Overview
flowchart TD
therapeutics["therapeutics"] -->|"protects against"| age_related_cognitive_decline["age-related cognitive decline"]
therapeutics["therapeutics"] -->|"inhibits"| neuroinflammation["neuroinflammation"]
Therapeutics["Therapeutics"] -->|"references"| SIRT6["SIRT6"]
Therapeutics["Therapeutics"] -->|"references"| AADC["AADC"]
Therapeutics["Therapeutics"] -->|"references"| CX3CR1["CX3CR1"]
Therapeutics["Therapeutics"] -->|"references"| BACE1["BACE1"]
Therapeutics["Therapeutics"] -->|"references"| APOE["APOE"]
Therapeutics["Therapeutics"] -->|"references"| VCP["VCP"]
Therapeutics["Therapeutics"] -->|"references"| GFAP["GFAP"]
Therapeutics["Therapeutics"] -->|"references"| NURR1["NURR1"]
Therapeutics["Therapeutics"] -->|"references"| BDNF["BDNF"]
Therapeutics["Therapeutics"] -->|"references"| NLRP3["NLRP3"]
Therapeutics["Therapeutics"] -->|"references"| TFEB["TFEB"]
Therapeutics["Therapeutics"] -->|"references"| PPARGC1A["PPARGC1A"]
style therapeutics fill:#4fc3f7,stroke:#333,color:#000
This page catalogs companies developing exosome-based or extracellular vesicle (EV) therapeutics for [Parkinson's disease](/diseases/parkinsons-disease). Exosomes are small vesicles (30-150nm) that naturally cross the [blood-brain barrier](/entities/blood-brain-barrier) and can be engineered to deliver therapeutic cargo—including siRNA, ASOs, small molecules, and proteins—to target brain cells affected by PD pathology["@kalluri2020"].
...Overview
Mermaid diagram (expand to render)
This page catalogs companies developing exosome-based or extracellular vesicle (EV) therapeutics for [Parkinson's disease](/diseases/parkinsons-disease). Exosomes are small vesicles (30-150nm) that naturally cross the [blood-brain barrier](/entities/blood-brain-barrier) and can be engineered to deliver therapeutic cargo—including siRNA, ASOs, small molecules, and proteins—to target brain cells affected by PD pathology["@kalluri2020"].
EVs play a dual role in PD: they propagate pathological [alpha-synuclein](/proteins/alpha-synuclein) between neurons["@stuendl2023"], but can also be harnessed as therapeutic delivery vehicles. This has spurred company interest in engineering EVs to:
- Deliver alpha-synuclein-targeting RNA therapeutics["@matsumoto2023"]
- Package neurotrophic factors for dopaminergic neuron survival
- Serve as diagnostic biomarkers for PD progression
- BBB Penetration: Exosomes cross the blood-brain barrier through receptor-mediated transcytosis (integrins, tetraspanins, transferrin receptors)
- Targeting: Surface peptides (RVG for neuronal targeting) can be fused to tetraspanins for specific delivery to substantia nigra neurons
- Cargo Loading: Electroporation for siRNA/ASOs targeting SNCA gene expression; genetic engineering of producer cells for neurotrophic factor delivery
- MSC-Derived EVs: Mesenchymal stem cell-derived exosomes carry anti-inflammatory and neurotrophic cargo relevant to PD
EV Biomarkers for PD
- Brain-derived exosomes in blood carry phosphorylated alpha-synuclein (Ser129), [LRRK2](/genes/lrrk2), and [DJ-1](/entities/dj1)
- L1CAM+/NCAM+ immunocapture isolates neuronal exosomes for diagnostic use
- EV miRNA panels (miR-19b, miR-153, miR-409-3p) show diagnostic promise
Therapeutic Strategies
RNAi-loaded EVs: Suppress SNCA expression to reduce alpha-synuclein aggregation
Anti-α-syn antibody-loaded EVs: Target and neutralize pathological alpha-synuclein
Neurotrophic factor delivery: GDNF-loaded EVs promote dopaminergic neuron survival
MSC-derived EVs: Paracrine effects reduce neuroinflammationCompany Profiles
Capricor Therapeutics
| Attribute | Value |
|-----------|-------|
| Founded | 2005 |
| Headquarters | San Diego, CA |
| Ticker | NASDAQ: CAPR |
| Platform | MSC-derived extracellular vesicles (CAP-2003) |
Capricor develops cell-derived extracellular vesicle therapeutics. Its lead candidate CAP-2003 (CK+EVs) leverages mesenchymal stem cell-derived EVs with anti-inflammatory and neurotrophic properties. The company has explored CNS applications including neuroprotection.
