Chameleon Biosciences


Chameleon Biosciences
San Francisco

Showcase
Chameleon Biosciences

Chameleon technology allows access to a greater number of disease applications and larger patient populations within each application. Focus will be orphan disease indications. The first clinical application will be adult severe Hemophilia B followed by severe Hemophilia A in patients that have pre-existing antibody levels too high to be treated with current AAV therapies. Once safe and efficacious repeat dosing is demonstrated in adults, Chameleon will target treating children with severe Hemophilia type A and B. Repeat dosing in children will allow Chameleon to access younger patient populations inaccessible to current AAV therapies. The projected total annual accessible Hemophilia market is about 20B per year. The second clinical target will be severe X-linked Myotubular Myopathy, a rare genetic disorder that affects muscles and results in death by about 30 months of age; and Niemann Pick Type C1, a rare progressive genetic disorder characterized by an inability of the body to transport cholesterol and other fatty substances (lipids) inside of cells. This leads to the abnormal accumulation of these substances within various tissues of the body, including brain tissue, leading to cell death and progressive deterioration.