Decision Output
Recommendation / Next Best Action
Capture recommended architecture, rationale, alternatives not selected, and highest-priority experiments in an export-style decision brief.
Recommended architecture
Engineered AAV systemic CNS delivery
Moderate-high confidence (0.72) with translation and immunity as dominant uncertainty drivers.
Why selected
- Best combined score for CNS coverage, durability fit, and strategic scalability.
- Maintains path to clinically realistic administration workflow.
- Engineering levers directly map to highest-value bottlenecks.
Why alternatives were not selected
- Intrathecal/local AAV: strong near-term feasibility but inadequate broad tissue coverage.
- LNP transient editor: repeat-dose upside offset by CNS penetration and durability gaps.
Next experiments
Priority 1
NHP biodistribution benchmark with CNS:liver quantitative endpoint.
Priority 2
Human sera panel for neutralizing antibody escape profiling.
Priority 3
Innate immune signature study at projected clinical dose band.
Priority 4
Manufacturing mini-pilot with capsid variant panel and full QC readout.
One-page export card
Program status: Proceed with constrained redesign; evaluate kill criteria after first translational package.