Exosome-Based Drug Delivery: Nature’s Nanocarriers Reimagined
Exosomes — tiny vesicles secreted by cells — have emerged as one of the most promising delivery vehicles in advanced therapeutics. Unlike synthetic lipid nanoparticles (LNPs) or viral vectors, exosomes can ferry complex cargoes like RNA, proteins, or small molecules across biological barriers with high specificity and minimal immunogenicity. Because they’re derived from the body’s own cells, exosomes offer the potential for biocompatible, tissue-targeted drug delivery, addressing some of the biggest limitations in gene therapy, RNA medicines, and cell-based immunotherapies.
Once dismissed as mere cellular waste, exosomes are now recognized as vital mediators of intercellular communication. Researchers have tapped into their innate ability to transport molecular messages to design next-generation therapeutics that can cross the blood–brain barrier (BBB), evade the immune system, and deliver potent payloads precisely where they’re needed most.
As this emerging modality matures, a wave of young biotechs is racing to engineer, load, and scale exosome production for clinical use to tackle challenges in manufacturing, cargo loading, and reproducibility along the way. Some companies are engineering exosomes purely as smart delivery vehicles, while others leverage their innate regenerative or immunomodulatory properties to function as therapeutics in their own right. Here are five companies — Evox Therapeutics, Aruna Bio, Capricor Therapeutics, Kimera Labs, and Aegle Therapeutics — leading the charge to turn exosomes into tomorrow’s next-generation biologics and delivery systems, offering fresh hope for delivering complex RNA, gene editing, or protein therapies where older delivery vehicles fall short.
Evox Therapeutics: Engineering Exosomes for Systemic Drug Delivery
Oxford-based Evox Therapeutics is widely recognized as one of the most advanced companies in the exosome therapeutics space, pioneering new ways to engineer and load exosomes to deliver complex biologics systemically. Evox’s core technology platform DeliverEX™ combines exosome biology, protein engineering, and proprietary manufacturing methods to enable the loading, targeting, and functional delivery of therapeutic cargo, including RNA, gene-editing tools, and proteins, across hard-to-reach biological barriers.
Unlike traditional delivery systems, exosomes can cross the BBB and reach intracellular targets without provoking significant immune responses. Evox has demonstrated that its engineered exosomes can deliver functional RNA therapeutics, antisense oligonucleotides (ASOs), and even enzyme replacement candidates to cells and tissues that are historically difficult to reach. This positions the platform for neurological, metabolic, and rare genetic diseases, where conventional therapies often fail due to poor biodistribution.
Evox’s pipeline includes wholly owned and partnered programs. A major highlight is its multi-program collaboration with Eli Lilly, announced in 2020, which focuses on leveraging DeliverEX for the systemic delivery of RNA interference (RNAi) and antisense therapies for undisclosed central nervous system (CNS) and rare disease indications. Under this deal, Evox received an upfront payment and significant potential milestone payments, with Evox retaining manufacturing rights for exosome drug product supply, an indicator of its GMP manufacturing capabilities, which remains a key hurdle in the exosome space.
Beyond the Lilly partnership, Evox has an internal lead program targeting argininosuccinic aciduria (ASA), a rare urea cycle disorder. Using exosomes to deliver functional argininosuccinate lyase (ASL) enzyme to liver cells, Evox aims to overcome the delivery limitations faced by conventional enzyme replacement therapies, which often fail to reach the cytoplasm where the enzyme is needed.
To support its pipeline, Evox operates dedicated in-house exosome manufacturing facilities in Oxfordshire, with expertise spanning process development, scale-up, purification, and quality control. The company holds a robust intellectual property estate, with over 70 granted patents and 190+ pending patent applications globally covering core aspects of exosome engineering, loading, and targeting.
As exosome therapies move from lab bench to clinic, manufacturing scalability and regulatory alignment remain critical hurdles. Evox’s early focus on process development and chemistry, manufacturing, and controls (CMC) standards puts it ahead in addressing these hurdles while providing pharmaceutical partners with a credible path from discovery to commercialization.
With DeliverEX as its backbone, Evox is positioning itself to deliver a first-in-class portfolio of exosome-based therapies that can transform how we treat severe and life-limiting conditions that conventional biologics and gene therapies struggle to reach.
