A sample due diligence case from the perspective of PlexoA’s scientific advisory team.
Background
A mid-sized pharmaceutical company was scouting new RNA platforms to license for their rare disease pipeline. A biotech startup had caught their attention with a proprietary siRNA delivery system, claiming improved tissue specificity and reduced immune activation. The pharma team was intrigued but skeptical: the startup had limited data and bold claims. Before moving forward, they needed an independent, science-driven evaluation of the platform’s real-world potential.
The Questions We Asked
When a novel platform is being considered for licensing, especially in RNA therapeutics, the scientific details matter. Our team at PlexoA asked:
- Does this platform offer meaningful innovation over current siRNA delivery systems?
- Is the targeting truly selective — or just statistically significant?
- How robust are their in vivo models, and are they predictive of human biology?
- Are there any hidden technical or translational risks?
- How mature is the technology for scale-up and regulatory filing?
“Financial security and independence are like a three-legged stool resting on savings, insurance and investments.” – Brian Tracy
What We Evaluated
✅ Scientific Feasibility
- We analyzed the platform’s core components, from vector chemistry to tissue targeting ligands.
- The startup used modified liposomal carriers with claimed enhanced uptake in hepatic and muscle tissues.
- The preclinical data was largely in rodents; only one non-human primate study was referenced without raw data.
✅ Innovation Assessment
- The delivery improvements were real but incremental, not disruptive.
- Targeting specificity was present — but not unique compared to competitors with published datasets.
- Our landscape analysis revealed two similar technologies already in Phase I, making differentiation a concern.
✅ Translational Readiness
- CMC plans were early-stage, with no formal reproducibility data on formulation scale-up.
- The animal models lacked disease relevance.
- Safety signals like mild hepatotoxicity were downplayed.
Key Findings
Good Science, But Not Yet “License-Ready”
The technology showed potential — but was still in an exploratory stage. The startup needed deeper data, better model validation, and early regulatory engagement before a serious deal.
Red Flags We Flagged
- Safety signals that need further exploration
- Overstated targeting performance
- Gaps in formulation scale-up
- Competitive IP already in motion
What Happened Next
Rather than rushing into a licensing deal, the pharma company chose to delay engagement. They returned to the startup with a clear list of scientific milestones and asked for additional validation before resuming talks.
This saved the company a potentially costly and premature agreement — and gave the startup a better roadmap to de-risk their platform.
The Bigger Picture
RNA-based therapies are exciting, but delivery is the real bottleneck. Many platforms claim specificity and low toxicity, but few deliver both — at scale, and in humans. That’s where PlexoA’s R&D due diligence process adds value. By stress-testing the science behind the claims, we help partners move forward with confidence, not guesswork.
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