Novo Nordisk A/S announced positive clinical data for its two hemophilia therapies and said each is shaping up to fill unmet needs in what is an increasingly competitive market.
DUBLIN – Gene therapy developer Freeline Therapeutics Ltd. added $80 million in new investment to take its series C round to $120 million in total. The new cash will help to fund a pivotal trial of its lead gene therapy program in hemophilia B, enable it to continue a phase I/II trial of a gene therapy in Fabry disease and allow it to progress its preclinical programs in Gaucher disease and hemophilia A, while also making ongoing investments in its adeno-associated virus (AAV) gene therapy technology and its manufacturing platform.
LONDON – Freeline Therapeutics Ltd. believes it has found the dose at which FLT-180a, its gene therapy for hemophilia B, will provide a functional cure, promoting expression of factor IX (FIX) blood clotting factor within the normal range.