Twelve patients, about half of them women, will take part in Britain's
second gene therapy trial which began last week at two centres in Oxford and Cambridge.
Katherine High, Spark's president and chief scientific officer, expressed her enthusiasm for the early clinical data related to SPK - 8011: «The encouraging start of our SPK - 8011 clinical
trial reinforces the strength of our
gene therapy platform, delivers human proof - of - concept in a
second liver - mediated disease — a significant achievement in the
gene therapy field — and positions us well to potentially transform the current treatment approach for this life - altering disease with a one - time intervention.»