cohort show a dose response with substantial improvement in disease state in all five patients, including the discontinuation of precautionary FIX infusions in all four patients that previously required chronic replacement therapy. To date, only one spontaneous bleed was reported after discontinuation of prophylactic FIX replacement therapy.

    All five patients in the low-dose cohort, whose bleedings were previously uncontrolled despite being managed with prophylactic therapy, continue to maintain robust, constant and clinically meaningful levels of FIX activity for up to 52 weeks post treatment, with a complete cessation of spontaneous bleedings in the last 14 weeks of observation.

    AMT-060 continues to be well-tolerated, and there have been no severe adverse events. Three out of the total of 10 patients (two in the second-dose cohort and one previously reported from the low-dose cohort) experienced mild, asymptomatic elevations of alanine aminotransferase (ALT) and received a tapering course of corticosteroids per protocol. Importantly, the temporary elevations in ALT were not associated with any loss of endogenous FIX activity or T-cell response.

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

    About PRIME Designation

    The PRIME program was launched by the EMA in March 2016, and the designation is designed to aid and expedite the regulatory process for investigational medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options. To be accepted, an investigational medicine must show the potential to benefit patients with unmet medical needs based on early clinical data. Medicines accepted into the PRIME program are considered priority medicines within the European Union (EU).

    About Hemophilia B

    Hemophilia B is a serious and rare inherited disease in males characterized by insufficient blood clotting. The condition can lead to repeated and sometimes life-threatening episodes of external and internal bleeding following accidental trauma or medical interventions. Severe hemophilia is characterized by recurrent episodes of spontaneous joint bleeds, that cause long-term damage to the joints resulting in disabling arthropathy. Bleeds may be fatal if they occur in the brain. The deficient blood clotting results from the lack of functional human Factor IX, or hFIX. Treatment of hemophilia B today consists of prophylactic or on-demand protein replacement therapy, in which one to three times weekly intravenous administrations of plasma-derived or recombinant hFIX are required to prevent bleeding and once daily infusions in case bleeding occurs. Hemophilia B occurs in approximately 1 out of 30,000 live births.
  6. Prof. Dollar 25 april 2017 13:52
    De weg naar goedkeuring in zowel US en EU lijkt te zijn geplaveid met goede voornemens van goedkeurende instanties. Nu nog de mededeling van QURE dat ze voldoende geschikte patiënten hebben die deel willen nemen aan de studie en dat de productie al gereed is. Vaak zijn dit de twee onverwachte spelbrekers.
  7. Prof. Dollar 25 april 2017 14:17

    cqtvld schreef op 25 april 2017 00:15:

    Ze zijn dus goed bezig Prof.we zullen zien wat dit brengt in de toekomst ,bedankt voor deze bijlage....

    Het zijn zeker positieve ontwikkelingen... toch zie ik graag twee dingen heel snel gebeuren:

    1. Dat ze dat platform als 'product' etaleren. Want als bijvoorbeeld dat GDNF-gen zo beloftevol is en je hebt zelf niet het geld voor doorontwikkeling, ga het dan 'verpakken' (vectoren, promotors, toediening) voor een ander en laat die het onderzoek doen op verschillende targets: Parkinson, ALS, Epilepsy, Alzheimer, Multiple Sclerosis, etc. Kan een mooie BMS-deal uit voortkomen. Nu legt QURE geld neer voor de licentie op het GDNF-gen; tot nu toe jammer van de investering. Maar ook andere farmaceuten zouden een licentie op het platform kunnen nemen. Voortzetting Sanfilippo B? Op de website van uniQure wordt niet echt 'uitgepakt' met het platform. Het is eerder stil rondom de ontwikkeling van nieuwe vectoren en promotors. Jammer.

    2. Dat ze resultaten laten zien uit 'multiple undisclosed programs'; mogelijk toekomstige eigen programma's. Het is veel te stil rondom Hemofolie A. En is er dan niks meer gedaan met de eerdere programma's AIP en Duchenne? Ze kunnen het niet maken te blijven spreken van 'undisclosed programs'.

