Volgens mij is dit de grootste troef van AMT. Resulataten zijn echt goed. Valt mij ook op dat AMT in het artikel niet wordt genoemd, maar het is wel degelijk hin technologie. Ik denk dat hier wel een grote partij instapt, maar dit is nog wel lang weg van de markt.
Quote from the AMT website:
AMT has been doing A Phase I/II exploratory clinical trial to assess the safety and efficacy of different doses of hemophilia B gene therapy. The trial is being conducted by St. Jude. AMT will build on the outcome of this trial, and is preparing for additional clinical development work to establish safety, tolerability and proof-of-concept with a Factor IX gene therapy produced using AMT’s proprietary production system.
This gene therapy has a huge market and partnering potential as it could replace Factor IX replacement entirely. AMT intends to partner it in the short- to mid-term.
Yesterday the results were published in the NEJM, December 10, 2011 (10.1056/NEJMoa1108046):
In summary, we (St Jude) et al. have found that a single peripheral-vein infusion of our scAAV2/8-LP1-hFIXco vector consistently leads to long-term expression of the FIX transgene at therapeutic levels, without acute or long-lasting toxicity in patients with severe hemophilia B. Immune-mediated, AAV-capsid–induced elevations in aminotransferase levels remain a concern, but our data suggest that this process may be controlled by a short course of glucocorticoids, without loss of transgene expression. Follow-up of larger numbers of patients for longer periods of time is necessary to fully define the benefits and risks and to optimize dosing. However, this gene-therapy approach, even with the associated risk of transient hepatic dysfunction, has the potential to convert the severe bleeding phenotype into a mild form of the disease or to reverse it entirely.