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Porosome Therapeutics – Creating a Therapeutic Approach to Issues with Porosomes and Generating Compelling Results in Treating Cystic Fibrosis and Type 1 and Type 2 Diabetes

Gil Marmol

President and CEO

Porosome Therapeutics, Inc.


Guillermo Marmol

916-601-8282 or 214-394-4549

Interview conducted by:

Lynn Fosse, Senior Editor

CEOCFO Magazine

Published – September 18, 2023

CEOCFO: Mr. Marmol, the first thing I see on your site is "Porosome Discovery Promises to Revolutionize Modern Medicine," would you tell us what you found?

Mr. Marmol: The porosome is the universal communication portal and secretory machinery of the human cell. Secretions in each cell have to be communicated to other cells. It is a nanomachine, so it is extraordinarily small. Its history has been framed around the work of Prof. Dr. Bhanu P. Jena, who is the lead scientist in support of our company.

You and I have porosomes in all of our cells, and when the porosomes work correctly, there are no issues. However, when the porosome begins to fail, and there can be a variety of reasons for that including genetic reasons, it can cause an array of health problems associated with secretory diseases. An example of those diseases that we are working on now are cystic fibrosis and Type 1 and Type 2 diabetes.

Because of the central role that the porosome plays in intracellular communication, we believe that it is the foundation for a new platform of science that is built around addressing issues that arise with the porosome. There are somewhere between thirty and forty proteins in the porosome and we believe those thirty or forty proteins can result in up to one hundred different disease indications, two of which are the ones that we are working on at the moment.

CEOCFO: If the porosome is so important, why isn’t it common knowledge?

Mr. Marmol: The basic science surrounding the porosme has been carried out over the last two decades including the discovery of the porosme in 1999. The porsome is now incorporated into some of the basic teaching text in U.S. medical schools. However, the reason most have not heard of the porsome is that it is only in the last few years that the scientists who have worked on basic research have turned their attention to creating therapeutic solutions around the porosome.

CEOCFO: What has become available in terms of therapeutics, that this is getting attention in the last few years where it has not before?

Mr. Marmol: Scientists have become more aware of the centrality of the porosome intracellular communication in certain disease states. It is not unusual for that to happen in science. MRNA was around for a long time before there was a successful therapeutic associated with it. The reality that there were a few decades of work done on the porosome in a research environment is not surprising. It is representative of this kind of science.

What has happened over the last three years or so is that, as a result of Dr. Bhanu P. Jena and his associates’ research, there was a fundamental change in his perspective about the world. I am paraphrasing him here a bit when he said that he was no longer content with having the work that he does and the results that he accomplished only reflected in a notebook, scientific journal, or publication. He wanted to see the results of this work felt in the world and benefit people.

The principal achievements in our laboratory over the last couple of years is generate some compelling results in cystic fibrosis and Type 1 and Type 2 diabetes.

CEOCFO: Why are those the indications you have looked at first?

Mr. Marmol: There are a couple of different mechanisms of action for dealing with a porosome and creating porosome-based therapies. One of the most revolutionary and exciting is what we call porosome reconstitution. Think for a moment that you had an automobile that had a bad tire. What we can do with porosome reconstitution is not remove the bad tire but extend the axel and place on the extended axel a new tire so that the automobile now runs on the new tire and not the old damaged tire. As a result, you have, in effect, displaced the malfunctioning porosome with an entirely new functioning porosome.

That radical new methodology for therapeutic results is particularly relevant in certain situations where you have disease states like cystic fibrosis and Type 1 diabetes. The original peer-reviewed science surrounding the creation of the reconstituted porosomes was published in 2016 in the Journal of Endocrinology and is therefore highly relevant to Type 1 diabetes.

With regard to cystic fibrosis, our approach was to introduce functional human secretory machinery with the associated functional CFTR protein – jointly the porosome complex – into CF-diseased human lung cells which are called mucus-secreting airways epithelial cells. We are pleased to report to you that these studies carried out in human cells have turned out to be successful. The implanted porosomes restore nearly 100% of the secretory functions in diseased cells providing, for the first time, a pathway to bringing a cure for cystic fibrosis. This therapeutic approach applies to treating all CF mutations and has the potential of reaching 100 percent of CF patients globally.

The short answer to why we went in this direction was because we thought that some of our more revolutionary science had the greatest impact on these types of diseases.

CEOCFO: How do you get the reconstructed porosome into the body in the right place?

Mr. Marmol: It depends on what part of the body we are talking about. With cystic fibrosis, the more advanced view of how to do that would be through the lungs, so it would be applied through the lungs. We can successfully implant the porosomes into existing cellular structures that will replace or displace through the reconstitution process of the original porosome.

Because the epithelial cells in the lungs are long-lived, we believe that the treatment may only be required either once a year or once every year and a half, or along those lines. While we are cautious about using words like “cure,” it does have long-lasting effects.

CEOCFO: Is there a certain point in time in the progression of the disease that you would need to do this or might it be done at almost any time?

