Drug Target Landscape

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

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

Related Pages

• [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)

See Also

• [Amyloid-beta (Aβ42/Aβ40)](/proteins/amyloid-beta)
• [BACE1](/genes/bace1)
• [Amyloid precursor protein (APP)](/genes/app)
• [Tau protein](/proteins/tau)
• [Tau aggregation](/mechanisms/tau-aggregation)
• [Tau kinases](/mechanisms/tau-phosphorylation)
• [α-Synuclein](/proteins/alpha-synuclein)
• [α-Synuclein aggregation](/mechanisms/alpha-synuclein-aggregation)
• [TREM2](/genes/trem2)
• [CD33](/genes/cd33)

External Links

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

References

Related Hypotheses

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

• [Purinergic Signaling Polarization Control](/hypothesis/h-0758b337) — <span style="color:#81c784;font-weight:600">0.74</span> · Target: P2RY1 and P2RX7
• [Mechanosensitive Ion Channel Reprogramming](/hypothesis/h-db6aa4b1) — <span style="color:#81c784;font-weight:600">0.65</span> · Target: PIEZO1 and KCNK2
• [Lipid Droplet Dynamics as Phenotype Switches](/hypothesis/h-7d4a24d3) — <span style="color:#ffd54f;font-weight:600">0.57</span> · Target: DGAT1 and SOAT1
• [TREM2-mediated microglial tau clearance enhancement](/hypothesis/h-b234254c) — <span style="color:#ffd54f;font-weight:600">0.55</span> · Target: TREM2
• [Smartphone-Detected Motor Variability Correction](/hypothesis/h-072b2f5d) — <span style="color:#81c784;font-weight:600">0.63</span> · Target: DRD2/SNCA
• [TREM2 Conformational Stabilizers for Synaptic Discrimination](/hypothesis/h-044ee057) — <span style="color:#ffd54f;font-weight:600">0.58</span> · Target: TREM2
• [Microbial Metabolite-Mediated α-Synuclein Disaggregation](/hypothesis/h-74777459) — <span style="color:#ffd54f;font-weight:600">0.57</span> · Target: SNCA, HSPA1A, DNMT1
• [Palmitoylation-Targeted BACE1 Trafficking Disruptors](/hypothesis/h-441b25ba) — <span style="color:#ffd54f;font-weight:600">0.55</span> · Target: BACE1

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