Research Opportunities

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The Ashkenazi Jewish population is at high risk for Fabry disease, a genetically inherited lysosomal storage disorder. Human recombinant α-galactosidase A enzyme is currently used for enzyme replacement therapy for Fabry disease. Derivatives of the enzyme that target receptors will be constructed to render the enzyme more active. Others will be completely non-immunogenic. Full scale production manufacturing is needed.

Advantages / Features

• The enzyme replacement therapy is more active.
• More enzyme is efficently taken up by the cells.
• Enzyme does not induce an antibody response.
• Enzyme costs less to manufacture.
• Simple production scheme less prone to problems.

Applications / Benefits

• Fewer patient infusions needed.
• Lower enzyme amounts delivered.
• Lower costs for patients.
• Large scale prodcution of enzyme.
• Extendable to other lysosomal storage diseases.

Current Project Status
Clinical trials demonstrated that infusions of smaller doses of enzyme led to fewer side effects. Derivatives of the enzyme are under development that are approximately 10-fold more active on a per milligram basis. Thus, a 10-fold reduction in the milligram dose would lead to the same therapeutic effect with a corresponding reduction in adverse reactions to the infusion.

Future Efforts Needed to Commercialize
Support is requested to establish a replacement drug manufacturing demonstration facility that will enable clinical evaluation required for appropriate FDA licensing.

Patents
"Recombinant α-Galactosidase A: Therapy for Fabry Patients", US Patent Number 5,179,023 issued January 12, 1993.

"Recombinant α-Galactosidase A: Therapy for Fabry Patients", US Patent Number 5,658,567 issued August 19, 1997.

"Recombinant α-Galactosidase A: Therapy for Fabry Patients", US Patent Number 6,461,609 issued October 8, 2002.

"Recombinant α-Galactosidase A: Therapy for Fabry Patients", US Patent Number 7,011,831, issued March 14, 2006.