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CEOCFO Magazine, PO Box 340
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Lynn Fosse, Senior Editor
Steve Alexander, Associate Editor
Bud Wayne, Marketing
& Production Manager
Christy Rivers -
Chameleon Biosciences, Inc.
Chameleon Bioscience Contact:
Interview conducted by:
Lynn Fosse, Senior Editor
Published – April 25, 2022
CEOCFO: Ms. Winslow, what was the concept when you founded Chameleon Biosciences, Inc. in 2017?
Ms. Winslow: When I founded Chameleon Biosciences about five years ago, I was working in the field of gene therapy on a program treating children suffering from a fatal genetic disease. These children did not live beyond two years. This patient population had an extreme unmet need, and we were working on a potentially lifesaving treatment for them.
However, at the time, I saw that the people working on gene therapy were not considering the immune aspects of their products. To put it simply, the immunologists and the gene therapy scientists were not sharing their knowledge with each other. I have a background in both fields as I had done graduate work in immunology and then I spent an entire career developing early-
That is what got me started on Chameleon. I thought that we could better serve the patient population by bringing the two fields-
CEOCFO: Where are you today? How has that played out for you so far?
Ms. Winslow: It turns out that we were spot on in anticipating that the immune response to gene therapy drugs is very important; it is perhaps the most important obstacle that this field of Adeno-
CEOCFO: How are you looking to move forward with the idea?
Ms. Winslow: It was a pretty big idea at the time. Technically, we are not actually doing anything that had not been done before; just bringing together different components, if you will. We designed a drug that incorporated what we have learned from different fields, such as the behavior of cancer cells that evade detection from immuno-
Our first big goal was to show that we could make this product, because it was something that was groundbreaking and paradigm-
In current gene therapy technology, manufacturing is the biggest risk in funding and growing our types of companies. Our investors have been steadfast and completely supportive in these efforts, and we are now in a position where we can make our product because its composition is very well understood and very well-
Our second goal was to show how well our technology platform worked; we had to show proof of concept. While we were developing the manufacturing approaches for our product, we tested it in different disease models. What we have found-
CEOCFO: Is this the EVADER™ Platform Technology that you are talking about?
Ms. Winslow: Yes.
CEOCFO: Why does coating with this extra layer work? What is the science?
Ms. Winslow: It is because, as I said, we start with a virus particle that has been gutted to act as a delivery vehicle, if you will. It shuttles the correct gene into our patient’s cells and gets it to the right place in a patient’s cells, to reverse the disease. We are starting with a virus, and even though it is a harmless virus, our patients’ immune systems see it as in the category of all viruses, and it is something that needs to be gotten rid of. Therefore, our patients’ immune systems see our drug like any other virus and attack it. This immune response is the underlying cause of some of the recent clinical setbacks in the field.
Our product adds a lipid membrane around each virus particle that effectively “cloaks” the virus so the patient’s immune system does not try to attack it and get rid of it. In addition, we attach checkpoint immune-
CEOCFO: Are there particular conditions or diseases where your drug is most effective? How have you decided what to test first? I am guessing that there are many different conditions that need attention.
Ms. Winslow: Yes, there are many different genetic diseases currently being addressed with gene therapy, and hundreds more that have not yet been worked on. In terms of what to work on first, we chose a disease that was one of the first to be treated with gene therapy about 10 years ago now, at the University College of London: hemophilia B.
Most people have heard of hemophilia. Hemophilia is a disorder where patients have a defect in a gene that is necessary for clotting blood. Normally, if most of us get a cut or an injury, our blood clots and stops the bleeding. People with hemophilia B are missing a key molecule needed to form blood clots and they bleed uncontrollably.
The reason that we chose hemophilia B as the first program is because we have the most clinical information about it, so we know how gene therapies work in humans for treating this disease. That gives us more information to start with, and to design our first clinical trial with safety as the highest priority. Remember I said our drug has a virus particle at the center? We will be using the same virus particle, the inner component of our product, that has already been used in clinical trials in humans. We know it works to reverse symptoms of the disease, and we know it is safe at lower doses in humans.
In addition, we plan to treat patients who have no other choices, meaning that we will treat hemophilia B patients who can’t be treated with the current gene therapy approaches, and treat them using our EVADER technology. We will maintain that strategy as much as possible going forward as we expand our pipeline.
In the future, we will pursue strategic alliances and partnerships to help us to test our platform to treat other types of genetic diseases as soon as possible. As the platform grows more over the long-
We have a very clear, focused strategy to get into the clinic, to demonstrate that our platform produces products that are safe and effective. Then beyond that, there is almost unlimited growth potential to utilize the platform, to treat patients and also to grow the company and get returns for our investors.
