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UCL Institute of Ophthalmology

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Prof Robin Ali

Prof Robin Ali

Professor of Human Molecular Genetics

Institute of Ophthalmology

Faculty of Brain Sciences

Joined UCL
1st Aug 1994

Research summary

The main focus of Robin Ali's research is the development of gene and cell therapy for the treatment of retinal disorders. He was one of the first to demonstrate efficient AAV-mediated photoreceptor transduction (Human Mol Genet, 1996). He then optimised production of vectors and efficiency of transduction, including development of new vector systems (Nature Med, 2006) and was also first to demonstrate functional rescue of a mouse model of retinitis pigmentosa (Nature Genet, 2000). This established proof-of-concept (POC) for ocular gene therapy. Over the last 10 years, he has demonstrated POC for gene therapy for a wide range of ocular disorders, with effective rescue of 10 animal models of retinal dystrophy, as well as demonstrating POC for gene therapy for ocular angiogenesis and uveitis. In doing so, he has established a pipeline of therapies, supported by charities, UK National Institute of Health Research, UK Medical Research Council and industry. As chief investigator, he established one of the world’s first clinical trials of gene therapy for retinopathy. The results from this trial reporting an improvement in vision (Bainbridge et al, NEJM, 2008, NEJM 2012), along with results from two other trials, established proof-of-principle of gene therapy for inherited retinal degeneration and are widely regarded as a landmark for the whole gene therapy field.  He has built a  broad programme of translational research that has the clinical and regulatory expertise to conduct a number of clinical trials of gene therapies over the next 5 years. The GMP vectors for these trials are being produced at the UCL GMP vector facility. The trials are supported through public and private funding, including from MeiraGTx, a company of which he is a Founder and Chief Scientific Officer. By the end of 2017, he will have initiated clinical trials for at least 4 inherited retinopathies, including LCA2, achromatopsia and X-linked RP. He has also established an extensive program to develop cell therapy for retinal degeneration focusing on the transplantation of photoreceptor cells (Nature, 2006, Nature 2012, Nature Biotech, 2013, Nature Comm, 2016).

Education

University College London
PhD, Genetics | 1993
University College London
BSc, Genetics | 1988

Biography

Robin Ali is Professor of Human Molecular Genetics at UCL Institute of Ophthalmology.  He is also Theme Leader for Gene Therapy at NIHR Biomedical Research Centre, Moorfields Eye Hospital and Director of the Wolfson Gene Therapy Unit, which is the UCL GMP facility for manufacturing clinical grade gene therapy vectors. He is Visiting Professor of Ophthalmology at Kellogg Eye Center, University of Michigan and Founder and Chief Scientific Officer of MeiraGTx, a UK incorporated gene therapy company with offices in London and New York. The company has secured substantial investment to develop a path to market for a number of ocular therapies licensed from UCL. The main focus of Robin Ali’s research is the development of gene and cell therapy for the treatment of retinal disorders. Since 1996, he has published over 170 peer-reviewed papers with landmark papers and preclinical proof-of-concept (POC) studies for many different ocular disorders. As chief investigator, he established the world’s first clinical trial of gene therapy for retinopathy. The results from this trial reporting an improvement in vision (NEJM, 2008; NEJM 2015) along with results from two other trials, established POC for gene therapy for inherited retinal degeneration. His group has also provided the first POC for effective transplantation of photoreceptors (Nature 2006; Nature, 2012) that has provided the basis for ES cell-derived photoreceptor transplantation, now a major programme in his laboratory (Nature Biotech 2013). Robin Ali and members of his team have received numerous prizes and awards for their work on developing new treatments for retinal degeneration, including the Pfizer/ARVO Translational Award for Ophthalmology (2010), the Alcon Research Institute Award (2009) and in 2014, Human Gene Therapy journal’s Pioneer Award (to honour the top 12 pioneers in gene therapy). In 2007 he was elected to the Academy of Medical Sciences as its youngest elected member and in 2009 elected NIHR Senior Investigator (re-elected in 2014). He is currently President of the European Society of Gene and Cell Therapy and has served on the advisory boards of a number of funding bodies including the UK Medical Research Council (Neurosciences and Mental Health Board and the Regenerative Medicine Research Committee), Research to Prevent Blindness USA and Fighting Blindness Ireland, Telethon, Italy, as well as advisory boards of several pharmaceutical and biotech companies, including Alcon/Novartis. 


Robin Ali obtained a degree in Genetics from UCL in 1988 and his PhD from UCL in 1993 working on developing transgenic mouse models. After a postdoc at the MRC National institute for Medical Research he moved to UCL Institute of Ophthalmology establishing his own laboratory there in 1999

Publications