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INNOVATIVE SCIENCE

Delivering Protein Therapeutics to the CNS for Gaucher Disease

ERAD Therapeutics, Inc. is currently developing our proprietary technology for delivery of our therapeutic protein to every cell in the body, as well as across the blood brain barrier (BBB)and into neuronal cells.

1/ Proteins that harbor a single amino acid mutation, though slightly mis-folded, still retain activity. Normally these mis-folded proteins are destroyed by the endoplasmic reticulum (endoplasmic reticulum-associated degradation-ERAD). Our modified Cholera toxin (mCT) used in this approach has the ability to reversibly interfere with the ERAD process. This allows the mis-folded, but functional protein, to transit to its site of action.

Modified Cholera toxin is composed of an A and B subunit. The B-subunit is the delivery vehicle which can be modified to carry a variety of other proteins to the CNS.

2/ Protein Replacement Therapy: To deliver therapeutic proteins across the Blood-Brain-Barrier. Our current focus is on Enzyme Replacement Therapy for Gaucher Disease where we utilize our proprietary CTB Delivery System to transport glucocerebrosidase to patients with Gaucher Disease. ERAD Therapeutics Inc. delivery will be a significant improvement to existing ERT markets by delivering replacement enzymes across the BBB, currently inaccessible by commercially available ERT formulations. We are investigating the delivery of other types of therapeutic proteins via our CTB Delivery System to treat other Orphan Diseases.