Case Library
Demo Case Library
Representative translational scenarios aligned with real AAV and next-generation delivery challenges.
Huntington's disease
Challenge
Broad CNS delivery with liver detargeting
Strategic thesis
Systemic engineered AAV is primary path; intrathecal backup maintained.
Inherited retinal disease
Challenge
Intravitreal approach with retinal barrier constraints
Strategic thesis
Route + tropism co-optimization for layered retinal penetration.
Duchenne muscular dystrophy
Challenge
Body-wide muscle distribution at acceptable dose
Strategic thesis
Dose efficiency and liver burden are co-primary optimization axes.
Immune escape scenario
Challenge
Pre-existing antibodies and repeat-dosing need
Strategic thesis
Escape-focused capsid engineering with redose plausibility program.
Liver-targeted gene replacement
Challenge
Potent expression with safety margin control
Strategic thesis
Conventional AAV baseline with selective detargeting logic.
Inner ear / cochlear targeting
Challenge
Compartment-specific delivery and procedural precision
Strategic thesis
Local route architecture dominates strategic fit.