VTX-PID as a novel recombinant immunoglobulin G–degrading enzyme (IdeS) for efficient AAV-based gene therapy in participants with neutralizing antibodies: results of the phase I first-in-human NAVIgATE study
Breaking barriers in AAV gene therapy : The NAb challenge has a solution for some patients
For too long, we’ve accepted that 30–60% of gene therapy–naïve patients are automatically excluded from AAV-based treatments due to pre-existing neutralizing antibodies (NAbs). That paradigm is shifting.
The first dedicated clinical trial in AAV3B NAb-positive subjects demonstrates that with VTX-PID (imlifidase), we could now treat patients previously left behind. But this isn’t just about eligibility. It’s about precision
These data deliver a strategic decision-making framework for the gene therapy field:
- Align patient selection and NAb thresholds with predictable, positive outcomes – We presented some data during American Society of Gene & Cell Therapy with our poster.
- De-risk clinical programs by modeling response probability upfront
- Maximize portfolio value by expanding addressable patient populations
The patient impact is profound: VTX-PID modeling based on our NAVIgATE clinical data enables us to identify who will respond and spare those who won’t from unnecessary intervention. This is what patient-centric drug development looks like in practice.
We are moving from a “one-size-fits-all” approach to precision patient selection in gene therapy. The question is no longer if we can overcome NAbs, it’s how we implement this strategically across the AAV landscape.
The future of systemic gene therapy depends on solving access barriers with the same rigor we apply to the science itself. This is how we unlock the full potential of transformative therapies.
Thanks to Vivet Therapeutics team for such achievement.
The full article here: https://www.frontiersin.org/journals/immunology/articles/10.3389/fimmu.2026.1824802/full
