Case Study · Regulatory Strategy · Translational Development
A translational strategy advisor used Urchin to develop a regulatory and clinical roadmap for a first-in-class therapeutic program.
"I knew there were clues to the right strategy scattered across the literature, but there was simply too much to get through, and I was worried I wasn't going to find them or make the right connections on my own. Urchin synthesized that information into a framework I could share directly with the board and with KOLs. What impressed me most was seeing several of the same nuanced recommendations emerge independently from both Urchin and the KOLs we were speaking with throughout the project. I'll absolutely be recommending it to other companies I work with."
Context
A pharmaceutical company was developing a product that physicians were independently using off-label, with encouraging early clinical results.
The company wanted to pursue regulatory approval for the new indication but first needed to answer a series of foundational questions. The mechanism of action was unknown; they lacked a validated potency assay to support manufacturing and quality control; and they needed a credible clinical development strategy for an indication with limited precedent.
Jonathan Thon engaged Urchin to help develop an integrated roadmap spanning mechanism, assay development, regulatory strategy, and clinical trial design.
The Challenge
The indication space is particularly challenging because outcomes are difficult to measure objectively. Clinical trials often rely on subjective endpoints, while there are few established in vitro assays that correlate with clinical outcomes. Without a robust potency assay, manufacturers are forced to rely on expensive and time-consuming in vivo testing to qualify each batch before release.
At the same time, the evidence needed to solve this problem was scattered across decades of research, regenerative medicine, and regulatory precedent. The challenge was not simply reviewing the literature — it was identifying the critical biological and regulatory connections hidden within an overwhelming volume of information.
What Urchin Did
Urchin synthesized evidence across the biological, regulatory, and clinical landscape to develop a unified translational strategy.
Across three iterative analyses, Urchin:
- Mapped regulatory precedent to identify how similar therapies had progressed through clinical development and what evidence regulators were likely to require.
- Designed a potency assay strategy linking disease biology, mechanism of action, manufacturing release testing, and clinical outcomes.
- Developed a clinical roadmap connecting preclinical studies, assay development, and Phase 1–3 trial design into a coherent development plan.
Rather than treating these as independent questions, Urchin connected them into a single evidence-based framework that could guide decision-making across the program.
Impact
Rather than delivering a static literature review, Urchin became part of the team's scientific workflow. Across three iterative rounds of analysis, the engagement evolved alongside the team's thinking: each iteration refined the regulatory strategy, potency assay recommendations, and clinical development plan as new questions emerged. Using Urchin, the team was able to reason across these topics simultaneously, rather than tackling them in sequence.
The result was an integrated roadmap connecting disease biology, mechanism of action, potency assays, regulatory precedent, and clinical trial design into a single evidence-based framework. This framework transformed a collection of disconnected scientific and regulatory questions into a coherent development strategy, providing a clearer path from promising clinical observations to a regulatory-ready development program.
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