Exosome illustration (National Cancer Institute)
26 September 2016. A biotechnology company is receiving a Department of Defense grant to develop a commercial-scale manufacturing process for exosomes, tiny bubbles that deliver drugs to cells in the body. The U.S. Army’s medical research agency awarded Capricor Therapeutics Inc. in Los Angeles some $2.4 million to establish a manufacturing process for exosomes, nanoscale bubbles of natural oils called lipids.
Exosomes are vesicles, tiny — 40 to 150 nanometer — lipid-membrane containers in cells that gather up and secrete cytoplasm, the gel-like material outside the cell nucleus. While originally believed to carry out waste removal and other maintenance tasks, exosomes were shown in recent years to perform useful delivery functions carrying proteins and genetic material to other cells, and drawing increased attention from a range of biological disciplines.
Capricor is a biotechnology enterprise specializing in regenerative medicine, in particular the extraction of cells known as cardiospheres producing stem cells that are introduced back into the coronary arteries of the patient. Capricor says cardiosphere-derived stem cells release growth factors and cytokines, cells that stimulate specific cell responses, signaling the heart to repair itself.
The company is licensing technology from Cedars-Sinai Medical Center in Los Angeles that also extracts exosomes from cardiosphere-derived cells. The company’s first application of exosomes is ocular graft-versus-host disease, a disorder, an immune system disorder that occurs after stem cell or bone marrow transplants. Complications of graft-versus-host disease affecting the eyes include dry eye syndrome and acquisition of eye-related allergies from donors. People with grant-versus-host disease may need to take drugs that suppress the immune system, which raises the risk for infection.
The U.S. military is interested in exosomes as vehicles for reliable, self-contained drug delivery and diagnostics in the field. Among the applications being considered are “nanobots” transported by exosomes with small interfering RNAs, genetic content, or fluorescent reporters. An essay published by the Army earlier this year finds “intriguing” the prospect of manufacturing exosomes loaded with diagnostic or therapeutic payloads.
Capricor first exosome product for ocular graft-versus-host disease is code-named CAP–2003, for which the company expects to file an investigational new drug application in 2017. Linda Marbán, Capricor’s president and CEO says in a company statement, “As a cell-free material, CAP-2003 can be handled and stored in similar fashion to a traditional biopharmaceutical, and may be better suited than cell-based therapeutics for certain applications.”
Marbán adds that “As we advance and expand our exosomes program, this grant from the DoD will support our objective of achieving a commercial manufacturing process for a candidate which we believe has broad treatment potential.”
Read more:
- Lung Tissue Grown in Lab from Stem Cells
- Stem Cells Aid Motor Functions in Spinal Cord Patients
- Stem Cells, Engineered Protein Reverse Stroke Damage
- Trial Underway Testing Stem Cells to Treat Alzheimer’s
- Stem Cells, Gene Therapy Produce Replacement Joint Cartilage
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