Will Thompson attended Advanced Therapies 2026. Below are some of his key takeaways, alongside discussions from the UK-focused AMC ATMP manufacturing community.

Advanced Therapy Medicinal Products (ATMPs), spanning cell, gene and tissue therapies, represent a clear step change in complexity compared to traditional biologics and small molecules. However, nicely highlighted by Scott Cowan of the Scottish National Blood Transfusion Service, that their complexity is matched only by their potential.

The overarching themes remain consistent with previous years: reducing cost of goods, accelerating clinical development, and evolving regulatory frameworks, particularly for ultra-rare diseases. All of this feeds into building sustainable companies and restoring investor confidence in the space ultimately all securing patient access.

Clinical Example: uniQure’s AMT-130

A well-known example discussed was uniQure’s Huntington’s disease gene therapy candidate AMT-130. This AAV-based therapy is delivered via MRI-guided neurosurgery to the striatum and is designed to reduce expression of mutant huntingtin protein.

In September 2025, the company announced 36-month topline results suggesting a significant slowing of disease progression based on cUHDRS. Hugely encouraging data for a previously devastating and largely untreatable disease, but important to view in the context of relatively small and still evolving datasets.

Beyond the headline, the practical challenges are clear. The 12–20 hour surgical procedure is highly specialised, and the cost and complexity of AAV vector manufacturing will likely limit access.

What stood out more in discussion was the regulatory aspect. There appears to be ongoing dialogue and some misalignment between U.S. Food and Drug Administration and the company around expectations for study design, particularly in relation to control arms in surgical interventions such as sham procedures.

While not a typical example, it highlights a broader point for ATMPs: regulatory frameworks are still evolving alongside the science, which can introduce uncertainty around development timelines and approval pathways.

For me, this example captures the current ATMP landscape well. The potential is enormous for previously untreatable diseases, but challenges remain around manufacturing cost, access, and regulatory clarity. This is the type of uncertainty that investors are very aware of.

That said, across the exhibition floor and in talks there was a sense of cautious optimism. Compared to this time last year, there appears to be more engagement from investors and partners, even if the broader environment remains challenging.

UK Perspective: Infrastructure and Manufacturing

From a UK perspective, AMC discussions highlighted the importance of supporting cell and gene therapy infrastructure. For autologous therapies, starting material collection such as apheresis is critical to final product quality, making standardisation of collection, handling and transport an important factor for successful integration into healthcare systems.

The UK may be relatively well positioned to address this through the National Health Service, although this would a huge effort with coordinated training, aligned procurement and cross-site standardisation and you wonder who will drive this undertaking.

Manufacturing regulation was another key theme, particularly where expectations for classic sterile manufacture and biological products intersect, which can be challenging to interpret in practice.

This is especially relevant for ultra-rare indications, where applying full GMP expectations, such as extensive aseptic process simulations, can be considered disproportionately burdensome relative to very small patient populations.

While these standards exist for good reason, there is a growing discussion around how to balance patient safety with enabling access to potentially life saving/changing therapies.