One giant LEAP for regenerative medicine

Melbourne-based regenerative medicine company Exopharm has finalised testing and lodged the complete specification for its disruptive LEAP technology, taking a further step in bringing extracellular vesicles as an acellular therapy to treat patients with degenerative conditions.

According to the company, the medical need for innovative, safe and effective regenerative medicine has largely not been met by using cells such as adult stem cells, despite significant investment and decades of clinical trials. Cellular therapies are where cells from the patient or a donor are injected into a patient to trigger regeneration, but these cellular treatments can have many problems such as rejection by the host, damaging fibrosis and high costs.

More recently, researchers investigating the way that injected cells work inside the body have made discoveries that overturn previous models. The cells themselves are not needed to trigger the regeneration; rather, it is the secretions of the cells that have the main effects.

Dr Ian Dixon was involved in the cell therapy field for more than a decade before taking on the challenge of acellular therapies in his new venture, Exopharm, in 2013. He revealed, “I saw that the promise of cell therapies just wasn’t being realised and the problems with cell therapies for regenerative medicine were escalating — it was time to think outside the square.”

Dr Dixon and his team at Exopharm have been working on a disruptive technology that sidesteps the problems and limitations of cellular therapy, harnessing extracellular vesicles produced by adult stem cells in a bioprocessing facility as an acellular (ie, no cells) product. Exopharm calls its acellular product Exomeres, and has already been shown to be safe and effective in studies across conditions such as kidney disease, fibrosis, wound healing, vascular disease and neurodegeneration.

Exopharm has worked for more than three years to solve the biggest problem in the field — how to isolate and purify intact Exomeres in large scale and at low cost. This quest has resulted in the patent-pending LEAP technology that extracts pharmaceutical-grade Exomeres from cells in a fraction of the time taken by conventional methods.

“I was determined that we could solve this problem and our team has applied some really complex chemistry and innovative screening techniques — our first efforts failed big-time but we kept trying,” said Dr Dixon. “We have been fortunate to have such dedicated and expert people in our biotechnology team, and access to equipment like electron microscopes located in the Parkville precinct.”

Having further optimised and protected its LEAP technology, Exopharm is now moving to the next step in the commercialisation of its product and preparing for clinical trials of Exomeres in age-related macular degeneration (AMD) in 2020 or earlier.

“We are seeing a number of long-established cellular therapy companies moving away from cellular products and into acellular vesicle products,” said Dr Dixon. “However, each of these companies faces the challenge of how to isolate and purify vesicles secreted by their cells during cell culture — a problem our LEAP technology solves.”

Ultimately, Exopharm aims to be a world leader in the manufacture and use of extracellular vesicles derived from adult stem cells. The company has so far been approached by groups in China, Europe and the US wanting to use the LEAP technology under licence, and discussions are underway.

Exopharm is presently 94% owned by Dr Dixon and has been funded internally so far by his private investment company Altnia Group. He stated, “Exopharm is likely to become a listed company in 2018 and then transition to the USA once we have clinical trials underway.”

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