Participants at the 10th BIC Conference.

To keep abreast with the latest scientific and medical updates on novel technologies in haemophilia and inhibitor treatment, EHC staff and steering committee members attended in September two major scientific conferences.

The first event was two joint conferences: the 10th edition of the BIC International Conference and the third edition of the International Conference on Inhibitors in Haemophilia. Both events were chaired by Prof Flora Peyvandi, who is also the Chair of the EHC Medical Advisory Group (MAG). The events were held in Genoa, Italy ahead of the 2020 Congress of the International Society for Thrombosis and Haemostasis (ISTH) taking place in Milan and also chaired by Prof Peyvandi. Mrs Amanda Bok, EHC CEO and Mrs Kristine Jansone, EHC Community Programmes Officer attended this meeting.

The second event was the Workshop on Novel and Gene Therapies organised by the National Hemophilia Foundation (NHF) in Washington DC, United States. Mr Brian O’Mahony, EHC President, Dr Radoslaw Kaczmarek, EHC Steering Committee Member and Mr Declan Noone, EHC PARTNERS Programme Consultant, attended this event.

We will report in detail the main findings from both meetings in our next issue of the ‘Novel treatments in haemophilia and other bleeding disorders: A periodic EHC review,’ to be released next week ahead of the EHC Conference. For now, we would like to inform our members that during these meetings we received, amongst others, updates on the developments of first-generation gene therapy programmes. These updates looked at how long gene therapy may last, which candidates may have a better response to the treatment, and, how can we also treat those who may not be eligible due to pre-existing antibodies to the gene therapy vector. During the meetings, there was also a glimpse into the future of the next generation of gene therapy that could use gene editing, different modes of delivery and different cells for gene production.
The meetings also included updates on other novel non-replacement treatments looking at rebalancing haemostasis. These treatments are currently in the clinical trial and could be an alternative to treating people affected by rarer bleeding disorders.

Panel discussions at the NHF Workshop on Novel and Gene Therapies. Mr Declan Noone is second from the left.