Mr. Marmol: We believe that the methodology is effective without regard to the development of the disease. If we were medical doctors, which we are generally not, we are mostly research scientists, there would be considerations around the strength of the patient, and other medical considerations, which would be applicable to when our therapy would be most effective and most highly desirable.

In terms of its capacity to help a patient, that is not dictated by whether the patient is fifteen or twenty-five, or thirty-five. That is not the parameter that we believe is relevant to where the efficacy of this therapy would be.

CEOCFO: What surprised you as reached this point in development?

Mr. Marmol: To be honest, what has surprised me, is that our results are so compelling. If we look at the big picture of the science and research that has been done for multiple decades, and the multiple scientists around the world that have been committed to doing this work, then it has not happened very quickly.

I think that the results we have exhibited and that we filed in our initial intellectual patent application that we made public, happened quite quickly in the context of people who have been working on this for a long time.

CEOCFO: What has been the response from the medical community?

Mr. Marmol: To be fair, we were cautious until very recently, and the reason we have been careful in describing what we have accomplished is that we wanted to make sure that we had done the proper work and established intellectual property and filings associated with that science.

We expect to be engaging members of the cystic fibrosis community. It is a relatively small community and the parties are well-known to each other.

CEOCFO: What are your next steps with the science and what are you working on in the lab today?

Mr. Marmol: The immediate next step is to go from the in vitro studies that we have conducted in our lab to in vivo studies with mice. There are two diseases that we have identified and that we will be working with. One is cystic fibrosis and one is Type 1, Type 2 diabetes. The organization of the relationship and the lead time for the cystic fibrosis initiative has gone further and faster. We believe that we will be beginning those in vivo studies shortly. The studies in Type 1 and Type 2 diabetes will take somewhat longer.

We are particularly excited about our work on porosome reconstitution that has demonstrated high levels of effectiveness in cell line studies in our laboratory recently. We were confident in our approach but now we have the results, and we will make the porosome reconstitution therapeutic approach a central part of the in vivo studies we are initiating.

We believe that both of those studies are expected to be completed in or around the first quarter of next year. At that point, we would be ready to engage with other partners. Those partners could potentially be other pharma companies that are interested in this arena or venture capital firms that invest in biotech, or foundations that might be interested in participating with us in our work. We would be seeking those kinds of relationships to strengthen our position to carry this forward into Phase 1 and Phase 2 human trials.

CEOCFO: Porosome Therapeutics, Inc., Chairman, Prof. Bhanu P. Jena, PhD, and Chairman of the Scientific Advisory Board, Prof. Dr. Joachim Frank, are going to be at the Nobel Prize Dialogue, and participating. What is the Nobel Prize Dialogue, and how important is it to get the story out?

Mr. Marmol: We think it is very important. The speech that our Chairman, Prof. Dr. Bhanu P. Jena, will be making in Seoul on September 24th, is an invitation that came to him through the collaboration of the Nobel organization that is responsible for the Nobel Prizes and the Korean Academy of Science and Technology. So it was a joining of the Nobel organization and the Korean Academy of Science and Technology to identify certain senior scientists who have made particularly meaningful contributions and to invite them to Seoul. I will be there with them as well.

Dr. Jena has spoken on a variety of things, but at that forum on September 24th, he is going to be speaking on the role that education and science can have in broadening the reach of science.

CEOCFO: What if anything might people miss or misunderstand about what you are doing at Porosome Therapeutics that should be recognized?

Mr. Marmol: The casual observer might underestimate how central the porosome is to so many disease states and how important a therapeutic approach would be, which is what we are committed to achieving with our science. In addition, it can have this impact on a whole range of diseases over time.

CEOCFO: Final thoughts?

Mr. Marmol: We are at the start of the beginning. The promise that we are making today is the first step in a long road that I think will have very significant benefits for humankind, across a whole range of disease indications. We are proud to be part of that journey.

Porosome Therapeutics, Inc. | Gil Marmol | Cystic Fibrosis Porosome Research | Cystic Fibrosis Porosome Testing | Type 1 diabetes Porosome Testing | Type 2 diabetes Porosome Testing | Porosome Therapeutics – Creating a Therapeutic Approach to Issues with Porosomes and Generating Compelling Results in Treating Cystic Fibrosis and Type 1 and Type 2 Diabetes | CEO Interviews 2023 | Medical Companies | Cystic Fibrosis Porosome Linkage Testing | Porosome Therapies | Porosome diseases, cellular communication | The cell’s secretory apparatus | defects in the porosome | a cellular secretory portal essential for intercellular communication | Porosome Therapeutics, Inc. Press Releases, News

“We are particularly excited about our work on porosome reconstitution that has demonstrated high levels of effectiveness in cell line studies in our laboratory recently. We were confident in our approach but now we have the results, and we will make the porosome reconstitution therapeutic approach a central part of the in vivo studies we are initiating.”
Gil Marmol