Treatments for Haemophilia

In 2016, Professor Amit Nathwani of UCL and the Royal Free Hospital, was awarded the European Society for Gene and Cell Therapy’s Outstanding Achievement Award for his pioneering work on gene therapy for Haemophilia.

In 2010, in collaboration with St. Jude Children’s Research Hospital, a team led by Professor Nathwani was the first in the world to show correction of bleeding in patients with severe Haemophilia B by using AAV-based gene transfer to express Factor IX.

Since demonstrating clinical Proof of concept, Prof Nathwani has extended this technology to other inherited bleeding disorders, working with the Translational Research Office to develop translational pathways for Haemophilia A and for Factor VII deficiency.

Partnership with biotechnology company Biomarin in the form of a licensing deal has enabled the progression of Haemophilia A gene therapy into phase I/II clinical trial in the UK; Biomarin has built its first gene therapy manufacturing facility to support GMP manufacture of the Factor VIII therapy for clinical trial and commercial production.

Professor Nathwani is founder, member and Chief Scientific Officer of Freeline Therapeutics, a spinout company launched in 2015 by UCL Business PLC and Syncona LLP with an initial investment of £33 million.

Freeline Therapeutics builds on Professor Nathwani’s AAV gene therapy platform technology to drive the clinical translation and commercialisation of treatments for bleeding disorders and other disorders that affect the liver.