New hope for haemophiliacs
Although treatments have improved in recent years, through, for example, prophylactic injections of the missing factor, in past decades patients had to rely on transfusions. This left them vulnerable to infectious diseases, such as hepatitis B in the ’70s, HIV in the ’80s and hepatitis C in the ’90s.
However, that has all changed with a pioneering new gene therapy treatment developed by Professor Amit Nathwani, Professor of Haematology at UCL and Director of the Katharine Dormandy Haemophilia Centre and Thrombosis Unit of Royal Free London NHS Foundation Trust.
Indeed, this innovative gene therapy has already begun to transform lives by providing a safe, reliable source of the blood clotting protein factor IX. A successful haemophilia B phase I/II trial conducted by Professor Nathwani at UCL in collaboration with St. Jude Children’s Research Hospital, Memphis, demonstrated that all treated haemophilia B patients on the trial showed safe, sustained expression of blood clotting factor IX from a single treatment, according to results published in the New England Journal of Medicine.
The Professor is also founder and Chief Scientific Officer (CSO) of a new company – Freeline Therapeutics – their lead programme is a factor IX gene therapy programme. Launched in December 2015 by Syncona LLP and UCLB, Freeline Therapeutics is based at the Royal Free Hospital in London. Their mission is to become a leading biopharmaceutical company for the successful development and commercialisation of liver-directed gene therapies for bleeding disorders and other severe diseases.
A single treatment, long term benefits
Freeline’s therapies, delivered in one single treatment, have the potential to provide long-term benefits to patients by carrying a therapeutic gene to a target cell in the body. These therapies are based on the company’s next-generation proprietary adeno-associated virus (AAV) vector platform.
Freeline’s lead programme is the gene therapy to treat haemophilia B, which is expected to be in clinics by late 2017.
Professor Nathwani commented, ‘Our initial study has shown the potential of AAV gene therapy. Freeline will bring industry-leading development people and our expertise together to ensure our clinical translation is rapidly converted to registered therapeutics.’
Cengiz Tarhan, Managing Director of UCLB, added, ‘UCL is a world leader in the biomedical sciences, with a commitment to outstanding research and translation into healthcare benefits for patients. I am pleased that Professor Nathwani’s work is being taken forward in a commercial environment in a way that may benefit patients globally. UCLB are delighted to be able to partner with Syncona to launch the company.’