Table 2.
Nanocarrier-mediated formulation under clinical trials against different neurodegenerative disorders (ClinicalTrials.gov accessed on 31 January 2022).
| Product (Active Molecules/Class) | Nanocarrier (Composition) | Indications | Clinical Phase, NCT Number |
|---|---|---|---|
| ALN-TTR02 (Patisiran) | Lipid nanoparticle (DLin-MC3-DMA; PEG2000-C-DMG; DSPC; and cholesterol) |
Transthyretin mediated amyloidosis | Approved for marketing, NCT02939820 |
| APH-1105 (an α-secretase modulator) | Nanoparticle | Mild-to-moderate AD, dementia | Phase 2, NCT03806478 |
| Short palindromic repeats (CRISPR)/Cas9 gene | Lipid nanoparticle (proprietary lipid nanoparticle (LNP) delivery-system proprietary ionizable lipid, combined with a phospholipid, a pegylated lipid (molecular weight of polyethylene glycol, 2000 Da), and cholesterol) |
Hereditary transthyretin amyloidosis | Phase 1, NCT04601051 |
| CNM-Au8 (Nanocrystalline gold) | Gold nanocrystals | ALS | Phase 1, NCT04081714 |
| CNM-Au8 (Nanocrystalline gold) | Gold nanocrystals | ALS | Phase 2, NCT04098406 |
| CNM-Au8 (Nanocrystalline gold) | Gold nanocrystals | ALS | Phase 2, NCT03843710 |
| CNM-Au8 (Nanocrystalline gold) | Gold nanocrystals | PD | Phase 2, NCT03815916 |
AD—Alzheimer’s disease; ALS—amyotrophic lateral sclerosis; DLin-MC3-DMA—dilinoleylmethyl-4-dimethylaminobutyrate; DSPC—distearoylphosphatidylcholine; PEG2000-C-DMG—1,2-dimyristoyl-rac-glycero-3-methoxypolyethylene glycol-2000; PD—Parkinson’s disease.