The National Bleeding Disorders Foundation (NBDF) is pleased to announce the availability of a series of video presentations on fundamental topics relevant to current gene therapy for hemophilia.

These talks were originally delivered live and in-person as part of NBDF’s 75th Bleeding Disorders Conference – Medical Track, which was held in August 2023, in National Harbor, Maryland. Due to the overwhelming onsite participation and follow-up interest in the subject expressed by health care providers and the greater bleeding disorder community, a few of the presentations were subsequently recorded, and made accessible on the NBDF website.

In Gene Therapy for Hemophilia: Progress and Conundrums Dr. Radek Kaczmarek, with the Indiana University School of Medicine, lays out the scientific advances underpinning two currently approved AAV-based gene therapies for hemophilia A and B, including product differences, challenges regarding patient eligibility, long-term efficacy concerns, and potential safety matters such as vector integration into the host genome. Dr. Kaczmarek also asserts that current and future efforts in the gene therapy space need to continually be informed by robust data collection, greater inclusivity, and transparency.

In The Science Required to Cure Hemophilia Dr. Glenn Pierce explains the scientific and technological advances of the past 20 years that have yielded safer and effective hemophilia therapies, leading up to the recent gene therapy approvals in the U.S. and abroad. His presentation highlights the early challenges with AAV vectors, tackles what remains unknown about gene therapy safety in the long term and provides a context for whether these therapies could be considered  “one and done cures” or not. Dr. Pierce also shares valuable progress updates on a few other non-viral approaches to gene therapy including lipid nanoparticle-based delivery and gene editing techniques, which are currently being explored in preclinical research.

In Impact of Gene Therapy for Hemophilia on Care Delivery in the Hemophilia Treatment Center Dr. Ulrike M. Reiss of St. Jude Children’s Research Hospital describes the many practical considerations for fully incorporating gene therapy within the collaborative care framework of a hemophilia treatment center. Some of these considerations include increased infrastructural capacity to be able to provide targeted patient education; added collaboration needed among internal and external MD specialists; more time spent on patient eligibility screening; better coordination of laboratory/diagnostic testing, and reimbursement planning. Dr. Reiss’ talk offers an insight into the preparedness, treatment, and monitoring involved in administering hemophilia gene therapies to patients.

Please note that these presentations were designed to educate physicians, and other medical prescribers and therefore contain a high volume of technical language, scientific terminology, and references.

Visit Online Education for Providers to watch additional educational videos for healthcare providers.