The World Federation of Hemophilia Gene Therapy Registry – Informational Webinar
Listen to this 45-minute webinar presented on 9 November 2020 to learn more about the World Federation of Hemophilia (WFH) Gene Therapy Registry (GTR). Barbara Konkle, MD (WFH Board of Directors), Glenn Pierce, MD, PhD (WFH VP Medical) and Wolfgang Miesbach, MD (GTR Steering Committee member) discuss the protocol and core data set, the implementation […]
Gene Therapy for Hemophilia
Adapted from: Global NMO Training 2018 plenary presentation by Glenn Pierce Reviewed by: Glenn Pierce Edited by: Georghia Michael The learner is strongly advised to complete Module 1 Introduction to Bleeding Disorders and Module 2 Evolution of Treatment Products, in the WFH Treatment Products eLearning Program before embarking upon Module 3. This third module in […]
Investigational SPK-9001: AAV-Mediated Gene Transfer for Hemophilia B – Persistent, Stable Factor IX Activity at One Year Independent of Downstream Purification Method
In the Tuesday morning session entitled Free Papers – Gene Therapy, Spencer Sullivan presents clinical results of FIX gene transfer using in vivo recombinant AAV delivery with the novel bioengineered vector SPA-9001, discussing vector design, FIX activity attained, and safety.
Gene Therapy for Haemophilia: What, Why, How, When, Who, and Where?
In the Sunday afternoon session entitled Gene Therapy: Back to Basics, Glenn Pierce explains the state of the art and the future prospects of gene therapy, gene editing, and cell therapy, in the context of hemophilia.
Updates on Clinical Trials of Gene Therapy in Hemophilia
The fifth and final activity in the Clinical Advances in Gene Therapy for Hemophilia CME curriculum, Updates on Clinical Trials of Gene Therapy in Hemophilia, is a clinical article. Follow Thierry VandenDreissche (Brussels, Belgium) as he takes a deeper dive into the advances made during the recent early phase gene therapy clinical trials in hemophilia […]
Why Gene Therapy for Hemophilia? Exploring the Challenges of Current Therapies
Re-certified for CME credits, to Sept 6, 2020! The fourth activity in the Clinical Advances in Gene Therapy for Hemophilia CME curriculum, Why Gene Therapy for Hemophilia? Exploring the Challenges of Current Therapies, is an expert video exchange on gene therapy in the context of therapies currently available for hemophilia around the world. Watch as […]
The Science of Gene Therapy for Hemophilia
Re-certified for CME credits, to Sept 6, 2020! The third activity in the Clinical Advances in Gene Therapy for Hemophilia CME curriculum, The Science of Gene Therapy for Hemophilia, is an expert video commentary on the vector technology behind gene therapy in hemophilia. Watch as Glenn Pierce, WFH Vice President Medical, examines early clinical trials, […]
Gene Therapy: Past, Present, and Future
The second activity in the Clinical Advances in Gene Therapy for Hemophilia CME curriculum, Gene Therapy: Past, Present, and Future, is a round table video discussion of the evolution of gene therapy in hemophilia. Watch three experts reflect on the development, ongoing research, and current and future uses of gene therapy in hemophilia. A transcript […]
Test Your Knowledge of Gene Therapy in Hemophilia
The first activity in the Clinical Advances in Gene Therapy for Hemophilia CME curriculum, Test Your Knowledge of Gene Therapy in Hemophilia, is an educational self-assessment survey. The questions allow you to see how much you already know about gene therapy in hemophilia and the answers explain the essential information required for a fuller understanding. […]
Stem Cell Gene Therapy of Hemophilia A
In the Tuesday morning session on gene therapy, Christopher B. Doering presents advances in stem cell gene therapy of hemophilia A.
Hemophilia A Gene Therapy: AAV-mediated delivery of an enhanced F8 cassette to the liver produces supraphysiological levels of human FVIII in vivo
In the Tuesday morning session on gene therapy, Brigit E. Riley explains her approach to the AAV-mediated delivery of an enhanced F8 cDNA.