Zo, ik ben er nog maar eens ingedoken:
Onderstaande tekst is samengesteld uit (fragmenten van) persberichten van uniQure/AMT (allemaal oud nieuws overigens):
15 juli 2008. AMT Starts Collaboration With St. Jude Children's Research Hospital on Gene Therapy for Hemophilia B. Company Accesses Exclusive Commercial Rights to Final Therapy:
Under the agreement AMT will sponsor research in Hemophilia B at St. Jude. Ronald Lorijn, CEO of AMT said: "The importance of this collaboration with renowned St. Jude Children's Research Hospital stretches beyond its scientific and business aspects. This collaboration will bring to these patients the hope that a real cure is on the horizon. Access to the Factor IX gene therapy program perfectly complements our gene therapy platform allowing us to develop an effective and long-lasting therapy for Hemophilia B. Dr. Arthur W. Nienhuis and his group at St. Jude have done very important scientific work on this disease and we really look forward to collaborating with them. "Amit Nathwani, M.D., Ph.D., who initiated his work on Factor IX while in Nienhuis' lab, has continued to collaborate with St. Jude faculty Andrew Davidoff, M.D., and John Gray, Ph.D., in developing a novel AAV vector that produces therapeutic levels of Factor IX when given intravenously in animal models. This vector has been licensed to AMT.
AMT uses gene therapy to introduce the functional gene into the patient's cells and thereby restore blood clotting
(http://www.amtbiopharma.com/news/99/182/AMT-Starts-Collaboration-With-St-Jude-Children-s-Research-Hospital-on-Gene-Therapy-for-Hemophilia-B-Company-Accesses-Exclusive-Commercial-Rights-to-Final-Therapy.html).
10 maart 2010. Start of Phase I/II Gene Therapy Clinical Trial for Hemophilia B:
The trial is an open label dose-escalation study using a vector-gene combination developed at the renowned St. Jude Children’s Research Hospital. Dr. Arthur W. Nienhuis of St. Jude is the principal investigator of the on-going trial. The work was initiated at St. Jude more than a decade ago by Drs. Andrew Davidoff and Amit Nathwani and the collaboration has continued following Dr. Nathwani’s return to London. The collaboration involves St. Jude and University College London and other institutions in the US and Britain. The objective of the trial is to assess the safety and efficacy of different doses of hemophilia B gene therapy.
AMT will build on the outcome of this exploratory trial and is preparing for additional clinical development to establish safety, tolerability and proof-of-concept with Factor IX gene therapy produced using AMT’s proprietary, clinically validated production system. AMT has the exclusive commercialization rights to the Factor IX gene used in the St. Jude trial and has the ability to produce gene therapy product for hemophilia B at high quality on a commercial scale. Additional developmental work using AMT’s production system is on-going at St. Jude with AMT support.
(http://www.amtbiopharma.com/news/70/182/Start-of-Phase-I-II-Gene-Therapy-Clinical-Trial-for-Hemophilia-B.html).
16 november 2011. Amsterdam Molecular Therapeutics Receives Orphan Designation in the European Union for Hemophilia B Gene Therapy:
AMT’s hemophilia B program, which consists of an adeno-associated viral (AAV) vector containing the human factor IX gene, is being investigated in a Phase I/II study conducted by St. Jude’s Children’s Research Hospital (Memphis, USA) and University College London (UK). Promising data from an initial 6 patients shows that gene therapy administration resulted in a reduced need for protein replacement treatment, the standard care for hemophilia patients. AMT is preparing for additional clinical development work to establish safety, tolerability and proof-of-concept with a factor IX gene therapy produced using its proprietary AAV production system.
(http://www.uniqure.com/news/128/182/Amsterdam-Molecular-Therapeutics-Receives-Orphan-Designation-in-the-European-Union-for-Hemophilia-B-Gene-Therapy.html).
14 januari 2012. Amsterdam Molecular Therapeutics Receives US Orphan Designation for Hemophilia B Gene Therapy:
AMT’s hemophilia B program, which consists of an adeno-associated viral (AAV) vector containing the human factor IX gene, is being investigated in a Phase I/II study conducted by St. Jude’s Children’s Research Hospital (Memphis, USA) and University College London (UK). Promising data from an initial 6 patients, recently published in the New England Journal of Medicine (N Engl J Med 2011; 365:2357-2365), shows that gene therapy adminis tration resulted in a reduced need for protein replacement treatment, the standard care for hemophilia patients. AMT is preparing for additional clinical development work to establish safety, tolerability and proof-of-concept with a factor IX gene therapy produced using its proprietary AAV production system.
“U.S. orphan designation provides additional support for our hemophilia B gene therapy program and supplements the designation in the EU received in November,” said Jörn Aldag, CEO of AMT. “The early clinical success seen with the program to date by our partners is very encouraging. We will build on this success in the coming months.”
(http://www.amtbiopharma.com/news/132/182/Amsterdam-Molecular-Therapeutics-Receives-US-Orphan-Designation-for-Hemophilia-B-Gene-Therapy.html).