Prof Amit Nathwani

What is haemophilia?
What is haemophilia
Research projects
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Prof Amit Nathwani

Professor of Haematology and Director of the Katharine Dormandy Haemophilia Centre and Thrombosis Unit

Prof. Amit Nathwani


Professor Amit C Nathwani
Professor of Haematology and Director of the Katharinee Dormandy Haemophilia Centre and Thrombosis Unit
Royal Free Hospital
Pond Street
London NW3 2QG 
amit.nathwani [at]
T. +44 (0)20 7794 0500 Ext. 34142/35985 (PA)

Research area

Gene Therapy for Monogenetic disorders and Cancer
Regenerative medicine using pluripotent stem cells
Inflammation and thrombosis

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. 

Cancer: 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.

Group members

Jenny Mcintosh
Deepak Raj 
Doyoung Lee
Marc (David) Davies 
Solange Paredes Moscosso
Ifrax Mahamoud
Cecilia Rosales
Marco Della Peruta 
Azadeh Kia Kojouri
Allison Dane
Nishil Patel 
Sara Caxaria
Susanne Arthold
Maria Notaridou
Sajjida Jaffer
Vania Coelho
Satyen Gohil 
Gavin Ling
Jonathan Foley
Carol Leung 
Jenny Mcintosh
Deepak Raj 
Doyoung Lee
Colleen Hamid
Emma Fosbury
Tadbir Bariana (PhD student)
Sandra Le Quellec

Recent publications

Nathwani AC et al; Long term safety and efficacy of human FIX gene therapy in hemophilia B. N Engl J Med. Accepted September 2014

McIntosh J et al; Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant. Blood. 2013 Apr 25;121(17):3335-44
Nathwani AC et al; Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med. 2011 Dec 22;365(25):2357-65. 

Binny C et al; AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage. Blood. 2012 Jan 26;119(4):957-66. 

McIntosh JH et al; Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporine. Gene Ther. 2012 Jan;19(1):78-85.

Herzog, R. W., et al. (2011). AAV Vector Biology in Primates: Finding the Missing Link? Mol.Ther. 19,1923-1924. doi:10.1038/mt. 2011.218.

Mattar, C. N., et al.(2011) Stable Human FIX Expression After 0.9G Intrauterine Gene Transfer of Self-complementary Adeno-associated Viral Vector 5 and 8 in Macaques. Mol.Ther. 19,1950-1960. doi: 10.1038/mt.2011.107.

David, A. L., et al. (2011). Recombinant adeno-associated virus-mediated in utero gene transfer gives therapeutic transgene expression in the sheep. Hum Gene Ther 22(4), 419-426 doi:10.1089/hum.2010.007. 

Ward NJ, et al. (2011). Codon optimization of human factor VIII cDNAs leads to high-level expression. Blood.117(3):798-807. 

Nathwani AC et al; Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins. Mol Ther. 2011 May;19(5):876-85.

Older publications

Nathwani, A. C. et al (2009). Enhancing transduction of the liver by adeno-associated viral vectors. Gene Ther. 16,60-69.

Nathwani AC et al; Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates. Blood. 2007 Feb 15;109(4):1414-21. 

Benjamin R et al; Continuous delivery of human type I interferons (alpha/beta) has significant activity against acute myeloid leukemia cells in vitro and in a xenograft model. Blood. 2007 Feb 1;109:1244-7. 

Nathwani AC et al; Self-complementary adeno-associated virus vectors containing a novelliver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver. Blood. 2006 Apr 1;107(7):2653-61.

Davidoff AM et al; Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway. Blood. 2003 Jul 15;102(2):480-8. 

Nathwani AC et al; Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques. Blood. 2002 Sep 1;100(5):1662-9. 

Nathwani AC et al; Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA. Blood. 2001 Mar 1;97(5):1258-65. 

Nathwani, A. C., et al (2000). Efficient gene transfer into human cord blood CD34+ cells and the CD34+CD38- subset using highly purified recombinant adeno-associated viral vector preparations that are free of helper virus and wild-type AAV. Gene Therapy 7(3), 183-195

Nathwani, A. C. et al. (1999). Adenovirus-mediated expresssion of the murine ecotropic receptor facilitates transduction of human hematopoietic cells with an ecotropic retroviral vector. Gene Therapy 6(8), 1456-1468

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