Institute of Immunity and Transplantation


Amit Nathwani

Professor of Immunology

Research area

Gene therapy for monogenetic disorders and cancer, regenerative medicine using pluripotent stem cells, inflammation and thrombosis.

Research programme

Research summary

The main thrust of my research is the development of novel treatment avenues for haematological disorders and cancer using gene transfer and cell based therapies.

Monogenetic disorder
My group has developed a distinctive approach for gene therapy of haemophilia B using a self-complementary adeno-associated viral vector (AAV), in collaboration with St Jude Children's Research Hospital in Memphis Tennessee.  We have demonstrated the preclinical safety and efficacy of this new vector system and are about to commence a Phase I/II study in subjects with severe haemophilia A. Other clinical targets that are currently being evaluated include: Congenital FVII and FX deficiency, urea cycle and lysosomal storage disorders.

Haemato-oncology research is focused on the development of two emerging strategies: immunotherapy and anti-angiogenesis. Gene-therapy approaches are central to each of these strategies. Immunotherapy strategies using engineered T cells are being evaluated for the treatment of chronic lymphocytic leukaemia, a disease that remains incurable. Angiogenesis is required for the growth of tumours including haematological malignancies thus making it an important target for anti-cancer strategies. Long-term expression of an angiogenesis inhibitor is likely to be required and the role of gene therapy-mediated delivery of these agents is being evaluated.

Stem cells
Recently we have begun a program of research on the use of induced pluripotent stem (iPS) cells for the treatment of degenerative conditions as well as disease models. Through funding from the UKRMP/MRC we are evaluating methods for the generation of iPS cells in a GMP environment.

Inflammation and thrombosis
We are developing novel insights into the roles of mediator of inflammation in the pathogenesis of Thrombosis.

Patient involvement

I am Director of the Katharine Dormandy Haemophilia Centre and Thrombosis Unit at the Royal Free London NHS Trust. Our clinical team cares for over 5000 patients with inherited bleeding disorder, almost 50% of whom receive clotting factor concentrate.  The majority have been extensively characterised at the phenotypic and genotypic level.

We have many research studies taking place in the department which we invite our patients to enrol for. These include novel clotting factor, gene therapy as well as studies focused on getting better, insight into the coagulation pathway.


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