    Kortom, die academische vorderingen zijn mooi maar nog mooier is als daar concrete producten uit voortkomen. De pipeline van QURE is krimpende en de onderneming steeds meer een 'closed box'.
  8. forum rang 10 DeZwarteRidder 25 april 2017 14:28
    UniQure veramerikaniseert

    UniQure is een Amsterdams bedrijf, maar is afgelopen jaren sterk veramerikaniseerd. Zo houdt topman Matt Kapusta kantoor in de Amerikaanse vestiging in Lexington, vlakbij Boston. Daar produceert UniQure ook zijn experimentele medicijnen. De Amsterdamse productievestiging wordt gesloten. De top van het bedrijf telt nog maar één Nederlander en dat is hoogleraar Sander van Deventer, ooit een van de oprichters van Amsterdam Molecular Therapeutics (AMT), waaruit UniQure voortkomt. Als niet-uitvoerend bestuurder blijft Van Deventer op de achtergrond. Kapusta verzekert dat er geen plannen zijn om Amsterdam te verlaten.
  9. forum rang 4 ch@rter 25 april 2017 23:16

    Prof. Dollar schreef op 25 april 2017 13:52:

    De weg naar goedkeuring in zowel US en EU lijkt te zijn geplaveid met goede voornemens van goedkeurende instanties. Nu nog de mededeling van QURE dat ze voldoende geschikte patiënten hebben die deel willen nemen aan de studie en dat de productie al gereed is. Vaak zijn dit de twee onverwachte spelbrekers.

    Klinkt in ieder geval enigszins hoopvol prof
    De koers is om te janken
  10. T. Montana 26 april 2017 13:22
    uniQure Presents New Preclinical Data on AMT-130 in Huntington’s Disease at CHDI’s 12th Annual Huntington’s Disease Therapeutics Conference

    -- One-time Administration of AMT-130 Demonstrates for the First Time Efficacy in Large Animal Model

    -- Strong Dose-Dependent Reduction of Mutant Huntingtin Protein and Widespread Vector Distribution in Brain

    -- IND-enabling Toxicology Study to Commence in 2H 2017 --

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, April 26, 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 preclinical data on AMT-130, a gene therapy candidate for the treatment of Huntington’s disease (HD), at the 12th Annual CHDI HD Therapeutics Conference in Malta.

    Data from the study demonstrate widespread and effective AAV5 vector distribution and extensive silencing of the human mutant huntingtin gene (HTT) in minipigs, among the largest HD animal models available for testing. AMT-130 consists of an AAV5 vector carrying a DNA cassette encoding artificial micro-RNA (miHTT) that silences the huntingtin gene. The proof-of-concept study was performed by uniQure in collaboration with Prof. Jan Motlik, Director of the Institute of Animal Physiology and Genetics in the Czech Republic and Ralf Reilmann, Founding Director of the George Huntington Institute in Germany.

    “Using AAV vectors to deliver micro-RNAs directly to the brain represents a highly innovative approach to treating Huntington’s disease,” stated Prof. Motlik. “This study demonstrated that a single administration of AAV5-miHTT resulted in significant reductions in HTT mRNA in all regions of the brain transduced by AMT-130, as well as in the cortex. Consistent with the reduction in HTT mRNA, we also observed a clear dose-dependent reduction in mutant huntingtin protein levels in the brain, with similar trends in the cerebral spinal fluid. Taking into account the similarities of CHDI’s proprietary transgenic pig model to the human brain, these results provide additional data to support moving forward with clinical trials of uniQure's promising gene therapy for Huntington’s disease.”

    Preclinical Data Findings

    Researchers in the study investigated the feasibility, efficacy and safety of AMT-130 in diseased animals with a larger brain size using a transgenic HD minipig model developed by Prof. Motlik and supported by the CHDI Foundation. AMT-130 was administered bilaterally into the striatum and thalamus of the minipigs using convection-enhanced, real-time MRI-guided delivery.

    Three months after treatment, widespread, dose-dependent distribution of the vector was observed throughout the minipig brain that corresponded strongly with the miHTT expression. Expression of mutant HTT mRNA was significantly reduced in all regions of the brain transduced by AMT-130 by 50% to 80%, as well in the cortex by up to 40%, compared with control. Researchers also observed a dose-dependent reduction in mutant huntingtin protein levels of more than 50% in the brain, as well as similar trends in cerebral spinal fluid. Both the surgical procedure and AAV5-miHTT treatment were well tolerated with no adverse events.

    “This study is an important step in our Huntington’s disease gene therapy program, demonstrating for the first time in a large animal model that AAV5 can be used safely and effectively to deliver micro-RNAs to silence mutant huntingtin,” stated Pavlina Konstantinova, Ph.D., director of new therapeutic target discovery at uniQure. “We are very encouraged by the significant reductions in mutant huntingtin protein, and believe that knock-down of this magnitude has the potential to significantly alter the course of the disease. The positive data from this study, together with data from our previous studies in rodent models showing strong reductions in huntingtin and prevention of neuronal dysfunction, provide strong proof of concept for AMT-130 as a potential groundbreaking treatment for patients suffering from Huntington’s disease. We look forward to commencing the toxicology study in non-human primates later this year, which we expect to support an Investigational New Drug (IND) application for AMT-130 in 2018.”
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