Our Science

Hemophilia B is bleeding disorder, caused by defects in the gene that encodes for coagulation factor IX (FIX). FIX is important for blood clotting and people with Hemophilia B suffer from frequent and spontaneous bleeding episodes.

Gene therapy strategies targeting hemophilia are inadequate as they result in strong immune responses, making them unsuitable for treating a lifelong disease.

Chameleon’s EVADER technology is intended to lower immune responses, potentially both allowing repeat dosing and enabling the treatment of children and patients with pre-existing immunity.

Niemann-Pick Type C (NPC) is a rare and devastating disease that affects the body's ability to metabolize cholesterol and lipids within cells. This accumulation causes affects neurologic and psychiatric functions, as well as various internal organs. Milder forms of the diseases are diagnosed in adults, but many children suffer from more severe forms of NPC.

NPC is often fatal and while there are some treatments being investigated, none of these treatments attack the genetic basis of the disease, and there is no cure.

Chameleon’s EVADER technology offers hope to those affected with NPC by potentially allowing for more than one dose of gene therapy.

Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE) is a genetic disease that causes a severe, multisystem disorder due to a defective thymidine phosphorylase enzyme. MNGIE characterized by degeneration of the muscles of the gastrointestinal tract, as well as degeneration of peripheral nerves. Some of the symptoms of this disease may include vomiting, nausea, diarrhea, abdominal pain, and numbness or tingling in the hands and feet.

There is no effective treatment for MNGIE, and most patients pass away by ages between late 30's to early 40's. Because the genetic cause of MNGIE is known, this disease is a good candidate for gene therapy.

Chameleon’s EVADER technology could improve gene delivery efficiencies and allow for repeated treatments of gene therapy.

Hemophilia A is a rare, X chromosome–linked coagulation disorder caused by deficiencies in factor VIII (FVIII), an essential blood clotting protein. People affected by Hemophilia A may experience spontaneous or internal bleeding, which can be life-threatening.

Current gene therapy strategies targeting hemophilia consist of recurring dosing and may trigger strong, long term immunological resistance, making the current gene therapies unsuitable for treating a lifelong disease.

By potentially lowering immune responses, Chameleon’s EVADER technology is intended to both allow repeat dosing and enable the treatment of children and patients with pre-existing immunity.