CureDuchenne backs immune-resistant AAV gene therapy platform

By Vassia Barba

- Last updated on GMT

(Image: Getty/Marcin Klapczynski)
(Image: Getty/Marcin Klapczynski)

Related tags Duchenne muscular dystrophy Drug development Gene therapy cell and gene therapy

CureDuchenne invests in Evader, a gene therapy platform created by Chameleon, which bypasses the immune responses obstructing repeated dosing.

CureDuchenne, a non-profit organization, has agreed to invest in California, US-based Chameleon Biosciences, a pre-clinical stage company with platform technology for the development of treatments with several indications, including Duchenne muscular dystrophy.

Evader is an adeno-associated virus (AAV)-based gene therapy platform technology that aims to overcome the ‘fundamental’ problem that current treatment approaches suffer from, the company stated.

More specifically, although viruses deliver the replacement gene required, an immune response is caused that prevents repeat dosing.

Treatments developed with the use of Evader appear more resistant to pre-existing immunity, as well as less immunogenic than current AAV gene therapies, enabling repeated dosing. Additionally, the therapies are more efficient, allowing the distribution of smaller doses.

“If successful, Evader technology will treat a greater number of patients, [...] for widely expanding potential disease applications,”​ CureDuchenne stated.

Debra Miller, CureDuchenne’s CEO, commented that the organization decided to support Evader after seeing ‘highly encouraging’ data.

On her side, Genine Winslow, CEO of Chameleon, said that CureDuchenne’s undisclosed funding will be used to further develop Evader.

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