Here is a summary of what happened at the 19th New Technology and Gene Therapy (NTGT) Workshop today, November 12, 2026.
Session 5 – “Improving transgenes”
Speakers presented ways to make liver-directed adeno-associated viruses (AAV) gene therapy for hemophilia A more durable, including factor VIII constructs that reduce ER stress and improve secretion, achieving sustained expression at lower vector doses. Other approaches included a SUMOylation inhibitor that boosted transgene expression and a factor IX Padua-albumin fusion with extended half-life. A discussion weighed these strategies against long-term safety, noting that models linking ER-stress pathways to liver cancer may be far more extreme than clinical AAV exposure.
Session 6 – “Novel therapies for other bleeding disorders”
Experts reviewed preclinical and clinical strategies beyond hemophilia, including AAV-delivered factor VIII-mimetic antibodies, an anti-protein S antibody for von Willebrand disease (VWD), and GalNAc-siRNA approaches targeting plasminogen and ZPI. A discussion addressed applicability across other bleeding disorders and regulatory benchmarks for bleeding reduction.
Session 7 – “Individual perspectives on new technologies and gene therapies”
Panelists agreed that a “hemophilia-free mind” is nearly within reach in many high-income countries but remains distant elsewhere, and that this vision must also include women and girls, moderate patients, and those without access to broader care. A discussion highlighted the need for shared decision-making, better collection of real-world evidence on treated patients, and dedicated bleeding outcome measures for women.
Session 8 – “Reaching the rest of the world”
The closing session examined how most of the world’s population is repeatedly left behind as new therapies emerge. Speakers noted that the costs associated with developing and manufacturing new products can influence their final pricing, and proposed new pricing models as a way forward. Experiences from China and India showed how lower-cost gene therapy programs can progress, while speakers stressed that affordability must be matched by in-country infrastructure, registration, and pharmacy systems.
To find out more about the 19th New Technology and Gene Therapy (NTGT) Workshop, click here.
