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P247 A novel AAV-based miQURE gene therapy for SCA3 www.liebertpub.com/doi/10.1089/hum.20... twitter.com/floszcrxl/status/11866263... P336 Prevalence and affinity/avidity assessment of pre-existing NABs against AAV2, 5 and 8 analyzed in the serum of 300 healthy donors www.liebertpub.com/doi/10.1089/hum.20... twitter.com/floszcrxl/status/11866266... P381 Stem cell derived brain organoids, a promising model to study Adeno associated viruses for CNS gene therapy ABSTRACT WITHDRAWN www.liebertpub.com/doi/10.1089/hum.20... MRI-guided injections in the brain: a method for gene therapy delivery for NHP and humans www.liebertpub.com/doi/10.1089/hum.20... twitter.com/floszcrxl/status/11866255... twitter.com/floszcrxl/status/11865709...
Offtopic. Sangamo receiving FDA Orphan drug Designation for Fabry Disease www.accessdata.fda.gov/scripts/opdlis... En hun pipeline: www.sangamo.com/pipeline QURE moet vaart maken!
Prevalence and affinity/avidity assessment of pre-existing NABs against AAV2, 5 and 8 analyzed in the serum of 300 healthy donors $QURE #ESGCT19 uniqure.com/2019.10.24_ESGCT_Immunolo... Cell and Gene meeting on the Mesa, Jon Garenwww.youtube.com/embed/2SuqxPmBsWI Panel: Cell and Gene Therapy for Neurological Product Indications $QURE $VYGR AskBio BlueRockwww.youtube.com/watch?v=PaVgx6PQEBk
Qure stijgt in afgelopen uur met vele procenten. Iemand een idee? Spark staat ook ruim in plus!
Frontrunner schreef op 24 oktober 2019 21:27 :
Qure stijgt in afgelopen uur met vele procenten. Iemand een idee? Spark staat ook ruim in plus!
Gerucht dat er door (nog bekend te maken) besluit van Federal Trade Commission zicht is op voortzetting overname van ONCE door Roche.
Prof. Dollar schreef op 24 oktober 2019 21:47 :
[...]
Gerucht dat er door (nog bekend te maken) besluit van Federal Trade Commission zicht is op voortzetting overname van ONCE door Roche.
Het lijkt nu meer dan een gerucht te worden: FTC staff recommends approval of Roche deal for Spark. Reuters www.reuters.com/article/us-spark-m-a-... Zou dit de overnamespeculaties rondom uniQure weer gaan voeden? Hoe dan ook, met die paar honderd miljoen dollar extra op de bankrekening van QURE na de laatste aandelenuitgifte en met een almaar voortschrijdende ontwikkeling van de onderzoekspijplijn is het bedrijf dusdanig goed gepositioneerd dat het management geen overhaaste beslissingen hoeft te nemen. Met elke stap voorwaarts in de ontwikkelingspijplijn worden de risico’s in dit bedrijf kleiner en het bedrijf meer waard. De tijd zal niet in het nadeel werken van de lange termijn beleggers (wat veel van de bezoekers van dit uniQure forum volgens mij zijn).
Ik heb 'm al een jaartje of wat, sinds AMT. Ik heb nog ff
uniQure Announces Third Quarter 2019 Results and Highlights Recent Company ProgressAnnounced Completion of Full Patient Enrollment in HOPE-B Pivotal Study of Etranacogene Dezaparvovec Achieved Site Initiation and Began Patient Screening for Phase I/II Clinical Study of AMT-130 in Huntington’s Disease Strong Balance Sheet with September 30, 2019 Cash and Cash Equivalents of $403 Million Expected to Fund Operations into Mid-2022 LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Oct. 28, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the third quarter of 2019 and highlighted recent progress across its business. “We achieved a major company milestone for 2019 with the earlier than expected completion of full patient enrollment in the HOPE-B pivotal study of etranacogene dezaparvovec in hemophilia B, as well as the initiation of clinical sites and patient screening for the Phase I/II clinical study of AMT-130 in Huntington’s disease,” stated Matt Kapusta, chief executive officer of uniQure. “We also strengthened our balance sheet with new capital that we expect will fund operations into mid-2022 and allows us to deliver on key corporate goals across the business. We look forward to continuing our momentum through the remainder of the year and sharing our progress during what we believe will be a very productive 2020.” Volledig bericht: tools.eurolandir.com/tools/Pressrelea...
Roche — surprise — delays $4.3B Spark buyout again as regulators hover (Endpoints News) Roche has yet again delayed their Spark buyout. The Swiss giant extended the window on the $4.3 billion gene therapy acquisition through November 25th. It is delay #8 for a deal first announced in February. Roche said the delay was to give UK and US regulators more time to review the acquisition. The Federal Trade Commission staff reviewing the deal reportedly recommended approval last week, months after a rare second information request some analysts thought could augur a divestiture demand. But a recommendation is not an official approval, and across the Atlantic, UK Competition and Markets Authority is not due to announce its Phase I decision until December 16. If the CMA decides the deal warrants a Phase II, it would initiate a more exhaustive probe. Roche CEO Severin Schwan has insisted the Spark acquisition will likely be completed by the end of the year, even as the company has prepared for a more protracted process. Schwan told investors in his third-quarter call last week he was confident the deal would be done before 2020, but the company filed back in July to extend the buyout’s final deadline 3 months to April 30. The regulatory process can be opaque and Roche has not commented on what lays behind the delays. Most of the speculation, though, has been that the holdup is over anticompetitive concerns around Roche’s hemophilia drug Hemlibra and a hemophilia gene therapy in development at Spark, SPK-8011. Conversely, investors have argued that regulators have been overly scrutinizing for a deal of this size and that the persistent delays have had a chilling effect on a gene therapy space that has answered once-big questions about its clinical feasibility but still has large blanks on the business end.
Our CEO Matt Kapusta spoke with @Nasdaq's #BehindTheBell after Tuesday's Opening Bell Ceremony to talk about about what sets uniQure apart and how the company is working to revolutionize the treatment of genetic diseases through GT.www.youtube.com/watch?v=TXl5LOHl8uY twitter.com/uniqure_nv/status/1189555...
Prof. Dollar schreef op 27 september 2019 14:23 :
Het valt te bezien of het nieuwe data betreft. In juni presenteerde QURE '36 Weeks of Follow-Up Data'. En 36 weken maakt ook 9 maanden.
tools.eurolandir.com/tools/Pressrelea... Nieuwe data verwacht ik pas tijdens de ASH Annual Meeting begin december.
Op 10 mei van dit jaar werden de gegevens na 6 maanden AMT 061 gepresenteerd, dan verwacht je toch 12 maanden follow-up ergens de komende 2 weken? tinyurl.com/yymg2gl7
Die verwacht ik tijdens de ASH Annual Meeting in december.
uniQure Announces Publication of Positive Interim Data from Phase IIb Clinical Trial of Etranacogene Dezaparvovec tools.eurolandir.com/tools/Pressrelea... Zouden ze het bewust doen? In nagenoeg elke zin 'Etranacogene Dezaparvovec' opnemen. Zo van dan leren ze het wel te lezen en uit te spreken. EtraDeza, allitereert en bekt beter. Ik ben voor!
What's in a name? It's the results, that matter !
wat is dit voor een persbericht, datum 31 oktober en dan gegevens die op 10 mei al zijn gecommuniceerd! erg vreemd
Tja, lezen is niet altijd makkelijk :-)
Het zijn wel degelijk nieuwe gegevens
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