Event Summary: ASGCT 2026 Annual Meeting

Founders Award: Development of AAV vectors, a journey approaching 50 years: Where are we now?

Jude Samulski, Alumni Distinguished Professor, UNC Gene Therapy Center

Jude Samulski’s Founders Award acceptance was a characteristically direct reflection on nearly five decades of AAV biology, and a candid assessment of where the field still has work to do. His central message: only 3% of AAV9 crosses the blood–brain barrier, and that figure demands a rethink. Samulski argued for reconsideration across three areas: delivery routes (including the possibility of split dosing, with part of the dose delivered directly to the brain and part systemically), dose size through next-generation transgene design, and the use of investigator-initiated trials in China to accelerate novel capsid evaluation.

He closed with a striking example: a gene therapy program that moved from mouse data to a treated patient in just 11 months. After nearly 50 years in the field, Samulski’s conviction that there is still significant headroom for innovation felt energising and hopeful.

Keynote: Designing what nature can’t: Cell and gene therapy for HIV

Paula Cannon, Keck School of Medicine, University of Southern California

Paula Cannon’s keynote was one of the meeting’s highlights. Her framing of HIV as a uniquely formidable challenge was pointed: four decades of intensive research have not produced a vaccine or cure, because HIV, as she put it, “read the immunology textbooks.” Its mutation rate, genomic integration, persistence under drug therapy, and targeted disruption of CD4² T cells have made it a problem that conventional immunity cannot solve.

Cannon’s argument was that engineering immunity, rather than trying to elicit it, may be the path forward. Her group’s work spans hematopoietic stem cell modification through CCR5 disruption and, more recently, reprogramming B cells to produce broadly neutralising antibodies that neither infection nor vaccination can generate. It was a compelling case for why HIV continues to be one of the field’s most productive drivers of innovation.

I also had the privilege of speaking with Dr Cannon at the meeting – look out for an interview with her coming soon in Cell & Gene Therapy Insights.

Journey to Cure Michael – From Hope to FDA Approval

Terry Pirovolakis, co-founder, CureSPG50 / Elpida Therapeutics

If there was one session that stayed with me after leaving Boston, it was this one. Terry Pirovolakis confronted his son Michael’s diagnosis of SPG50, an ultra-rare neurodegenerative disease, in 2019. Rather than accept the timelines the field typically operates on, he co-founded CureSPG50, raised the funds, drove the science, and helped get three children treated within three years. He has since established Elpida Therapeutics, structured as a corporation with a nonprofit ethos, reinvesting profits to keep rare disease programs viable. The portfolio now spans SPG50, CMT4J, and CLN7, with three additional diseases targeted for 2025/2026.

It is easy, in a meeting full of platform optimisation and regulatory strategy discussions, to lose sight of what is driving the urgency. Hearing directly from a parent who refused to accept that the development timeline couldn’t be compressed was a powerful reminder. The level of energy that families facing rare pediatric disease can bring to this work is extraordinary, and the results speak for themselves.

Top abstract: ETX101 (AAV9) for Dravet Syndrome

Encoded Therapeutics

Among the abstracts presented at this year’s meeting, the data from Encoded Therapeutics on ETX101 for Dravet syndrome was, for me, the standout. The program reported an 83.5% reduction in seizures at high doses, with near-typical cognitive development observed at Week 16, representing the first clinical proof of cell-type-selective AAV gene regulation. For a condition as devastating and difficult to treat as Dravet syndrome, these early data are genuinely exciting, and this is a program worth following closely.

Closing reflections

Beyond the scientific program, ASGCT 2026 reinforced something I think we already suspected at Cell & Gene Therapy Insights: the community wants content that engages honestly with where things are still hard. Manufacturing scalability, regulatory uncertainty for novel modalities, the gap between clinical promise and commercial reality – these are the conversations people are actually having. It was valuable to have that confirmed in person, and it will continue to shape our editorial direction.

We also have some exciting conference partnerships ahead: next up is Cell & Gene Therapy World Asia in Singapore at the end of June, where growing our APAC readership is a real priority.

If you’d like to stay close to the topics shaping the field coming out of ASGCT, including in vivo CAR-T, gene editing, and rare disease gene therapy, our upcoming CAR-T Special Interest Group is a great place to start. Register here to receive outputs first.