Aruna Bio: Pioneering Neural-Derived Exosomes for CNS Delivery
Aruna Bio, headquartered in Atlanta, Georgia, is forging a unique path in the exosome space with its focus on neural-derived exosomes that inherently home to the CNS. By leveraging the innate BBB-crossing capability of these exosomes, Aruna aims to unlock transformative treatments for some of the most intractable neurological conditions, where conventional biologics, small molecules, or viral vectors often fail to penetrate the brain effectively or safely.
At the core of Aruna Bio’s innovation is its ABEx™ platform, which combines proprietary neural exosome production with advanced methods for isolation, loading, and targeting. Unlike exosomes derived from generic cell lines or mesenchymal stem cells, neural exosomes exhibit natural tropism to damaged neural tissue, a property Aruna uses to develop both standalone neurotherapeutics and smart exosome-based delivery vehicles.
Aruna’s lead clinical program AB126 is being developed for acute ischemic stroke, a condition where current treatments are extremely limited and time-sensitive. Preclinical studies suggest that AB126 can reduce post-stroke inflammation, modulate the local immune response, and promote neuronal regeneration, translating into improved functional recovery in animal models. This approach highlights exosomes not merely as passive carriers but as biologically active therapeutics with regenerative and immunomodulatory properties.
Beyond AB126, Aruna Bio is extending its platform to serve as a precision delivery system for RNA, small molecules, and gene-editing payloads aimed at neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS) and multiple sclerosis (MS). The company’s work explores ways to load therapeutic cargos into neural-derived exosomes while retaining their BBB-crossing capabilities and intrinsic safety profile, addressing key limitations of lipid nanoparticles and viral vectors, which can provoke immune responses or struggle with brain penetration.
To deliver on this promise, Aruna has invested in scalable GMP-compliant manufacturing of its neural exosome product, working to demonstrate batch consistency, purity, and potency — all critical regulatory hurdles for emerging exosome modalities. Its intellectual property portfolio spans proprietary methods for neural exosome production, surface modification for enhanced targeting, and loading techniques for delivering nucleic acid and protein cargos.
While many in the exosome field are targeting oncology, gene therapy, or regenerative medicine more broadly, Aruna’s laser focus on neurotherapeutics gives it a distinctive edge. By pairing the regenerative and protective benefits of neural stem cell exosomes with the modularity of engineered payload delivery, the company is well positioned to pioneer first-in-class treatments for patients suffering from devastating brain and spinal cord conditions.
Capricor Therapeutics: Cardiac- and Muscle-Derived Exosomes for Regenerative and Rare Diseases
Capricor Therapeutics, based in Los Angeles, California, is an exosome-focused biotech developing cardiosphere-derived cell (CDC) exosomes for both regenerative medicine and rare genetic diseases. Capricor began as a cardiac cell therapy company but rapidly expanded its focus to leverage the powerful paracrine signaling of exosomes secreted by CDCs, vesicles rich in microRNAs, proteins, and other bioactive factors that naturally modulate inflammation and fibrosis.
Capricor’s lead exosome candidate CAP-2003 harnesses these cell-derived nanovesicles as a regenerative therapeutic for a range of inflammatory and fibrotic disorders, including myocardial infarction, Duchenne muscular dystrophy (DMD), and severe lung injury. Preclinical data have shown that CAP-2003 can reduce cardiac scarring, modulate inflammatory pathways, and support tissue regeneration, benefits that traditional cell therapies often struggle to deliver reproducibly.
Beyond direct regenerative effects, Capricor is also exploring the use of CDC-derived exosomes as delivery vehicles for RNA and protein-based therapeutics. By combining the immunologically quiet profile of natural exosomes with the company’s established exosome purification and scalable GMP manufacturing capabilities, Capricor is working to overcome the consistency and scale hurdles that have challenged other players in the space.
In the rare disease realm, Capricor has a notable footprint in DMD. Its lead cell therapy candidate CAP-1002 is in late-stage development, but the company’s exosome work is positioned as the next-generation pipeline, potentially offering a cell-free option with similar anti-inflammatory and muscle-protective benefits but with simpler storage, handling, and administration.
Capricor has secured funding support from the Department of Defense and other agencies to advance its exosome programs, with multiple Investigational New Drug (IND)-enabling studies underway. With an experienced cell therapy team pivoting naturally into the exosome field, Capricor’s strategy blends decades of cardiac cell biology with the promise of next-generation delivery and regenerative platforms.
In a space where many exosome players are just beginning to tackle scalable production and clinical translation, Capricor’s established GMP processes, regenerative proof-of-concept data, and rare disease focus make it a credible player helping push exosomes from preclinical hype to clinical reality.
