Our Science

EVADER™ Platform Technology

a new approach to gene therapy that builds on current technology

EVADER™ immune suppressive particles are co-administered with any AAV gene therapy to:

Reduce antibodies and activated T cells

Confer resistance to NAbs

Increase AAV potency

Enable repeat dosing

Potentially Effective for More Patients

Hope for Adults with Prior Exposure to AAV

Today, up to 50% of all adults can’t be treated due to prior exposure to the gene therapy virus. Our therapy shows resistance to neutralizing antibodies so that EVADER products can potentially treat these patients.

Addresses the One‑Dose Limit

Most gene therapies lose effectiveness over time—as the cells with modified DNA become diluted or die—but immune response to the first dose prevents re-dosing. With EVADER, re-dosing is possible.

Expected to be Safer

Our therapy should be easily tolerated and potentially given at lower doses to increase safety.

Offering Hope for Children

Children aren’t currently treated for many diseases because the treatment will not be durable. With safe and effective EVADER therapies, additional doses are possible.

Fighting More Types of Diseases

Our EVADER platform technology can be customized for a variety of payloads, for example, gene replacement or gene editing. Repeat dosing potentially enables us to target growing organs or rapidly dividing tissues.

Our current focus is:

Severe Hemophilia B (Factor IX Deficiency)

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 ("Childhood "Alzheimers")

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.

Severe Hemophilia A (Factor VIII Deficiency)

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.

More Cost Effective to Develop and Deliver

The platform technology can be mobilized to target many types of genetic disorders.

Any standard capsid could potentially benefit from the addition of EVADER™ technology.

Initial nonclinical and in vivo results indicate at least a ten-fold increase in potency.

Our potent treatment can potentially be given at lower doses, which could provide lower production cost per systemic dose.

Scalable cGMP manufacturing technology is utilized.

Compelling Competitive Advantages

Sustainable Lead with Protected Intellectual Property

EVADER™ technology is based on intellectual property owned by Chameleon Biosciences and is further protected by issued IP of Chameleon co-founder Casey Maguire at Massachusetts General Hospital covering enveloped AAV.

Publications

Our work is supported by peer-reviewed research.

Scalable Purification Method for Enveloped AAV

by Dr. Casey Maguire, Chameleon Biosciences co-founder, and Collaborators:

Neutralizing antibody evasion and transduction with purified extracellular vesicle-enveloped AAV vectors (2021)

Engineered exosomes reduce immune activation

by M Poggio et al:

Suppression of Exosomal PD-L1 Induces Systemic Anti-tumor Immunity and Memory (2019)

Rescue of hearing using Ex-AAV1 (enveloped AAV1)

shown by Dr. Casey Maguire lab:

Rescue of Hearing by Gene Delivery to Inner-Ear Hair Cells Using Exosome-Associated AAV (2017)

Enveloped AAV with AAV6 & AAV9 for gene delivery to the brain

researched in the lab of Nathalie Cartier by Nicola Salvatore Orefice et al:

Real-Time Monitoring of Exosome Enveloped-AAV Spreading by Endomicroscopy Approach: A New Tool for Gene Delivery in the Brain (2019)

Advantages of Enveloped AAV over standard AAV

discovered by Chameleon Biosciences co-founder Dr. Casey Maguire and collaborators at Harvard University:

Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors (2017)

Partnerships

Partner Opportunities

We are open to exploring other potential research collaborations or co-development.

Learn About Our Team