- Pipeline: CAP-2003 (preclinical, inflammatory/neurological), CAP-1002 (cardiac, Phase 2)
- Rationale: MSC-derived EVs carry anti-inflammatory cytokines and neurotrophic factors (BDNF, GDNF) that may protect dopaminergic neurons
- PD Relevance: MSC-EVs reduce neuroinflammation and support neuronal survival in PD models
- Status: Active development; exploring neurological indications
Evox Therapeutics
| Attribute | Value |
|-----------|-------|
| Founded | 2016 |
| Headquarters | Oxford, UK |
| Ticker | Private |
| Platform | Exosome engineering (DeliverEX™) |
Evox Therapeutics develops exosome-based therapeutics using its DeliverEX™ platform, focused on CNS delivery of RNA therapeutics. The company has partnerships with major pharma to leverage exosome delivery across the blood-brain barrier.
- Pipeline: EV platform for CNS disease, including neurodegenerative indications
- Rationale: Exosomes enable crossing BBB and targeted CNS delivery; can load therapeutic RNA to silence disease-causing genes
- PD Relevance: Platform capable of delivering SNCA-targeting siRNA to neurons
- Status: Preclinical/partnering; platform available for CNS partnerships
PureTech Health
| Attribute | Value |
|-----------|-------|
| Founded | 2005 |
| Headquarters | Boston, MA |
| Ticker | NASDAQ: PRTC |
| Platform | Glymphatic delivery, LyoPho™ formulation |
PureTech Health develops platform technologies for CNS drug delivery. While primarily focused on the glymphatic pathway for brain delivery, the company has capabilities relevant to EV-based CNS delivery systems.
- Platform: Glymphatic pathway modulation, particle-based delivery
- Rationale: Enhanced brain penetration for therapeutics via perivascular routes
- PD Relevance: Multiple CNS programs in neuroinflammation; potential for EV integration
- Status: Research stage for EV platform; internal CNS pipeline active
Codiak BioSciences (Historical)
| Attribute | Value |
|-----------|-------|
| Founded | 2015 |
| Headquarters | Cambridge, MA |
| Ticker | NASDAQ: CDAK (delisted 2024) |
| Platform | engEx™ exosome platform |
Codiak was a pioneer in exosome therapeutics with its engEx™ platform. The company developed exosomes engineered with targeting moieties and loaded with therapeutic cargo. While the company underwent restructuring, its platform technology remains influential and available for licensing.
- Programs: exoSTING, exoIL-12 (oncology), CNS programs (historical)
- Technology: Surface engineering, cargo loading, scalable manufacturing
- PD Relevance: Platform technology theoretically applicable to alpha-synuclein targeting
- Status: Platform available for licensing/partnerships
Other Companies and Research Programs
| Company | Focus | Status |
|---------|-------|--------|
| NeuroDex | EV biomarkers for PD | Clinical |
| Exosome Diagnostics (Bio-Techne) | EV-based diagnostics | Commercial |
| amsbio | Exosome isolation reagents | Commercial |
| BreHealth | EV drug delivery platform | Research |
| Systemic蜗牛 | EV therapeutics | Preclinical |
Competitive Landscape
| Factor | Traditional AAV | EV-Based | MSC-EVs |
|--------|----------------|-----------|----------|
| BBB crossing | Limited (serotype-dependent) | Natural capability | Natural capability |
| Target specificity | High (promoter-dependent) | Tunable (surface engineering) | Moderate (paracrine) |
| Cargo capacity | ~4.7 kb | Limited (siRNA/ASO optimal) | Proteins, miRNA |
| Immunogenicity | Moderate | Low | Very low |
| Manufacturing scale | Established | Developing | Established (MSC culture) |
Clinical Trial Landscape
Currently limited specific EV trials for PD, but multiple trials using MSC-derived products have indirect relevance to EV therapeutics:
- MSC trials in PD: Multiple Phase I/II trials establishing safety of MSC administration
- EV biomarker trials: CSF exosomal pSer129 alpha-syn in diagnostic development
- RNAi delivery: Challenges with BBB crossing driving interest in EV platforms
Cross-Links
- [Extracellular Vesicles in Parkinson's Disease](/mechanisms/extracellular-vesicles-parkinsons) — Mechanism background
- [Exosomes](/entities/exosomes) — General exosome biology
- [Blood-Brain Barrier](/entities/blood-brain-barrier) — EV crossing mechanisms
- [AD Exosome Companies](/companies/ad-exosome-therapeutics) — Broader EV therapeutic landscape
- [PD Pipeline](/companies/pd-pipeline) — Broader PD therapeutic landscape
- [Stem Cell Therapy for Parkinsonism](/therapeutics/stem-cell-therapy-parkinsonism) — MSC programs
References
[Kalluri & LeBleu, Science (2020)](https://doi.org/10.1126/science.aau6977)
[Stuendl et al., Movement Disorders (2023)](https://doi.org/10.1002/mds.28739)
[Matsumoto et al., Movement Disorders (2023)](https://doi.org/10.1002/mds.28645)
[Capricor Therapeutics](https://www.capricor.com/)
[Evox Therapeutics](https://www.evoxtherapeutics.com/)
[PureTech Health](https://www.puretechhealth.com/)
[International Society for Extracellular Vesicles](https://www.isev.org/)