Table 2.
Gene therapies targeting C9orf72, SOD1, FUS and ATXN2.
| Target Gene | Drug | Company | properties | Aim | Result | Phase | References |
|---|---|---|---|---|---|---|---|
| SOD1 | Tofersen | Biogen; Ionis | ASO | Evaluate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics | Ongoing | 3 | ClinicalTrials.gov: NCT02623699; (Nicolas et al., 2018) |
| FUS | Jacifusen | Ionis | ASO | Evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics | Ongoing | 3 | ClinicalTrials.gov: NCT04768972; (Ling et al., 2015) |
| C9ORF72 | BIIB078 | Biogen | ASO | Evaluate the safety, tolerability, and pharmacokinetics | Safe, no benefit | 1 | ClinicalTrials.gov: NCT03626012 |
| WVE-004 | Wave Life Sciences Ltd. | ASO | Evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics | Ongoing | 1b/2a | ClinicalTrials.gov: NCT04931862 | |
| ATXN2 | BIIB105 | Biogen | ASO | Evaluate the safety, tolerability, and effect on disease progression | Ongoing | 1 | ClinicalTrials.gov: NCT04494256 |
ASO, Antisense Oligonucleotide.