Congress medical program to feature prominent speakers

The World Federation of Hemophilia (WFH) 2020 World Congress will feature some of the biggest names in both bleeding disorders and gene therapy. A number of key opinions leaders will be there to share their insights on the medical developments that will impact patients and treaters in 2020 and beyond.

Here are four short profiles of notable speakers who will be taking part in the WFH 2020 World Congress in Kuala Lumpur, Malaysia, from June 14 to 17, 2020.

Name: Luigi Naldini, MD, Ph.D 

Country: Italy

Positions: Director, San Raffaele Telethon Institute for Gene Therapy; Head, Gene Transfer Technologies and New Gene Therapy Strategies Unit; Full professor, Università Vita-Salute San Raffaele, Italy

WFH 2020 Congress speaker topic: Non-Adeno-Associated Virus (AAV) platforms

Bio: Luigi Naldini pioneered the development and application of lentiviral vectors for gene transfer, which have become some of the most widely used tools in biomedical research, offering hope for a cure for several currently untreatable and deadly human diseases. He has investigated new strategies to overcome the major hurdles to safe and effective gene transfer, and has translated these strategies into new therapeutic approaches for genetic disease and cancer. This has allowed for new insights into hematopoietic stem cell function, the induction of immunological tolerance, and tumour angiogenesis. His work has also contributed to advancing the use of artificial nucleases for targeted genome editing in cell and gene therapy.

Name: Mark A. Kay, MD, Ph.D

Country: U.S.A

Positions: Dennis Farrey Family Professor in Pediatrics; Professor of Genetics, Stanford University, U.S.A

WFH 2020 Congress speaker topic: Next generation Adeno-Associated Virus (AAV) platforms

Bio: Mark Kay’s hope for gene therapy is that gene transfer technologies can be developed for hepatic gene therapy for the treatment of genetic and acquired diseases. His team’s general approach to research is to develop new vector systems and delivery methods, test them in the appropriate animal models, uncover the mechanisms involved in vector transduction, and use the most promising approaches in clinical trials. His team’s research focuses on a variety of viral and non-viral vector systems. The major disease models being looked at as part of his research are hemophilia, hepatitis C and B viral infections, and diabetes. His team is also looking at the role that small RNAs play in mammalian gene regulation.

Name: Matthew Porteus

Country: U.S.A

Positions: Professor of Pediatrics (Stem Cell Transplantation), Stanford University, U.S.A.

WFH 2020 Congress speaker topic: Cell-based therapies and gene editing

Bio: Matthew Porteus was an intern and resident in pediatrics at Boston Children’s Hospital before completing his pediatric hematology/oncology fellowship in the combined Boston Children’s Hospital/Dana Farber Cancer Institute program. Porteus was the first to demonstrate that gene correction could be achieved in human cells at frequencies that were high enough to potentially cure patients. He is considered one of the pioneers and founders of the field of genome editing—a field that now includes thousands of labs and several companies around the world. His research program continues to focus on developing genome editing by homologous recombination as a curative therapy for children with genetic diseases. Porteus also has an interest in the clonal dynamics of heterogeneous populations and the use of genome editing to better understand diseases that affect children, including infant leukemia and genetic diseases that affect muscles.

Name: Ton Lisman

Country: The Netherlands

Positions: Full Professor of Experimental Surgery, Surgical Research Laboratory, Department of Surgery, University Medical Center Groningen, The Netherlands

WFH 2020 Congress speaker topic: Acquired bleeding disorders: liver disease

Bio: Ton Lisman is one of the few clinical and fundamental research experts on bleeding and thrombosis in patients with liver diseases. In the past decade—through a combination of careful analysis of the clinical phenotype of patients and in-depth laboratory analyses—his team has been able to redefine the clinical consequences of hemostatic changes associated with liver diseases. In particular, they demonstrated that patients with chronic and acute liver diseases are in a hemostatic “rebalance” due to a concomitant decline in pro- and anti-hemostatic forces. These novel insights have had profound consequences for clinical management, and have led to world-wide changes in the approach to treatment and prevention of bleeding in patients with liver diseases.

In the coming months we will be releasing more information about the WFH 2020 World Congress program. We hope you visit Hemophilia World Online to find out more!

WFH 2020 World Congress in Kuala Lumpur

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