Amsterdam Molecular
Therapeutics (Euronext: AMT), a leader in the field of human gene therapy,
announced today that the first patient has been dosed in the Phase I/II
exploratory clinical trial with a gene therapy product for hemophilia B, a
seriously debilitating and potentially lethal disease.
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. Hemophilia B is an inherited
condition in which patients may have repeated and sometimes life threatening
bleeds after accidental trauma or medical interventions, because they do not
have sufficient functioning of an essential blood clotting factor, called
Factor IX.
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.