In June, the British Society for Gene and Cell Therapy (BSGCT) was held at the Royal Holloway, University of London in Egham, Surrey. The Grade 1 listed Founder’s Building provided a fabulous backdrop for the meeting, comprising of a full day of talks including from the Outstanding Achievement Award Winner, Adrian Thrasher, providing some personal reflections on his career developing gene therapies for rare diseases.

As well as the wide range of talks from use of stem cells in clinical trials to use of CRISPR activation for Duchenne Muscular Dystrophy, and preclinical development of antisense oligonucleotides, the poster session provided further dissemination of research. ARDAT was represented by researchers in both the poster and oral presentation sessions.

Luisa Hering, University of Liverpool

ARDAT’s Luisa Hering, who is completing her PhD at the University of Liverpool, presented her poster “Ex vivo Investigation of AAV Vector-Specific Adaptive Immunogenicity” described her work on determining the percentage of individuals who have an immune response to AAV2 and AAV9 in a healthy donor cohort, as well as to AAV2 in a smaller cohort of individuals treated with Zolgensma. In addition, PBMCs from participants were exposed to AAV2 and AAV9 peptides to establish those cytokines that were increased following exposure. Overall, Luisa really enjoyed the entire event and particularly talking about her research with other colleagues in the field.

Ouidad Khechaoui, University of Sheffield

Amongst the presenters giving a talk was Ouidad Khechaoui, a PhD in her final year from the University of Sheffield, who is supervised by ARDAT members Prof Mimoun Azzouz and Prof Janine Kirby, as well as Prof Guillaume Hautbergue. Ouidad gave an excellent presentation describing her work on a dual AAV9 gene therapy strategy for the treatment of a rare autosomal dominant neurodevelopmental disorder caused by mutation of the SYNGAP1 gene. Ouidad received an award for her talk, the ESGCT Oral Presentation Winner, so an enjoyable and successful day for our ARDAT researchers.