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COMPOSITIONS ET PROCEDES POUR LA SUPPRESSION DE LA FORMATION D'UN INHIBITEUR CONTRE LE FACTEUR VIII DE L'HEMOPHILIE A CHEZ DES PATIENTS PAR ADMINISTRATION D'ANTIGENES BIOENCAPSULES DANS DES CELLULES VEGETALES
专利权人:
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA;UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC.
发明人:
HERZOG, ROLAND W.,DANIELL, HENRY
申请号:
CA2967269
公开号:
CA2967269A1
申请日:
2014.11.17
申请国别(地区):
CA
年份:
2015
代理人:
摘要:
Protein replacement therapy for patients with hemophilia or other inherited protein deficiencies is often complicated by pathogenic antibody responses, including antibodies that neutralize the therapeutic protein or that predispose to potentially life -threatening anaphylactic reactions by formation of lgE. Using murine hemophilia as a model, we have developed a prophylactic protocol against such responses that is non-invasive and does not include immune suppression or genetic manipulation of the patient's cells. Oral delivery of coagulation factor expressed in chloroplasts, bioencapsulated in plant cells, effectively blocked formation of inhibitory antibodies in protein replacement therapy. Inhibitor titers were mostly undetectable and up to 100-fold lower in treated subjects when compared to controls. Moreover, this treatment eliminated fatal anaphylactic reactions that occurred after 4 to 6 exposures to intravenous coagulation factor protein.
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