Kimera Labs: Exosome-Based Regenerative and Anti-Inflammatory Therapeutics
Kimera Labs, headquartered in Miramar, Florida, is a pioneer in the large-scale manufacture and commercialization of natural exosomes derived from mesenchymal stem cells (MSCs). While many players in the exosome field remain in preclinical development or industrial supply scale-up, Kimera has carved out a notable position as both a producer and translational research innovator, providing purified, clinical-grade exosomes for regenerative and anti-inflammatory applications.
Kimera’s flagship exosome product, XoGlo®, is a purified, cell-free MSC-derived exosome formulation rich in growth factors, microRNAs, and anti-inflammatory molecules. While not yet an FDA-approved therapeutic, XoGlo has been used in investigator-led regenerative protocols ranging from wound healing, skin rejuvenation, and orthopedic soft-tissue repair to early research in ophthalmic and neurologic applications. Its platform demonstrates the practical promise of exosomes as off-the-shelf biologics for tissue repair and immunomodulation.
What sets Kimera apart is its vertically integrated model: the company controls the entire process from donor cell sourcing and exosome isolation to large-scale purification and GMP-compliant manufacturing. Its proprietary purification and quality control processes focus on removing cell debris, proteins, and residual DNA to yield consistent, ultra-pure exosome batches, an area that has historically challenged other players.
In parallel with its supply business, Kimera invests in translational science, funding preclinical and early-stage clinical research into how MSC-derived exosomes can modulate inflammation in diseases like chronic wounds, osteoarthritis, and neurodegeneration. The company also supports regenerative medicine practitioners, providing exosomes for use in IRB-approved protocols while advancing discussions around regulatory pathways for standardized exosome-based therapeutics.
While the field wrestles with regulatory clarity for naturally sourced exosomes, Kimera Labs stands out for demonstrating practical routes to industrial-scale exosome purification and building commercial awareness for exosome biologics beyond the lab. With its combination of robust manufacturing, early research, and a scalable supply chain, Kimera is helping set the stage for the broader adoption of exosomes in regenerative and precision medicine.
Aegle Therapeutics: Clinical-Stage Cell-Derived Exosomes
Aegle Therapeutics, headquartered in Miami, Florida, is a standout among exosome biotechs for advancing one of the few clinical-stage exosome therapeutics focused on treating rare and severe skin disorders. Its lead candidate, AGLE-102™, uses exosomes naturally secreted by allogeneic mesenchymal stem cells (MSCs) packed with proteins, microRNAs, and lipids that play critical roles in modulating inflammation and promoting tissue regeneration.
AGLE-102 is being developed initially for dystrophic epidermolysis bullosa (DEB), a devastating genetic skin disorder characterized by extreme skin fragility, chronic wounds, and scarring. Patients with DEB lack collagen VII, which is needed to anchor the skin’s layers together, leading to painful blistering and non-healing wounds. There is no cure, and current treatment is limited to supportive wound care, making this an area of urgent unmet need.
Unlike conventional cell therapy approaches that require direct transplantation of living stem cells, Aegle’s strategy isolates the therapeutic benefits of MSCs through their exosomes alone. These nano-sized vesicles carry regenerative and anti-inflammatory signals to the injury site without the complexities and risks associated with cell engraftment, immunogenicity, or tumorigenicity.
Preclinical and early clinical data indicate that AGLE-102 can promote wound closure, reduce inflammation, and support healthier tissue remodeling in DEB and potentially other severe wounds. In 2022, Aegle received FDA clearance for its phase I/II trial, a landmark milestone demonstrating that an exosome-based product can reach human studies under the FDA’s CBER framework.
Beyond DEB, Aegle is exploring broader applications for its platform in burns, chronic non-healing ulcers, and surgical wound healing, where the regenerative capacity of MSC exosomes may offer an alternative to synthetic dressings or skin grafts. The company’s allogeneic sourcing and scalable manufacturing process are designed to ensure consistent exosome quality and potency while maintaining GMP standards.
By advancing a biologically active exosome therapy rather than using exosomes purely as inert delivery vehicles, Aegle is helping to validate the concept that exosomes themselves — when sourced, purified, and formulated correctly — can be therapeutics in their own right. This approach not only expands the possibilities for regenerative medicine but also sets a clinical precedent for the next wave of cell-free, exosome-driven therapies.