Regenerative Medicine: Questions and Answers
What is regenerative medicine?
Regenerative medicine is a term used to cover different ways scientists can harness the body’s own regenerative capabilities.
What are some examples of regenerative medicines?
An old example is blood. Blood (or blood plasma) from donors is used routinely in hospitals if the patient needs more blood urgently. Another example would be cell therapy, where cells are injected or infused into the patient to trigger regeneration or replace lost cells.
What problems does regenerative medicine seek to solve?
Examples would be osteoporosis (bone density reduction), sarcopenia (muscle loss), arthritis, neurodegeneration, diabetes, heart degeneration, macular degeneration, tendonitis (degeneration of tendons), autoimmune disease (e.g. multiple sclerosis). Many of these are age-related conditions.
Why are there so many medical problems still poorly treated?
This is true, and needs to change. Finding new answers to old problems requires innovation and new thinking. At Exopharm we believe that exosomes (extracellular vesicles naturally secreted by cells and naturally present in our bodies) are a potential game-changer innovation in medicine.
Why are there no exosome products on the market for regenerative medicine yet?
Before a new drug can be sold (registered for sales), it needs to progress through clinical trials to demonstrate safety and efficacy. Running clinical trials through to registration typically requires treating hundreds (or even thousands) of patients.
Whilst exosomes are naturally occurring and plentiful, the technology to harvest and purify exosomes as a proper biologic product has been missing until recently.
Only when the “purification technology” problem is solved can sufficient exosomes be available to run proper clinical trials.
What is the “purification technology” problem?
Exosomes are invisible nano-sized natural lipid particles produced by stem cells and other cells in our bodies.
Whilst exosomes are described as “cell juice”, the limitation has been a proprietary commercial-scale purification process that produces a biologic product.
The process needs to be proprietary (i.e. patentable) to protect the investment to run clinical trials and commercialise. The process needs to be commercial-scale, as bench-scale production is unsuited to treating millions of patients.
The process needs to produce a biologic product – not some heterogeneous variable “soup” of molecules and contaminates.
Why is Exopharm seen as an innovator in this field?
Exopharm has acquired the LEAP Technology from Dr Ian Dixon. The LEAP Technology solves the “purification technology” problem and is a proprietary commercial-scale purification process that produces a biologic product.
Other companies and researchers are using purification technologies that are different from the LEAP Technology, and in many cases clearly inferior (e.g. ultracentrifuge or tangential flow filtration).
Having solved the “purification technology” problem, Exopharm is positioned to manufacture its exosome products (Plexaris and Exomeres) and run clinical trials to test safety and efficacy of these biologic products.
What is Plexaris?
Plexaris is our trade mark name for the exosome product from blood platelets purified with our LEAP Technology.
What is Exomeres?
Exomere is our trade mark name for the exosome product from stem cells (not embryonic stem cells) purified with our LEAP Technology.
How do Plexaris and Exomeres compare ? Why have both?
We expect that Plexaris may suit treating some conditions better than Exomeres, and may also be less expensive to produce. The strategy is to partner Plexaris and Exomeres separately, leaving room for two transactions instead of one.
Why does Exopharm believe its products will be safe?
Previous testing of platelet derived and adult stem cell derived exosomes/extracellular vesicles in animal studies have not demonstrated safety problems or toxicity to date.
Exopharm is presently conducting testing of its own. There have also been some recent small-scale human studies involving adult stem cell derived exosomes/extracellular vesicles and no serious adverse events were recorded in publications.
Why does Exopharm believe its products will work?
Previous testing of platelet derived and adult stem cell derived exosomes/extracellular vesicles in animal studies have demonstrated signals of efficacy in a range of models.
There have also been some recent small-scale human studies involving adult stem cell derived exosomes/extracellular vesicles and these studies have demonstrated signals of efficacy in a range of medical conditions including chronic kidney disease and graft versus host disease.
Is there interest from bigger companies?
Yes, some of the bigger companies are following the field.
Will Exopharm sell exosomes to others?
Our present strategy is not to supply clinical grade exosome product(s) to potential competitors. However, there may be ways to support companies we see as outside of our own focus areas.
What is the difference between autologous and allogeneic?
Autologous means the donor and the recipient are the same person (i.e. matched). Allogeneic means the donor and the recipient are different people.
What is a biologic product?
Biologic products come from living things e.g. proteins from cells. A synthesised peptide is not a biologic. Exosomes are biologics because they are secreted by cells naturally.
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