Exosomes

The use of extracellular vesicles for regenerative and therapeutic applications is gaining currency. Notably, exosomes derived from mesenchymal stem cells (MSCs), have garnered attention. However, even manufacturing exosomes for relatively small-scale, preclinical and clinical activity has proved challenging. How will scalable production of vast quantities of exosomes for routine therapeutic use be achieved?

Blood is a complex, dynamic mixture of cells, proteins, ions, sugars, hormones, nutrients, gases and more. The composition of blood constantly varies in response to our diet, exercise status, hydration, time of the day, injury and challenges from pathogens. As well as its role in mammals, blood products such as serum and albumin are important reagents for cell culture. What are the components of blood? Where do these components of blood come from?

Acute pancreatitis (AP) is a sudden and severe inflammation of the pancreas sometimes caused by gallstones. In the most severe cases, AP can also lead to injury of the lung. The pathway from pancreatic disease to lung injury is anything but direct and a fundamental role for exosomes has emerged.

Ultracentrifugation is often described as the “gold standard” to isolate or purify extracellular vesicles (EVs) including exosomes. Now, a collaborative EV study led by researchers at Clemson University in South Carolina, has shown that for purification of EVs from urine samples, this gold standard can be improved with a simple modification.

In this report, we provide details on 45 companies that have emerged over the last decade to pursue exosome therapeutics and diagnostic goals. Two tables provide a quick reference for this information on investor funding and founding date. A further table provides a summary of 242 exosome-focused clinical trials.

Therapeutic RNA delivery can be accomplished by a variety of viral or non-viral methods. The type of RNA structure carried by these diverse delivery methods also varies (e.g., oligonucleotides, miRNA, siRNA, lncRNA, mRNA, saRNAs). The particular delivery method used is dependent on the types of RNA and the target and is a hugely important consideration for the development of effective drugs.

Exosomes represent a potential sweet spot between small molecules, biologics, and cell therapies for a future as highly potent multifaceted biopharmaceuticals. Studies that are leading towards clinical applications have raised expectations that exosomes will eventually provide treatment for a wide range of orphan diseases. From basic research to clinical application, the ability to stably store exosomes whilst maintaining their unique characteristics is fundamental to their research and therapeutic development.

Exosomes and viruses have many things in common, including their size, lipid coat, and the ability to transport RNA and proteins between cells. Exosomes and viruses co-purify using techniques including size exclusion chromatography. Are these similarities a coincidence or is there a shared past?

Fetal maturation rate and birth timing are both regulated by factors including hormones, nutrients, and adipokines. Increasingly, the contribution of exosomes to the establishment of a successful pregnancy and delivery process is becoming clear. Exosomes regulate the physiology and metabolism of both the mother and the fetus by acting as messengers that carry specific biological signals between cells/tissues—generating an intimate relationship between them.

Regenerative medicine aims to address the underlying causes of a patient’s condition to replace lost tissue and restore normal function. But the challenge for regenerative medicine of translating basic research into clinical therapies is immense.