[Whitepaper] The Science Is Proven. The Systems Are Not

This whitepaper, written by Elizabeth Figueroa PhD, Practice Expert at Dark Horse Consulting, and informed by 14 contributors from the Phacilitate Advisory Board -- spanning clinical, investment, manufacturing and nonprofit leadership -- sets out why operational systems, not science, are the defining bottleneck for the field.

What this whitepaper covers:

Five forces shaping the next phase of advanced therapies: 

Global Regulatory Evolution - Why FDA and EMA shifts are creating both opportunity and instability -- and what developers must account for in how they design trials, choose partners, and plan for launch.

Decentralisation and Patient Access - Why geographic concentration of treatment at academic medical centres is excluding most eligible patients, and what manufacturing, distribution and access models are changing that.

In Vivo Momentum - The investment case is clear. But with limited data maturity, 2026 clinical readouts will be decisive for the direction of the whole sector.

Data as Currency and the Geography Shift - Clinical data is gating investment. Developers are increasingly generating first-in-human data in China, Australia and the Middle East to move faster and at lower cost. What that means for Western biotechs.

Business Model Recalibration - Capital is not returning to advanced therapies broadly. It is returning selectively -- to programmes with clear clinical differentiation, credible manufacturing, and commercial infrastructure built earlier than feels comfortable.

"Advanced therapies will no longer be financed as scientific experiments but as integrated healthcare delivery businesses. Winners will demonstrate not only clinical efficacy, but repeatability, manufacturability, payer-aligned value, and commercial infrastructure."

Who contributed: 

This whitepaper draws on the perspectives of 14 members of the Phacilitate Advisory Board, including leaders from the Mayo Clinic, University of Pennsylvania, Children's National Hospital, Kiji Therapeutics, Siren Biotechnology, Cryoport Systems, Blood Centers of America and Saisei Ventures, among others.

Why download now:

This whitepaper is normally a paid resource. We are making it available free for a limited period because we believe the conversation it opens matters more than the download fee.

If you are building, funding, manufacturing, or enabling advanced therapies in Europe or globally, the five themes covered here are the ones shaping investment decisions, regulatory strategy and commercial outcomes right now.

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