This page catalogs companies developing exosome-based or extracellular vesicle (EV) therapeutics for Parkinson's disease. Exosomes are small vesicles (30-150nm) that naturally cross the 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 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
- Brain-derived exosomes in blood carry phosphorylated alpha-synuclein (Ser129), LRRK2, and DJ-1
- L1CAM+/NCAM+ immunocapture isolates neuronal exosomes for diagnostic use
- EV miRNA panels (miR-19b, miR-153, miR-409-3p) show diagnostic promise
- 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 neuroinflammation
| 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
| 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
| 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
| 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
- Kalluri & LeBleu, Science (2020)
- Stuendl et al., Movement Disorders (2023)
- Matsumoto et al., Movement Disorders (2023)
- Capricor Therapeutics
- Evox Therapeutics
- PureTech Health
- International Society for Extracellular Vesicles