Update 2020

CEO Update | June 19, 2020

WFH Virtual Summit

The WFH Virtual summit has been taking place on-line every day since last Sunday and finishes today. This meeting is taking place in lieu of the WFH International Congress which was due to take place this week in Malaysia. I greatly appreciate that registration is free of charge. It has provided an opportunity for people with haemophilia and family members who normally would never have an opportunity to attend a world congress to attend free of charge. I know that several members of the Society in addition to members of the Board and staff have been logging in daily. There have been some very impressive speakers and topics covered. The Society will be producing a special newsletter supplement after the Summit covering most of the programme. Some of the highlights for me included new data on the ongoing FVIII and FIX Gene Therapy trials, updates on novel technologies leading toward an eventual cure, the session on Low Dose Prophylaxis in developing countries and the overview session on von Willebrands. It was also very encouraging to see Sobi and Sanofi /Genzyme commit to a further donation of 500 million IU of Elocta and Alprolix to the WFH Humanitarian Aid programme which will help thousands of people with haemophilia over the next 5 years. Significant donations were also made by Roche (of Hemlibra) and by Bayer, CSL Behring and Grifols (of clotting factor concentrates).

New Gene Therapy data was presented on the Biomarin FVIII Gene Therapy showing continued factor expression after 4 years with some fall off in the factor level. This Gene Therapy is expected to be licenced in the USA later this year and in Europe in early 2021. New data was also presented on the Uniqure FIX Gene Therapy showing stable expression after 1 year in 3 people in the dose finding study. There were impressive results from low dose prophylaxis regimens in India, Indonesia, China, Tunisia and Thailand. I saw at first hand the benefits of this regime compared to on- demand treatment when I visited Pakistan last year. This is a very productive use of humanitarian aid from WFH.

The Conference also gave updates on von Willebrands and on women with von Willebrands. Ireland is a global leader in the treatment of and research on low von Willebrand levels in women and it was good to see Dr. Michelle Lavin speak at the summit. An excellent 2 hour session on new Novel Therapies gave an update on the wide range of novel approaches being taken to treat haemophilia in the future. This ranged from those in advanced clinical trials such as a new extended half-life FVIII which should allow prophylaxis once a week with a high trough level to updates on other gene therapy clinical trials. It also featured novel approaches including gut implantable spheres which could be used to dispense FVIII, FIX or FVII as required to a non-viral vector based durable and re-dosable Gene Therapy using lipid nano particles in place of an AAV virus vector delivery system. It included advances in Gene Editing which could be applicable to children as well as adults. Our hope is that many, if not all, of these approaches come to fruition.

Zoom Meetings

This morning, Friday, June 19th, we hosted an excellent Zoom lecture from Prof. Mike Makris from Sheffield who spoke on Haemophilia Care in the year 2030. He covered developing in therapy, his view of the future of treatment centres and te3lemedicine. It was a very enjoyable Zoom meeting for the 39 participants.