“Here in Chameleon Biosciences, we are science-oriented and patient-oriented, ”said Gene Winslow, founder, CEO and chairman of the gene therapy company. “Instead of hoping for an empty claim, we are really going for a walk. We set our mission to look at gene therapy from a family perspective. ”
Since the founding of Chameleon in 2017, Winslow and her team have been working to develop an innovative approach to gene therapy called EVADER ™. By combining exposures designed by the virus with molecules that suppress the immune response, EVADER violates the traditional limit of gene therapy in a single dose, offering treatment to patients for whom gene therapy would otherwise be unavailable.
Winslow has been involved in a long history of exciting innovations in the field, beginning her career in a gene therapy startup 30 years ago. “I’m a bit of a scientist and I think viruses are very cool,” she said in an interview with Biospace. “When you look at viruses as a biological organism, they are amazing. Of course, with viruses like COVID, they can also be scary. We can never underestimate how smart they are. ”
Winslow never lost sight of the patients she fought for, despite the fact that she was driven by science. “Patients play a central role in drug development. Our task is to try to eliminate the cause of genetic diseases. In the most severe cases, we deal with young children who do not live to adulthood.
One day, when Winslow was taking a tour of his former lab for one of the scientists, he shared a proposal that would change her life forever. His young son was diagnosed with X-linked myotubular myopathy, a rare genetic disease that weakens muscles, leading to impaired motor skills and severe breathing difficulties, leaving many patients unsustainable after early childhood.
“Scientists are usually analytical and dependent on data. There aren’t many feelings here; either you answered the question or you didn’t, “said Winslow.” I still remember the exact words of this scientist. After he heard about his son’s diagnosis, he said, ‘My world has turned black.’ “Coming to the world through the rules of nature, to say ‘my world is blackened’ is incredibly strong. I remember thinking about my own children at the time. They’re babies. I knew we had to do better,” she said.
Unfortunately, the young child has since passed away, but Winslow has done the work of his life to pave the way for others, to increase the effectiveness of gene therapy in order to save the most vulnerable sections of society.
“Gene therapy works surprisingly well in a very limited way. We can treat the cause of genetic diseases by providing the correct version of the gene. We use stabbed viruses as taxis so that the correct version of the gene can enter the cell and edit it to correct the disease. We replace the gut with a corrective gene. Because it is a Frankenstein virus, it cannot create more viral particles. However, due to the limitations of this first-generation technology, there are patients who cannot be treated. ”
Current restrictions mean that patients can only be given a single dose. “Gene therapy uses the virus to act as a shuttle for DNA,” said Monica Miller, senior vice president and director of program development and operations at Chameleon. “We use environmental viruses that are everywhere. Once you are exposed, an immune response occurs in the body, which means we cannot treat people who have already been exposed. ”
This traditional single-dose reality of gene therapy can be detrimental to many. “One dose should be enough for a patient for life, enough to break the disease. There is only one chance to do it right. Because of this, many physicians want their patients involved in clinical trials to receive the highest dose. High doses can trigger a serious immune response, so are not always ideal for children. Some children have been able to get a small correction of the disease, but this is usually not enough, ”Winslow added.
With EVADER, the Chameleon team hopes to make a one-time treatment a relic of the past. “Our virus hides an extra layer that physically protects it from antibodies,” she explained. “We used two molecules from immunoancology, CTLA-4 and PD-1, which are normally blocked to boost the immune response. We wanted to be sure we were beating the immune response with a hammer. We have introduced these molecules into our drug as potent immune suppressors, allowing us to repeat the dose effectively. ”
This ability to penetrate the patient’s immune system allows several times to offer EVADER to patients. In addition to this, the technology uses a lower dosage, which is less likely to trigger a serious immune response and is therefore expected to be safer for children. It also means that adults who have previously been exposed to AAV could be successfully treated. Chameleon says the therapy shows resistance to neutralizing antibodies, allowing products made with the platform to potentially treat such patients.
With several women on the executive board, Chameleon serves as an example of a leader in the life sciences industry. Other women at STEM Winslow are advised to “do what you love. If that’s what you love, don’t give up. For women in senior management positions there is a glass ceiling. Go to the management teams and go to the boards. No one can do it alone. Developing drugs is difficult, and a team approach is important. More smart people at the table are better than one smart person. Continue. Don’t give up! ”
Miller urged women at STEM to “continue to pursue the cultural change we need”. As an example, she described an awkward situation that occurred during one of her first jobs after graduate school. “One supervisor told me that you can’t be a good scientist if you’re not in the lab for 12 hours a day. This is simply not true. Abandon these cultural norms. If you are interested in something, you can find a way to make it your life, ”she said.
Winslow said: “Combining to do what I really enjoy, while being able to contribute something to society, is an ideal situation in many ways.”