AptaBLE

Stages

1. 1.
   Aptamer generation and LNP design(library_design)

   This stage provides targeting ligands for constructing CD4-directed LNPs.

   Selection: Use a validated CD4-binding aptamer and novel aptamers generated by a transformer-based AI language model for CD4-targeted LNP functionalization.

2. 2.
   LNP formulation and physicochemical characterization(library_build)

   This stage creates the aptamer-LNP formulations to be tested and characterizes their physical properties before biological evaluation.

   Selection: Formulate LNPs with SM102 or MC3 and conjugate aptamers at controlled densities.

3. 3.
   Binding assessment(functional_characterization)

   This stage tests whether aptamer-functionalized LNPs engage the intended CD4 target before downstream delivery studies.

   Selection: Assess selective binding to recombinant CD4.

4. 4.
   In vitro transfection assessment(secondary_characterization)

   This stage tests whether target binding translates into selective cellular delivery in a cell-based assay before in vivo studies.

   Selection: Assess enhanced transfection of CD4+ versus CD4- T cells in vitro.

5. 5.
   In vivo biodistribution and safety evaluation(confirmatory_validation)

   This stage confirms whether the targeted formulations retain delivery enrichment and suitable safety in vivo.

   Selection: Assess enrichment of mRNA delivery to immune-rich tissues and evaluate safety.