This year’s BSGCT Annual conference was held at University College London, Institute of Child Health on Friday 15th April and organised as a one day conference with a focus on adeno-associated virus (AAV) vector design and innovative approaches to implement the technology into human gene therapy.
It was an excellent opportunity to be able to attend this conference as I have taken advanced courses in Gene and Nucleic Acid based Therapies as part of my MSc in Genes Drugs and Stem Cells at Imperial College this academic year. The meeting therefore reinforced my knowledge of AAV gene therapy whilst learning about the cutting edge advances in this field of novel therapeutics. AAV gene therapy is currently limited by its small transgene insert capacity of 4.6Kb and its immunogenicity in the host. However, AAV are able to transduce quiescent cells and remain episomally stable in the host cell. This means AAV gene therapy has a wide range of clinical applications in particular for the central nervous system, including treatment of chorideremia by targeting retinal host cells, as well as the cardiovascular system in targeting cardiomyocytes in heart failure patients.
The BSGCT conference was structured into three sessions broadly divided into AAV vector technology and immunological challenges, pre-clinical advances and future visions for AAV therapies. I found it most interesting to learn about the vision for AAV therapies, which was a session chaired by Professor Andy Baker. As part of this session, Professor Robert MacLaren discussed that in order to advance clinical gene therapy for choroideremia, it is vital to optimise delivery, purity and efficacy of AAV gene therapy. Professor MacLaren also introduced Nightstar which is a private pharmaceutical spin-out company from the University of Oxford that is backed by the Wellcome trust to drive phase II clinical trials across 15 centres and 7 countries for patients with inherited retinal dystrophies.
The Fairbairn prize was awarded, in memory of Les Fairbairn, to a PhD student for the best presentation. The prize was awarded to Dr Joanne Ng from UCL for her talk and response to questions from the panel on her presentation entitled Novel therapeutic approaches for childhood parkinsonism.
Following the sessions, Professor Uta Griesenbach chaired the keynote presentation by Professor David Schaffer from University Berkeley. The talk was focused on engineering the AAV capsid to optimise its therapeutic efficacy. I was very intrigued by the talk, which led me to ask Professor Schaffer the extent to which the epigenetic landscape of the target cell is a determinant of the success of AAV therapy. The talk was altogether very engaging and I found it a great opportunity to ask a question at the conference.
During the conference, there was an opportunity to view posters and meet delegates in the field of cell and gene therapy. I was particularly honoured to meet Professor Charles Coutelle, who is an Emeritus professor of Gene therapy at the NHLI and contributed to the first clinical gene therapy trial for cystic fibrosis with non-viral vectors in 1992. I was interested to gain Professor Coutelle’s perspective on the progress of cystic fibrosis gene therapy and the outlook for the future, discussing specifically whether genome editing or gene therapy may be the most suitable and promising approach, or a combination of the two.
Attending the BSGCT Annual conference as a postgraduate student was altogether an invaluable experience and I would encourage fellow students at Imperial College to also attend wherever possible to broaden their subject knowledge of their research field of interest and also to engage with the scientific community.
Mozhgon Jeddi is an MSc Genes Drugs and Stem Cells – Novel Therapies student at National Heart and Lung Institute, Imperial College London, UK.
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