Their dedicated professionals working together to avoid wasting the lives of individuals with genetic diseases.
Chameleon Bioscience was founded in 2017 in California. They need a longtime laboratory and leadership team additionally as their scientific, clinical, and regulatory consultants. Their private laboratory is inside Bonneville Labs, a collaborative space for emerging biotechnology companies, community partners, and bioscience market leaders. Chameleon Biosciences incorporates a talented team of dedicated professionals working together to avoid wasting the lives of individuals with genetic diseases.
Every year 8 million babies are born with serious genetic disorders, accounting for 20-30% of all infant deaths. Treatment options for survivors are often limited. Chameleon technology can address multiple limitations of current gene therapy. Most notably, repeated doses further expand treatment options for adults and youngsters.
Their exclusive EVADER gene delivery platform technology. Innovations developed by Chameleon founders have enabled them to style a potentially strenuous, less immunogenic gene therapy that overcomes existing treatment barriers. Their EVADER platform technology will be customized for a spread of payloads, for example, gene replacement or gene editing. Repeat dosing potentially enables them to focus on growing organs or rapidly dividing tissues.
Their Current Focus:
- Severe Hemophilia
Hemophilia B could be a bleeding disorder, caused by defects within the gene that encodes for plasma protein IX (FIX). FIX is very important for clotting and folks with hemophilia suffer from frequent and spontaneous bleeding episodes.
Gene therapy strategies targeting hemophilia are inadequate as they lead to strong immune responses, making them unsuitable for treating a lifelong disease. Chameleon’s EVADER technology is meant to lower immune responses, potentially both allowing repeat dosing and enabling the treatment of kids and patients with pre-existing immunity.
- Niemann-Pick Type C
Niemann-Pick Type C (NPC) could be 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, yet as various internal organs. Milder kinds of the disease are diagnosed in adults, but many children suffer from more severe sorts of NPC.
NPC is commonly fatal and while some treatments are being investigated, none of those treatments attack the genetic basis of the disease, and there’s no cure. Chameleon’s EVADER technology offers hope to those affected with NPC by potentially with over one dose of gene therapy.
- Mitochondrial NeuroGastroIntestinal Encephalomyopathy
Mitochondrial NeuroGastroIntestinal Encephalomyopathy (MNGIE) may be a genetic defect that causes a severe, multisystem disorder because of a defective thymidine phosphorylase enzyme. MNGIE is characterized by degeneration of the muscles of the duct, additionally with degeneration of peripheral nerves. Several symptoms of this disease may include vomiting, nausea, diarrhea, abdominal pain, and numbness or tingling within the hands and feet.
There is no effective treatment for MNGIE, and most patients expire by the ages of the late 30s to early 40s. Because the genetic explanation for MNGIE is understood, this disease may be a good candidate for gene therapy. Chameleon’s EVADER technology could improve gene delivery efficiencies and permit repeated treatments of gene therapy.
- Severe Hemophilia
Hemophilia A may be a rare, X chromosome-linked coagulation disorder caused by deficiencies in antihemophilic globulin (FVIII), a vital blood-clotting protein. People full of hemophilia may experience spontaneous or internal bleeding, which might be life-threatening.
Current gene therapy strategies targeting hemophilia comprise recurring dosing and should trigger strong, long-term immunological resistance, making the present gene therapies unsuitable for treating a lifelong disease. By potentially lowering immune responses, Chameleon’s EVADER technology is meant to both allow repeat dosing and enable the treatment of youngsters and patients with pre-existing immunity.
Their partnership with Mitopath provides a non-clinical package with standard AAV to quickly move toward testing EVADER gene therapy delivery with identified MNGIE patients. Their effective treatment can potentially run at lower doses, which may provide a lower product cost per systemic dose.
Chameleon Bioscience is pleased to partner with Mitopath for the advanced treatment of patients living with MNGIE. They’re hospitable to exploring other potential research collaborations or co-developments. Their platform technology is widely applied during a style of disorders. They anticipate that their treatment is going to be ready to effectively address these conditions over the subsequent five years.