12-002 - Determination of immunogenic peptides in lysosomal enzymes and induction of oral tolerance

Researchers

Adriana Montano, Angela Sosa Molano, Shunji Tomatsu, Alan Knutsen, Barbara Kariuki and Clifford Bellone.

Summary

This invention comprises a novel peptide-based immunotherapy to induce specific tolerance to N-acetyl galactosamine-6-sulfate sulfatase (GALNS) used in enzyme replacement therapy to treat mucopolysaccharidosis type IVA (Morquio A syndrome). Three immunodominant peptides were identified that elicited strong immune responses to GALNS and were incorporated into an oral tolerance protocol to condition patients prior to initiating enzyme replacement therapy.  This invention also discloses a method of identifying immunodominant peptides for use in inducing oral tolerance with other enzyme replacement therapies.

Intellectual Property Status

• U.S. patent pending