CEOCFO: How often would someone need to take the drug? Would it be on an ongoing basis? Does it depend on what condition or is that yet to be determined?
Ms. Winslow: That is a very, very good question, because the current AAV technology can only be given once in the lifetime of a patient. It was referred to as a “one-
We are finding that one-
We want to be able to treat every patient, and we are changing that one-
One difference between the way our drugs work and the way traditional pharmaceuticals work is when you talk about dose escalation with traditional pharmaceuticals, you literally start out low in a clinical trial and give higher and higher doses, to show that it is safe. We administer a low, safe dose, and then cumulatively add to that, with subsequent low safe doses. Therefore, we end up with a cumulative therapeutic effect, with a two-
To some extent, we are planning to do individualized medicine for AAV-
CEOCFO: When you are talking to the medical community, as well as the investment community, do they understand? Is there something you say where the light bulb goes off? How do you address both of these communities, so they recognize the importance of what you are doing at Chameleon?
Ms. Winslow: That is a good question, too, because of course, the medical community and the investor community have a shared mutual end goal, but how you get there is where they differ. The medical community wants to help patients to be as safe as possible. They want the very best drug that they can possibly get. We need the investment community in order to develop drugs, but they also have to see returns on their investments. We marry the conversation we have with those two by emphasizing that first of all, it is a new paradigm and a new way to treat gene therapy patients.
We have got a lot of data. Data speaks volumes. We can show in our animal models for example, that we can safely administer two doses with less of an immune response than the standard technology ends up resulting in now. It is a combination of showing that we can overcome the limitations that we have seen in the clinics with the first-
CEOCFO: Would it be harder to get people for trials as some of these diseases are not very prevalent, or is the fact that people have fewer options make it more likely someone would go into a trial? What do you see there?
Ms. Winslow: Trial participation was another reason we chose hemophilia B for our lead program. In our first clinical trial, we are planning to treat patients with higher levels of pre-
CEOCFO: How do you deal with some of the frustration personally, in knowing you have such a good idea that can help so many people, and yet, it takes quite a while to get to the point where it can actually be used?
Ms. Winslow: One thing I have learned, as a CEO and in starting Chameleon, is that you cannot ever give up. You have to persevere no matter what. I think of a parent I met. This was way back before I started Chameleon. His child was suffering from a fatal genetic disease, he was part of a patient advocacy group, and I was giving him a tour of our lab. He explained to me how, when he heard his son’s diagnosis, his world went black. Those were literally his words. His world went black.
Yes, it takes longer than I like. Yes, it is hard. I may get knocked down a lot by various parties along the way, but if I think about that parent and those children, I can pick myself up when I need to.
CEOCFO: Why should I pay attention to Chameleon Biosciences? Also, what might someone miss that really needs to be understood?
Ms. Winslow: What we are doing is unlike anything that anyone else is doing. We have very strong IP [intellectual property]. We have freedom to operate, and we have a platform with massive potential growth. We are addressing a need, a gaping hole in the gene therapy market, by striving to treat all patients regardless of age or previous exposure to the AAV virus, and believe that our products may prove to be much safer than what is used currently. Once we have shown strong clinical results with EVADER gene therapies, we plan to apply this technology to treat more common diseases, not just to rare diseases.
I think that the potential is not always completely apparent at first. The more data we get, the more I see that we should be able to safely redose patients. We reduce immune response indicators in our models. Those models also show that our efficacy is much better than the current technologies, even than our closest competitor. It is a game changer.
Initially, there was more resistance to it than there is now. I think that what I am trying to convey to investors is very new. It has been significantly de-
We are developing our own products, gene therapy drugs using EVADER technology, to generate proof of concept in clinical trials, and realize the largest value inflection point in drug development. At the same time, we cannot treat every disease. Therefore, we will strategically license or partner with large pharmaceutical companies to accelerate development of gene therapies to treat additional diseases as we grow the company to help fund internal efforts.
CEOCFO: You have all of the bases covered. A really good overall plan, taking into account the business as well as the medical aspects at Chameleon Biosciences. I do not hear this often, so kudos to you.
Ms. Winslow: Thank you very much!
Chameleon Biosciences, Inc. | Genine Winslow | AAV Gene Therapy | EVADER | Adeno Associated Virus Gene Therapy | Redosable Gene Therapy | Chameleon Biosciences -
“We are addressing a need, a gaping hole in the gene therapy market, by striving to treat all patients regardless of age or previous exposure to the AAV virus, and believe that our products may prove to be much safer than what is used currently. Once we have shown strong clinical results with EVADER gene therapies, we plan to apply this technology to treat more common diseases, not just to rare diseases.”