AD Exosome and Extracellular Vesicle Companies

Overview

This page catalogs companies developing exosome-based or extracellular vesicle (EV) therapeutics for Alzheimer's 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["kalluri2020"][sun2025].

Technology Platforms

Overview

This page catalogs companies developing exosome-based or extracellular vesicle (EV) therapeutics for Alzheimer's 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["kalluri2020"][sun2025].

Technology Platforms

EV-Mediated Drug Delivery

• BBB Penetration: Exosomes cross the blood-brain barrier through receptor-mediated transcytosis (integrins, tetraspanins, transferrin receptors) and macropinocytosis
• Targeting: Surface peptides (RVG for neuronal targeting, T7 for transferrin receptor) can be fused to tetraspanins
• Cargo Loading: Electroporation for siRNA/ASOs; genetic engineering of producer cells for mRNA/protein

EV Biomarkers

• Brain-derived exosomes in blood carry [Aβ](/proteins/amyloid-beta-protein), [p-tau](/biomarkers/p-tau-217), [alpha-synuclein](/proteins/alpha-synuclein), and [NfL](/proteins/nfl-protein)
• L1CAM+/NCAM+ immunocapture isolates neuronal exosomes for diagnostic use

Manufacturing Platforms

• Mesenchymal stem cell (MSC)-derived exosomes
• Engineered producer cell lines
• GMP-scale isolation and characterization

Company Profiles

Capricor Therapeutics

| Attribute | Value |
|-----------|-------|
| Founded | 2005 |
| Headquarters | San Diego, CA |
| Ticker | NASDAQ: CAPR |
| Platform | MSC-derived exosomes (CAP-2003) |

Capricor develops cell-derived extracellular vesicle therapeutics. Its lead candidate CAP-2003 (CK+EVs) is in development for inflammatory and neurological conditions. The company leverages proprietary isolation technology to produce clinical-grade EVs.

• Pipeline: CAP-2003 (preclinical), CAP-1002 (cardiac, Phase 2)
• Rationale: MSC-derived exosomes carry anti-inflammatory and neurotrophic factors
• Status: Active development

PureTech Health (Incl. Karuna)

| Attribute | Value |
|-----------|-------|
| Founded | 2005 |
| Headquarters | Boston, MA |
| Ticker | NASDAQ: PRTC |
| Platform | Glymphatic delivery, LyoPho™ formulation |

PureTech Health develops platform technologies for CNS drug delivery. Its internal pipeline includes LYT-100 (deupirfenone) for AD-related neuroinflammation. The company has established R&D capabilities in EV-based delivery systems.

• Platform: Glymphatic pathway modulation, particle-based delivery
• Rationale: Enhanced brain penetration for therapeutics
• Status: Research stage for EV platform

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. The company focuses on CNS delivery of RNA therapeutics and has partnerships with major pharma.

• Pipeline: EV platform for CNS disease
• Rationale: Exosomes enable crossing BBB and targeted CNS delivery
• Status: Preclinical/partnering

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 exosome engineered with targeting moieties and loaded with therapeutic cargo. While the company underwent restructuring, its platform technology remains influential.

• Programs: exoSTING, exoIL-12 (oncology), CNS programs
• Technology: Surface engineering, cargo loading, scalable manufacturing
• Status: Platform available for licensing/partnerships

Other Companies and Research Programs

| Company | Focus | Status |
|---------|-------|--------|
| NeuroDex | EV biomarkers for AD | Clinical |
| Exosome Diagnostics (Bio-Techne) | EV-based diagnostics | Commercial |
| amsbio | Exosome isolation reagents | Commercial |
| Systemic蜗牛 | EV therapeutics | Preclinical |
| BreHealth | EV drug delivery platform | Research |

Academic and Research Programs

Key Research Groups

• MIT/Kalluri Lab: Exosome biology and engineering
• UCSF/Goetzl Lab: Brain-derived exosome biomarkers in AD
• UCLA/Butler Lab: MSC-derived exosomes for neurodegeneration

Clinical Trials (AD, EV-Based)

Competitive Landscape

| Factor | Traditional mAb | EV-Based |
|--------|----------------|--------|
| BBB crossing | Limited | Natural capability |
| Target specificity | High | Tunable |
| Manufacturing scale | Mature | Developing |
| Cargo flexibility | Proteins only | siRNA, ASO, small molecules |

Cross-Links

• [Exosomes](/entities/exosomes) — General exosome biology](/entities)
• [Blood-Brain Barrier](/entities/blood-brain-barrier) — EV crossing mechanisms](/entities)
• [AD Pipeline](/companies/ad-pipeline) — Broader AD therapeutic landscape](/companies/ad-pipeline)
• [Novel Mechanism AD Companies](/companies/emerging-novel-mechanism-ad-companies-2024-2025) — Next-gen approaches
• [PD Exosome and EV Companies](/companies/pd-exosome-therapeutics) — PD-specific EV therapeutics

References