Advances in Gene Therapy for Hemophilia

Key Considerations:
Advances in Gene Therapy for Hemophilia

Recorded Live as a Friday Satellite Symposium preceding the 62nd ASH Annual Meeting and Exposition

Adeno- Associated Viral Vectors (AAV)

Lindsey George, MD

AAV Expression Cassette

Lindsey George, MD

AAV Gene Transfer for Hemophilia

Lindsey George, MD

Short Term Safety

Lindsey George, MD

Durability of Expression

Lindsey George, MD

Optimization of Transgene

Barbara A. Konkle, MD

FIX Padua Variant

Barbara A. Konkle, MD

SPK-9001

Barbara A. Konkle, MD

Current AAV Hemophilia B Gene Therapy Trials

Barbara A. Konkle, MD

AAV5-FVIII

Glenn F. Pierce, MD, PhD

BMN 270- 4-Year Data

Glenn F. Pierce, MD, PhD

Ongoing Hemophilia A Gene Therapy Trials

Glenn F. Pierce, MD, PhD

Giroctocogene Fitelparvovec Phase 1/2 Trial

Glenn F. Pierce, MD, PhD

SPK8011 AAV-FVIII Phase 1/2 Trial

Glenn F. Pierce, MD, PhD

BAY 2599023 Phase 1/2 Trial

Glenn F. Pierce, MD, PhD

AAV Path Toward FVIII Expression

Glenn F. Pierce, MD, PhD

Live Biopsies From AAV-FVIII

Glenn F. Pierce, MD, PhD

Canine Studies of AAV-Mediated Gene Transfer

Glenn F. Pierce, MD, PhD

Panel Discussion: Presenting the Option of Gene Therapy to a Patient

Glenn F. Pierce, MD, PhD, Barbara A. Konkle, MD, Lindsey George, MD, Alfonso Iorio, MD

Gene therapy holds much promise for individuals with a wide variety of conditions, including types of cancers, AIDS, diabetes, heart disease, and hemophilia. This innovative approach to treatment alters genes inside the body’s cells to stop the disease. 

Gene therapy attempts to replace or fix mutated genes and make diseased cells more evident to the immune system. Hemophilia A and B are inherited as part of an X-linked recessive pattern. Hemophilia genetics involve genes found in the X chromosome, and it takes just one faulty gene to lead to this bleeding disorder. 

Cell and gene therapies are still under study, but the results are promising. Clinical trials have shown success, and the Food and Drug Administration (FDA) has approved one type of cell therapy. In our Key Considerations: Advances in Gene Therapy for Hemophilia led by Glenn F. Pierce, we discuss an overview of the disease, the current state of gene therapy, and more.