Today, the World Federation of Hemophilia (WFH) marked 30 years of scientific innovation with the start of the 19th New Technology and Gene Therapy (NTGT) Workshop in Montreal, Canada. Experts from around the world are in the city on November 12 to 13, 2026 to explore the latest developments in hemophilia treatment and research.
Here is a summary of what happened at the 19th New Technology and Gene Therapy (NTGT) Workshop today, November 12, 2026.
Session 1 – Welcome and “New Protein Technologies”
The workshop opened with a look back at 30 years of innovation, from viral vectors and factor proteins to non-factor therapies and gene therapy. Speakers noted that current treatments greatly reduce bleeding but do not fully eliminate it. New factor VIII mimetics and rebalancing agents continue to improve protection, while gene therapy remains more predictable in hemophilia B than hemophilia A. Discussion focused on safety, thrombosis risk, treatment switching and how bleeding outcomes should be measured.
Session 2 – “Curative AAV Gene Therapies”
This session reviewed progress and remaining challenges in adeno-associated virus (AAV) gene therapy. Hemophilia B programs have shown durable expression, while factor VIII gene therapy remains more variable, with declining expression and liver-related concerns. Speakers explored the biology behind these differences, vector composition, immune responses and potential genotoxicity. Discussion emphasized the importance of improving vector design, understanding long-term expression and increasing transparency around results from discontinued gene therapy programs.
Session 3 – “Other delivery systems”
Speakers explored alternative gene-delivery technologies designed to improve targeting, safety, durability and the potential for redosing. Approaches included retrotransposon-based gene insertion, targeted lipid nanoparticles, plasmid electroporation, ultrasound-activated microbubbles, synthetic capsids and engineered AAV variants. Discussion focused on how these platforms reach different cell types, how long expression may last and how safety can be improved compared with conventional AAV-based approaches.
Session 4 – “Gene Editing”
The Gene Editing session reviewed CRISPR-based technologies, including nucleases, base editing and prime editing, and their potential applications in bleeding disorders. Examples included personalized editing approaches, hemophilia A therapies and correction of von Willebrand disease in preclinical models. Speakers also addressed unintended genetic changes, off-target risks, pediatric considerations and evolving regulatory guidance, while expressing cautious optimism about the potential for durable and scalable gene-editing therapies.
To find out more about the 19th New Technology and Gene Therapy (NTGT) Workshop, click here.
