Drug Target Landscape

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Drug Target Landscape

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Drug Target Landscape

<table class="infobox infobox-therapeutic">
<tr>
<th class="infobox-header" colspan="2">Drug Target Landscape</th>
</tr>
<tr>
<td class="label">Target</td>
<td>Drug Examples</td>
</tr>
<tr>
<td class="label">[Amyloid-beta (Aβ42/Aβ40)](/proteins/amyloid-beta)</td>
<td>[Lecanemab](/entities/lecanemab), [Donanemab](/entities/donanemab), Aducanumab</td>
</tr>
<tr>
<td class="label">[BACE1](/proteins/bace1)</td>
<td>Verubecestat, Umibecestat</td>
</tr>
<tr>
<td class="label">[Amyloid precursor protein (APP)](/proteins/app)</td>
<td>None</td>
</tr>
<tr>
<td class="label">Target</td>
<td>Drug Examples</td>
</tr>
<tr>
<td class="label">[Tau protein](/proteins/tau)</td>
<td>Anti-[tau](/proteins/tau) antibodies</td>
</tr>
<tr>
<td class="label">[Tau aggregation](/mechanisms/tau-aggregation)</td>
<td>Methylene blue</td>
</tr>
<tr>
<td class="label">[Tau kinases](/proteins/gsk3-beta)</td>
<td>Tideglusib</td>
</tr>
<tr>
<td class="label">Target</td>
<td>Drug Examples</td>
</tr>
<tr>
<td class="label">[α-Synuclein](/proteins/alpha-synuclein)</td>
<td>PRX002, BIIB054</td>
</tr>
<tr>
<td class="label">[α-Synuclein aggregation](/mechanisms/alpha-synuclein-aggregation)</td>
<td>NPT200-11</td>
</tr>
<tr>
<td class="label">Target</td>
<td>Drug Examples</td>
</tr>
<tr>
<td class="label">[TREM2](/proteins/trem2)</td>
<td>AL002, GV1004</td>
</tr>
<tr>
<td class="label">[CD33](/genes/cd33)</td>
<td>None</td>
</tr>
<tr>
<td class="label">[Microglial receptors](/cell-types/microglia)</td>
<td>Various</td>
</tr>
<tr>
<td class="label">Target</td>
<td>Drug Examples</td>
</tr>
<tr>
<td class="label">[Dopamine receptors](/proteins/dopamine-receptor)</td>
<td>Pramipexole, Rotigotine</td>
</tr>
<tr>
<td class="label">[Acetylcholinesterase](/proteins/ache)</td>
<td>[Donepezil](/entities/donepezil), [Rivastigmine](/entities/rivastigmine)</td>
</tr>
<tr>
<td class="label">[NMDA receptor](/entities/nmda-receptor)</td>
<td>Memantine</td>
</tr>
<tr>
<td class="label">Target Gene</td>
<td>Drug</td>
</tr>
<tr>
<td class="label">[SOD1](/genes/sod1)</td>
<td>Tofersen</td>
</tr>
<tr>
<td class="label">[HTT](/genes/htt)</td>
<td>Tominersen</td>
</tr>
<tr>
<td class="label">[C9orf72](/genes/c9orf72)</td>
<td>Various</td>
</tr>
<tr>
<td class="label">Category</td>
<td>Total Genes</td>
</tr>
<tr>
<td class="label">Kinases</td>
<td>500+</td>
</tr>
<tr>
<td class="label">GPCRs</td>
<td>400+</td>
</tr>
<tr>
<td class="label">Ion channels</td>
<td>250+</td>
</tr>
<tr>
<td class="label">Proteases</td>
<td>500+</td>
</tr>
<tr>
<td class="label">Epigenetic</td>
<td>400+</td>
</tr>
<tr>
<td class="label">Target Class</td>
<td>Examples</td>
</tr>
<tr>
<td class="label">[TREM2](/proteins/trem2) agonists</td>
<td>AL002, JNJ-798</td>
</tr>
<tr>
<td class="label">SOD1 modulators</td>
<td>ALS-2059</td>
</tr>
<tr>
<td class="label">Antisense oligonucleotides</td>
<td>tofersen, wve-004</td>
</tr>
<tr>
<td class="label">Alpha-synuclein antibodies</td>
<td>cinomerersen, ABBV-0805</td>
</tr>
<tr>
<td class="label">Tau antibodies</td>
<td>semorinemab, gosuranemab</td>
</tr>
</table>

Comprehensive overview of molecular targets being pursued for neurodegenerative disease therapy, including protein targets, receptors, enzymes, and genetic approaches.

Overview

NeuroWiki documents 2,965 protein entries and 3,662 gene entries, many with known or potential therapeutic relevance.

