Gene therapy

Location: Pacifico

CE: 1

Chair(s)

Len Valentino, MD (he/him/his)

CEO
National Hemophilia Foundation
New York City, United States

Speaker(s)

Barbara Konkle, MD (she/her/hers)

Professor of Medicine
Washington Center for Bleeding Disorders
University of Washington
Seattle, United States
Glenn Pierce

Vice-President, Medical
World Federation of Hemophilia
La Jolla, United States
Denise Sabatino, PhD (she/her/hers)

Research Associate Professor
Depart of Pediatrics
The Children's Hospital of Philadelphia
Philadelphia, United States
Benjamin Samelson-Jones, MD, PhD (he/him/his)

Assistant Professor
Pediatrics
Division of Hematology, Children's Hospital of Philadelphia
Philadelphia, United States

Learning Objectives:

Contrasting gene therapy for hemophilia A/B : The goal of hemophilia gene therapy is to safely provide durable factor expression at a level sufficient to prevent bleeding in all recipients. For hemophilia B, current gene therapy vectors using the hyperactive factor IX variant R338L are close to achieving this goal. For hemophilia A, unique features of factor VIII biology limit current gene therapy approaches from reaching this target.  
HTC preparedness for gene therapy and registry planning: With gene therapy products for hemophilia coming to market, HTCs need to be prepared to help patients decide if gene therapy is right for them.  This optimally involves shared decision making between the patient and HTC providers.  In addition, we need to be ready to monitor patients who receive gene therapy for ongoing safety and efficacy and the WFH Gene Therapy Registry can fulfill this need.
Next generation transgenes for hemophilia A: Current gene therapy approaches for hemophilia A deliver a wild type B-domain deleted factor VIII transgene.  Novel factor VIII transgenes that express a protein that has increased secretion and/or higher activity provide a strategy for improving this approach in next generation AAV vectors for hemophilia A.
Next generation vectors for hemophilia A/B: Adeno-associated virus (AAV) remains the mainstay in delivering Factor VIII and IX genes to persons with hemophilia, but requires high doses that can cause toxicities. Many research efforts are underway to modify the viral coat (capsid) proteins to decrease off-target effects, increase liver tropism, and decrease immunogenicity. Ultimately next generation engineering of more efficient AAV capsids will yield improved gene therapeutics for hemophilia and other diseases.

Presentations:

14:00 - 14:01 ART

Chair
Chair: Len Valentino, MD (he/him/his) – National Hemophilia Foundation
14:01 - 14:12 ART

Contrasting gene therapy for hemophilia A/B
Speaker: Benjamin Samelson-Jones, MD, PhD (he/him/his) – Division of Hematology, Children's Hospital of Philadelphia
14:12 - 14:24 ART

HTC preparedness for gene therapy and registry planning
Speaker: Barbara Konkle, MD (she/her/hers) – University of Washington
14:24 - 14:36 ART

Next generation transgenes for hemophilia A
Speaker: Denise Sabatino, PhD (she/her/hers) – The Children's Hospital of Philadelphia
14:36 - 14:48 ART

Next generation vectors for hemophilia A/B
Speaker: Glenn Pierce – World Federation of Hemophilia
14:48 - 15:00 ART

Discussion
Chair: Len Valentino, MD (he/him/his) – National Hemophilia Foundation