Pfizer is establishing “a gene therapy platform” by signing a haemophilia B pact with Spark Therapeutics and recruiting a leading UK scientist to head up rare disease genetic research.

First up, the firms will collaborate on SPK-FIX, a programme using bio-engineered AAV vector for the potential treatment of haemophilia B. Philadelphia-based Spark, which is getting $20 million upfront, will be responsible for development through Phase I/II and the deal could be worth up to more than $260 million.

Geno Germano, head of the global innovative pharma business at Pfizer, said  “we believe the SPK-FIX programme could add to our existing portfolio of haemophilia products and could pioneer a potential new treatment technology for patients with bleeding disorders”. The current haemophilia B market leader is Pfizer's own BeneFix (recombinant coagulation factor IX).

Pfizer also noted that it has appointed Michael Linden of King’s College London and director of the University College London Gene Therapy Consortium, who will be with the company for a two-year secondment to head up the new group.

Pfizer R&D chief Mikael Dolsten said “the fundamental understanding of the biology of hereditary rare diseases, coupled with advances in the technology to harness disarmed viruses as gene delivery vehicles, provide a ripe opportunity to investigate the next wave of potential life-changing therapies”. He added that “by establishing our gene therapy capabilities, we hope to gain a deeper understanding of the mechanisms that could potentially bring true disease modification for…hematologic and neuromuscular diseases”.