Table 4. ASO-Based Therapeutics for the Treatment of Brain Disease That Are FDA-Approved or Currently in Clinical Trialsa.
| drug | disease | route | status | sponsor | NCT number |
|---|---|---|---|---|---|
| ASOs | |||||
| IONIS MAPTRx | Alzheimer disease | Intrathecal | Phase 1 | Ionis Pharmaceuticals | NCT03186989 |
| BIIB094 | Parkinson disease | Intrathecal | Phase 1 | Ionis Pharmaceuticals | NCT03976349 |
| WVE-120101 | Huntington disease | Intrathecal | Phase 1/2 | Wave Life Sciences | NCT03225833 |
| WVE-120102 | Huntington disease | Intrathecal | Phase 1/2 | Wave Life Sciences | NCT03225846 |
| Imetelstat | Glioblastoma brainstem tumors | Intravenous | Phase 2, terminated | Geron | NCT01836549 |
| Tominersenb | Huntington disease | Intrathecal | Phase 3, recruiting | Roche | NCT03842969 |
| NCT03761849 | |||||
| Trabedersen | Glioblastoma | Intratumoral | Phase 3, terminated | Isarna therapeutics | NCT00761280 |
| Eteplirsen (Exondys 51) | DMD | Intravenous | FDA approved | Sarepta Therapeutics | |
| Nusinersen (Spinraza) | Spinal muscular atrophy | Intrathecal | FDA approved | Biogen |
a
Abbreviations: ASOs, antisense oligonucleotides; DMD, Duchenne muscular dystrophy. NCT: ClinicalTrials.gov identifier number. Data were collected from ClinicalTrials.gov and ref (153).
b
Tominersen (previously known as IONIS-HTTRx and RG6042).