Batch manufacture of high titer/titre GMP (U.S.A.) compliant lentiviral vector for FDA approval

Описание

We are planning a phase I/II clinical trial to treat a rare genetic disease that causes progressive dementia in children. Our therapy will use autologous CD34+ haematopoietic stem progenitor cells (HSPCs) which are modified ex vivo with LVV to express supraphysiologic levels of a secreted enzyme. In order to progress this therapy, we require a high titre LVV produced to good manufacturing practice (GMP) standard. The supplier must have extensive previous experience manufacturing LVVs to this specification which have achieved regulatory approval for phase I/II clinical trials, ideally for use…