More than 600 colleagues and friends signed up for the promising developments in the treatment of the rare bleeding disorder hemophilia during the second WuXi AppTec webinar series “Collaborations That Transform”. The event aired on Thursday, July 16th and featured a fireside chat with Glenn Pierce of the World Hemophilia Federation and a panel discussion which brought together other key opinion leaders, industry experts, patients and patient advocates.
“I think transformative treatments coming down the line can almost give people that absolute normality of life which is something that we truly believe is in our grasp for the first time and forever,” noted John Pasi, one of the panelists and Professor of Haemostasis and Thrombosis at Barts and London School of Medicine and Dentistry, Queen Mary University of London.
It is estimated the disease affects 400,000 patients globally and only within the last several years treatments options are becoming available to treat the pediatric and adult hemophiliac communities with the anticipation of gene therapy approval on the horizon. With a variety of clotting factor approaches and the rise of non-factor therapies, patients will have an opportunity for a more normal life. While there are still a number of key issues to be resolved including immune responses of gene therapy and accessibility to all, the promise of these treatments offers hope and confidence that hemophilia is a condition with prospects of a cure in sight.
Thank you to our partners, BioCentury, Children’s Tumor Foundation, CLSA, Informa, New England Hemophilia Association and UK Bioindustry Association for their great support, and to everyone who participated and tuned in. We invite you to be on the lookout for our next “Collaborations That Transform” webinar series on the topic of the rare disease spinal muscular atrophy (SMA), to be aired on August 20th, 2020. We also welcome you to watch our previous webinar on neurofibromatosis, and follow us on Twitter to stay up-to-date on upcoming news and events.