Comprehensive overview of molecular targets being pursued for neurodegenerative disease therapy, including protein targets, receptors, enzymes, and genetic approaches.
NeuroWiki documents 2,965 protein entries and 3,662 gene entries, many with known or potential therapeutic relevance.
| Target Gene |
Drug |
Target |
Disease |
Status |
| SOD1 |
Tofersen |
mRNA |
ALS (SOD1) |
Approved |
| HTT |
Tominersen |
mRNA |
HD |
Failed |
| C9orf72 |
Various |
expansion |
ALS/FTD |
Phase 1 |
| Category |
Total Genes |
With Drug |
% Druggable |
| Kinases |
500+ |
50+ |
10% |
| GPCRs |
400+ |
100+ |
25% |
| Ion channels |
250+ |
75+ |
30% |
| Proteases |
500+ |
25+ |
5% |
| Epigenetic |
400+ |
15+ |
4% |
| Target Class |
Examples |
Mechanism |
Development Stage |
| TREM2 agonists |
AL002, JNJ-798 |
Microglial activation |
Phase 1/2 |
| SOD1 modulators |
ALS-2059 |
Reduce misfolding |
Preclinical |
| Antisense oligonucleotides |
tofersen, wve-004 |
Gene silencing |
Phase 1/3 |
| Alpha-synuclein antibodies |
cinomerersen, ABBV-0805 |
Reduce aggregation |
Phase 1/2 |
| Tau antibodies |
semorinemab, gosuranemab |
Block tau spread |
Phase 2/3 |
- Genetic evidence: Targets with strong genetic support (GWAS, Mendelian) have higher success rates
- Biomarker linkage: Targets should modulate disease-relevant biomarkers
- Brain penetration: CNS drugs require blood-brain barrier penetration
- Safety margin: Neurodegeneration therapies require exceptional safety profiles
- Combination approaches: Multi-target strategies may be necessary
This page is auto-maintained. Last updated: 2026-03-15