4th Gene Therapy Round Table continues conversation on new challenges and opportunities


The WFH Gene Therapy Round Table (GTRT) series began in 2018, and is designed to bring WFH national member organizations (NMOs), patient advocates, health care professionals, regulators and industry representatives together to discuss current global developments and expected challenges related to gene therapy for hemophilia. The second webinar of the 4th WFH GTRT webinar series was held on June 10, 2021 and featured a number of experts discussing this important field of development for the bleeding disorders community.

The first session of this webinar focused on how to interpret emerging data. Radoslaw Kaczmarek (U.S.A) and David Lillicrap, MD, (Canada) discussed the current challenges with assays used in the assessment of gene therapy for hemophilia and offered suggestions on how to improve them. The presentations were followed by a panel of six healthcare professionals and people with hemophilia that covered how to interpret data from clinical trials to help people with hemophilia (PWH) make an informed decision.

The second session focused on the future of gene therapy for hemophilia. Steven Pipe, MD (U.S.A), Wolfgang Miesbach, MD (Germany), and Margareth Ozelo, MD (Brazil), each presented their perspectives on the organization and delivery of care for gene therapy for hemophilia. The last speaker—Robert Peters, MD—discussed the possibility of applying different gene editing techniques to hemophilia.

To find out more about the WFH Gene Therapy Round Table (GTRT) series, click here. The next WFH Gene Therapy Round Table webinar will take place in September 2021.