uniQure « Terug naar discussie overzicht

quote:

rationeel schreef op 19 juni 2019 15:07:

Dank voor de informatie:)

Je hoopt er altijd op, in de wandelgangen iets meer op te vangen dan wat er zoal op papier staat;)

Voorlopig maar dromen over een overname met koers 200:).

quote:

streber schreef op 19 juni 2019 16:20:

[...]
Wat betreft die overname, het gevoel dat er iets staat te gebeuren is bij mij na vandaag niet minder geworden.

quote:

DeZwarteRidder schreef op 19 juni 2019 16:25:

[...]
Ik denk dat de verspreiders van dit gerucht een paar miljoen verdiend hebben.

• In July, presentation of nine month follow-up data;
• At year end, probably one year follow-up data.

• To date, several patients have completed the lead-in period of six months and are actually dosed;
- At year end, company expects completion of enrollment (approximately 55 patients). Thus last patient will receive dosing in mid 2020);[/li]
• At year end, company will communicate outlook for next year. Probably, as part of their undisclosed market approval strategy (and they have several options), they will announce full data at year end 2020 at ASH conference (no intermediate top-line data expected, although this is not yet certain);
• Company emphasizes to strongly believe to be first and best in class.

• On track to submit an IND before year end.

• Company is very enthusiastic about this program and is strongly convinced of their unique product. It is the pride of all Qurians, the passion to give patients with this disease a better future;
• It is not about the highest knockdown (100% knock down, as far as possible, has possibly negative consequences). Their preclinical work indicates the right range;
• At year end, publication of top-line safety data.

• In the fall, company expects to hold an R&D session (probably not as extensive as last year);
• Company explores possibilities of miQURE technology in therapeutic area of liver. Thus potential for new programs.

• No further comments on BMS partnership (agreed research period is coming to an end and QURE does not intend to extend it; see annual report);
• No comment on potential partnership/acquisition ('there has always been interest in the company');
• Company has sufficient money to run operation into 2021.

quote:

DeZwarteRidder schreef op 19 juni 2019 16:25:

[...]
Ik denk dat de verspreiders van dit gerucht een paar miljoen verdiend hebben.

quote:

flosz schreef op 19 juni 2019 20:20:

Hartelijk dank pim, streber, Prof.!
@streber: thx.& groeten terug!

• AMT-061 (AAV5-Padhua hFIX variant) an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: Follow-up up to 9 Months in a Phase 2b Trial (OC 01.1)
  Oral Session Title: Gene Therapy Clinical I
  Date and Time: Saturday July 6, 1:00 – 1:15 p.m. AEST/ Friday July 5, 11:00 p.m. ET
  Location: Melbourne Room 2


• Stable Expression of FIX and Maintained Reductions in Bleeding and Factor IX Consumption Following AMT-060 Gene Therapy with up to 3.5 Years of Follow-up in Adults with Severe or Moderate-Severe Hemophilia B (OC 01.4)
  Oral Session Title: Gene Therapy Clinical I
  Date and Time: Saturday July 6, 1:45 – 2:00 p.m. AEST/ Friday July 5, 11:45 p.m. ET
  Location: Melbourne Room 2


• AAV5-mediated Gene Therapy for Hemophilia A with Factor IX Variant that Functions Independently of FVIII (OC 22.3)
  Oral session title: Gene Therapy Basic I
  Date and Time: Sunday July 7, 3:15 – 3:30 p.m. AEST/ Sunday July 7, 01:15 a.m. ET
  Location: Melbourne Room 1


• No Evidence of Germline Transmission of Vector DNA Following Intravenous Administration of AAV5-hFIX to Male Mice (PB 0303)
  Poster Date and Time: Sunday, July 7, 6:30 – 7:30 p.m. AEST/ Sunday July 7, 4:30 a.m. ET
  Location: Exhibition Hall


• Low Predicted Immunogenicity Risk Associated with FIX Variants that can Promote Coagulation in the Absence of FVIII: in vitro and in silico assessments (PB 0301)
  Poster Date and Time: Sunday, July 7, 6:30 – 7:30 p.m. AEST/ Sunday July 7, 4:30 a.m. ET
  Location: Exhibition Hall