Amyloid-Based Targets

Tau-Based Targets

Synuclein-Based Targets

Neuroinflammation Targets

Neurotransmitter Targets

Genetic Targets

Antisense Oligonucleotides (ASOs)

Gene Therapy Vectors

[AAV2-GAD](/therapeutics/aav2-gad) — Parkinson's
[AAV2-AADC](/therapeutics/aav2-aadc) — Parkinson's
[AAVrh.10](/therapeutics/aavrh10-nap) — Alzheimer's

Druggable Genome Coverage

Target Validation Status

Well-Validated (Genetic + Functional)

[APP](/genes/app) — AD
[SNCA](/genes/snca) — PD
[LRRK2](/genes/lrrk2) — PD
[SOD1](/genes/sod1) — ALS
[C9orf72](/genes/c9orf72) — ALS/FTD

Genetically Linked, Uncertain Mechanism

[TREM2](/genes/trem2) — AD
[PLD3](/genes/pld3) — AD
[GBA](/genes/gba) — PD

Emerging Target Classes

Protein Homeostasis

[Ubiquitin-proteasome](/mechanisms/ubiquitin-proteasome-system)
[Autophagy](/mechanisms/autophagy-lysosome-pathway)
[Molecular chaperones](/mechanisms/molecular-chaperones)

Mitochondrial Function

[PGC-1α](/proteins/pgc1-alpha)
[Mitophagy](/mechanisms/mitophagy)
[Mitochondrial dynamics](/mechanisms/mitochondrial-dynamics)

Emerging Targets

Target Validation Considerations

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](/entities/blood-brain-barrier) penetration

Safety margin: Neurodegeneration therapies require exceptional safety profiles

Combination approaches: Multi-target strategies may be necessary

[Treatment Pipeline Overview](/treatment-pipeline-overview)
[Protein Drug Targets](/proteins/protein-drug-targets)
[Druggable Genome Coverage](/genes/druggable-genome-coverage)
[Therapeutic Targetability](/mechanisms/_therapeutic-targets)

This page is auto-maintained. Last updated: 2026-03-15

External Links

[PubMed](https://pubmed.ncbi.nlm.nih.gov/)
[KEGG Pathways](https://www.genome.jp/kegg/pathway.html)

References

Unknown, Gene/Protein databases (n.d.)

Related Hypotheses

From the [SciDEX Exchange](/exchange) — scored by multi-agent debate

[Purinergic Signaling Polarization Control](/hypothesis/h-0758b337) — 0.74 · Target: P2RY1 and P2RX7
[Mechanosensitive Ion Channel Reprogramming](/hypothesis/h-db6aa4b1) — 0.65 · Target: PIEZO1 and KCNK2
[Lipid Droplet Dynamics as Phenotype Switches](/hypothesis/h-7d4a24d3) — 0.57 · Target: DGAT1 and SOAT1
[TREM2-mediated microglial tau clearance enhancement](/hypothesis/h-b234254c) — 0.55 · Target: TREM2
[Smartphone-Detected Motor Variability Correction](/hypothesis/h-072b2f5d) — 0.63 · Target: DRD2/SNCA
[TREM2 Conformational Stabilizers for Synaptic Discrimination](/hypothesis/h-044ee057) — 0.58 · Target: TREM2
[Microbial Metabolite-Mediated α-Synuclein Disaggregation](/hypothesis/h-74777459) — 0.57 · Target: SNCA, HSPA1A, DNMT1
[Palmitoylation-Targeted BACE1 Trafficking Disruptors](/hypothesis/h-441b25ba) — 0.55 · Target: BACE1

Related Analyses:

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[Digital biomarkers and AI-driven early detection of neurodegeneration](/analysis/SDA-2026-04-01-gap-012) 🔄
[What are the mechanisms by which gut microbiome dysbiosis influences Parkinson's disease pathogenesi](/analysis/SDA-2026-04-01-gap-20260401-225155) 🔄
[Lipid raft composition changes in synaptic neurodegeneration](/analysis/SDA-2026-04-01-gap-lipid-rafts-2026-04-01) 🔄
[Synaptic pruning by microglia in early AD](/analysis/SDA-2026-04-01-gap-v2-691b42f1) 🔄

📖 View canonical wiki page →

Drug Target Landscape

Drug Target Landscape

Drug Target Landscape

Overview

Amyloid-Based Targets

Tau-Based Targets

Synuclein-Based Targets

Neuroinflammation Targets

Neurotransmitter Targets

Genetic Targets

Antisense Oligonucleotides (ASOs)

Gene Therapy Vectors

Druggable Genome Coverage

Target Validation Status

Well-Validated (Genetic + Functional)

Genetically Linked, Uncertain Mechanism

Emerging Target Classes

Protein Homeostasis

Mitochondrial Function

Emerging Targets

Target Validation Considerations

Related Pages

See Also

External Links

References

Related Hypotheses