Exosomes: What They Do and Who’s Doing What

Exosomes, small membrane-bound vesicles ranging from 30 to 100 nanometers, actively facilitate communication between cells. Cells produce these vesicles within their endosomal compartments and release them through exocytosis. Packed with proteins, lipids, and nucleic acids, exosomes carry critical messages that influence recipient cells, driving processes like immune responses and tissue repair. A 2019 study in Cell and Bioscience confirms exosomes reprogram distant cells by delivering cell-specific cargos, making them pivotal in intercellular signaling. Companies like Evox Therapeutics leverage this by engineering exosomes to deliver therapeutic payloads, such as siRNAs, for precise gene modulation in diseases like cancer. Their technology enhances exosome stability and targeting, positioning them as a leader in this space.

Biological Roles of Exosomes

Exosomes regulate complex intracellular pathways, impacting diverse physiological functions. They transfer genetic material, such as microRNAs, to modulate gene expression in target cells. In immune responses, exosomes carry cytokines and antigens, enhancing the body’s defense against infections. They also support nervous system development by delivering growth factors and signaling proteins to neurons. These functions position exosomes as essential players in maintaining cellular harmony, though their full roles in health and disease remain under investigation. Coya Therapeutics, for instance, focuses on neurodegenerative diseases, using exosome engineering to deliver anti-inflammatory molecules, capitalizing on exosomes’ ability to cross the blood-brain barrier. Market trends show a surge in R&D, with over 388 clinical trials underway globally, driven by the potential of exosomes in precision medicine.

Exosomes vs. Stem Cells: A Key Distinction

Exosomes differ significantly from stem cells, despite their shared therapeutic potential. Stem cells, undifferentiated and capable of developing into specialized cell types, drive tissue regeneration. Exosomes, however, act as messengers, transferring bioactive molecules without differentiating. Mesenchymal stem cells (MSCs) produce abundant exosomes, which some studies suggest may mediate MSC therapeutic effects. Unlike stem cells, exosomes offer a targeted delivery mechanism, bypassing risks associated with cell transplantation. Capricor Therapeutics harnesses MSC-derived exosomes for cardiovascular and inflammatory conditions, with their platform showing promise in early-phase trials for heart attack recovery. The exosome therapeutics market, valued at $550.58 thousand in 2022, is projected to reach $1.98 million by 2030, growing at a CAGR of 17.4%, fueled by such innovations.

Market Insights: A Growing Industry

The exosome market is expanding rapidly, driven by increasing prevalence of chronic diseases like cancer and neurodegenerative disorders. North America currently dominates due to advanced healthcare infrastructure and significant R&D investment, with companies like Exosome Diagnostics developing exosome-based liquid biopsies for cancer detection. Their ExoDx Prostate Test has received FDA Fast Track approval, highlighting the diagnostic potential of exosomes. The market’s growth is further propelled by strategic collaborations, such as Sartorius AG and RoosterBio partnering to streamline exosome manufacturing processes, addressing scalability challenges. However, high R&D costs and regulatory hurdles, particularly from the FDA, remain barriers to widespread adoption.

Aegle Therapeutics Advances AGLE-102 in Multiple Indications

Aegle Therapeutics is developing AGLE-102, a topical therapy made of extracellular vesicles (EVs) including exosomes, derived from mesenchymal stem cells (MSCs). The company aims to treat serious dermatological conditions with high unmet needs. In January 2024, Aegle reported positive results from the first patient in a phase 1/2 trial for severe second-degree burns. The patient received a single topical dose within 48 hours of injury. By day seven, the wound showed 99% epithelialization and reduced edema. AGLE-102 is also under investigation in a phase 1/2 trial for dystrophic epidermolysis bullosa, a rare blistering disease. Additionally, a preclinical program is evaluating AGLE-102 for graft-versus-host disease.

Aruna Bio Targets CNS with Neural-Derived Exosomes

Aruna Bio is developing AB126, a neural exosome therapy derived from human neural stem cells. The therapy targets central nervous system (CNS) conditions. AB126 has shown the ability to cross the blood-brain barrier and concentrate in the cerebellum and basal ganglia. These brain areas control motor functions and cognition. This is especially important due to the difficulty of delivering drugs across the blood-brain barrier, which shields the CNS from blood-borne toxins and pathogens. In January 2024, the FDA cleared Aruna’s IND application for AB126. A phase 1b/2a clinical trial for acute ischemic stroke was expected to begin in the second half of 2024.

Capricor Therapeutics Develops Exosome-Based COVID-19 Vaccine

Capricor Therapeutics is advancing its StealthX platform, which enables protein expression, drug loading, and targeted delivery via exosomes. The company’s lead candidate is a multivalent vaccine against SARS-CoV-2. Capricor partnered with the NIH to investigate the vaccine. The vaccine is also part of Project NextGen, an HHS initiative for next-generation COVID-19 vaccines. As part of the project, the National Institute of Allergy and Infectious Diseases (NIAID), a division of the NIH, will conduct a phase 1 trial pending regulatory approval.

Evox Therapeutics Expands CNS Pipeline with Eli Lilly Partnership

Evox Therapeutics develops exosome-based therapies for rare genetic diseases. Its DeliverEX platform enables targeted drug delivery via engineered exosomes. Evox partnered with Eli Lilly in 2020 in a $1.2 billion deal to develop RNA-based therapies for neurological disorders. The company raised $95.4 million in 2021. Evox’s two lead assets target undisclosed CNS indications and remain in early development.

EXO Biologics Advances Lead Candidate for Neonatal Lung Disease

EXO Biologics is developing EXOB-001 to treat bronchopulmonary dysplasia, a life-threatening lung disease in premature infants. The therapy, based on MSC-derived exosomes, received orphan drug designation from the FDA in 2022. In June 2024, EXO completed dosing in the first cohort of its phase 1/2 EVENEW study—the first EMA-approved trial for MSC-derived exosomes. In April 2024, EXO raised €16 million ($17 million) in series A funding to support clinical trials and manufacturing expansion.

ILIAS Biologics Reports Positive Safety Results from Phase 1 Trial

ILIAS Biologics uses its EXPLOR platform to load therapeutic proteins into engineered exosomes. Its lead candidate, ILB-202, delivers the anti-inflammatory protein super-repressor IκB (srIκB), which inhibits NF-κB activity. This blocks inflammation at the cellular level. In July 2024, ILIAS announced final results from a phase 1 trial completed in Australia in November 2023. The trial reported no dose-limiting toxicities, serious adverse events, infusion reactions, or cytokine storms. All participants completed the study without dropouts.

Exosomes Promise Transformation in Drug Delivery and Diagnostics

The expanding field of exosome-based therapeutics is rapidly redefining the landscape of precision medicine. From engineered delivery platforms to clinical breakthroughs in dermatology, neurology, and infectious diseases, leading biotech companies are harnessing the natural communication capabilities of exosomes to develop safer, more targeted therapies. With global investment rising, regulatory milestones being achieved, and promising early-phase trial data emerging, exosomes are poised to become a cornerstone technology across diagnostics and drug delivery. As research accelerates and commercial interest intensifies, the exosome revolution is well underway.

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Author

Bernice Lottering