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2025-04-17|

Exosomes: The Small Couriers Moving Beyond Traditional Drug Delivery

by Bernice Lottering
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Image source: Getty Images

Over the years, drug delivery systems like liposomes, micelles, and synthetic nanoparticles have helped improve the way medications are absorbed and distributed in the body. However, these systems come with a set of problems. They often trigger immune responses, struggle to cross biological barriers, and sometimes deliver drugs to the wrong place. These issues limit their effectiveness, especially for complex diseases like cancer or neurodegenerative disorders. Enter exosomes —natural, nanosized vesicles released by virtually all cells in the body.

These biological “couriers” are between 30 to 100 nanometers in size and travel through fluids like blood, saliva, and urine. Originally dismissed as cellular waste, researchers now know they play a huge role in cell-to-cell communication. More importantly, they can carry therapeutic cargo, including drugs, RNA, and proteins, directly into target cells.

Take Parkinson’s disease, for example. Dopamine can’t easily cross the blood-brain barrier (BBB), but when it’s packaged into blood-derived exosomes, it can. In animal models, this method improved dopamine delivery to the brain and helped restore motor function—something conventional pills can’t achieve alone.

Why Exosomes Work So Well

In an article published in Extracellular Vesicle, researchers highlight that what makes exosomes so appealing is their biological design, which enables them to serve as highly efficient and biocompatible drug delivery vehicles.What makes exosomes so appealing is their biological design. Their lipid membrane offers excellent protection to whatever’s inside, shielding drugs from enzymes that might otherwise degrade them. Plus, they’re naturally compatible with the human body, which reduces the risk of side effects or immune rejection.

Exosomes are also highly customizable. Researchers can modify their surfaces with peptides or antibodies to help them find and bind to specific cells. For instance, in breast cancer research, scientists used exosomes tagged with iRGD—a tumor-penetrating peptide—and loaded them with the chemo drug doxorubicin. In mouse models, this targeted approach shrank tumors by over threefold compared to standard treatment.

Another advantage is their ability to cross biological barriers. Most drug carriers can’t reach the brain or inflamed tissues, but exosomes can. This opens doors for treating brain tumors, Alzheimer’s, or traumatic injuries in ways we couldn’t before.

Real-World Medical Applications in Action

Let’s look at a few concrete examples. In one study, exosomes were used to carry curcumin—an anti-inflammatory compound found in turmeric—into the brains of mice with Alzheimer’s. Not only did the curcumin stay stable, but it also reduced inflammation and improved memory in the animals.

In another case, researchers used exosomes loaded with linezolid, an antibiotic, to fight off MRSA (a drug-resistant bacterial infection). Compared to free antibiotics, the exosome-bound version was more effective and had fewer toxic effects.

Beyond drugs, exosomes are proving their worth in gene therapy. Scientists have successfully used them to deliver CRISPR/Cas9 gene editing tools, miRNA, and siRNA to specific tissues. These exosomes protect fragile RNA molecules from degrading in the bloodstream and help them enter target cells more effectively.

For example, exosomes have been engineered to carry siRNA that silences genes responsible for triple-negative breast cancer progression. These were further modified with bovine serum albumin for improved targeting and stability. In animal models, this approach significantly reduced tumor recurrence after surgery.

Even protein-based therapies are getting a boost. Researchers have used exosomes to deliver antioxidant enzymes like catalase across the BBB to treat neuroinflammation. Others have used them to transport membrane proteins that help regulate immune responses or inhibit tumor growth.

From Breakthrough Science to Commercial Products

Big names in biotech are moving fast to bring exosome-based therapies to clinics. In the U.S., Aegle Therapeutics developed AGLE-102, an FDA-approved exosome product for healing burns and skin injuries. Meanwhile, Capricor Therapeutics is testing exosome-based vaccines and gene therapies for conditions like Duchenne muscular dystrophy.

Over in the U.K., ReNeuron is working on ExoPrO, a system targeting cancer and neurological diseases. Australian company Exopharm has two promising exosome platforms in clinical trials: Cognevo (for brain diseases) and PlexoDOX (for cancer).

Another exciting area is vaccine development. Exosomes can be used to deliver antigens without using live viruses. This method is especially helpful for making next-generation vaccines, such as those targeting COVID-19. Researchers are now exploring multivalent protein-based vaccines using exosome platforms to improve immune response and coverage against viral variants.

The food industry is also getting involved. Plant-derived exosome-like particles from ginger, grapes, or carrots show promise in gut health and liver protection. They may one day serve as edible drug delivery tools or natural supplements.

Looking Ahead: Challenges and Opportunities

Despite the excitement, some challenges remain. Large-scale production of exosomes is still tricky. Separating them from other particles takes time and expensive equipment. Scientists are testing newer methods like microfluidic devices and immunoaffinity techniques to isolate exosomes more efficiently.

Standardization is another hurdle. Since exosomes vary depending on the cell they come from, it’s important to develop protocols that ensure consistent quality and safety—especially for clinical use.

Still, the future looks promising. Exosomes could soon become the gold standard for delivering medications, editing genes, healing tissues, and even creating safer vaccines. Their ability to navigate the body naturally, without triggering unwanted reactions, makes them stand out in a crowded field of drug delivery systems.

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