ORLANDO, Florida—A new-generation gene therapy to treat Duchenne muscular dystrophy (DMD) was introduced by Genine Winslow, MSc, chief executive officer and president of Chameleon Biosciences, at the Next Generation Technology session of the CureDuchenne 2022 FUTURES National Conference.
“I founded Chameleon about 5 years ago. My dream then was to be able to treat all patients with gene therapy,” Winslow said.
Adeno-associated virus (AAV)-based gene therapies have several limitations. These include pre-existing immunity against AAV in some patients, the need for high-dose therapy (which is costly to manufacture), issues associated with the durability of the treatment, and the fact that patients are mostly growing children and treatment cannot be given early in life.
Chameleon’s EVADERTM technology is designed to elicit a minimal immune response, Winslow explained. This can allow repeat dosing and improve the efficacy of the gene therapy.
The viral particle has an extra layer surrounding it which includes 2 potent immune suppressor molecules used by tumor cells to evade immune detection, Winslow said.
Read more about gene therapy and other experimental therapies for DMD.
She stressed that Chameleon’s technology is built on the knowledge gained from other gene therapies currently in use.
Winslow also presented data from preclinical studies testing the company’s technology. She said that in mice, the second dose of the treatment was “just as effective as the first,” which does not happen with other technologies available today. The treatment also seems to be safe in larger animal models and reduces B- and T-cell-mediated immune response as well as inflammation, she said.
She added that, based on these results, no patient will be excluded from clinical trials testing the new technology because of the presence of neutralizing antibodies in their blood. “I am very excited to be able to take the field in that direction,” she said.
What’s next? Next-generation technology panel. CureDuchenne 2022 FUTURES National Conference; May 29, 2022; Orlando, FL.