C1 inhibitor produced in the milk of transgenic mammals
AssigneePharming Intellectual Property B.V.
InventorsJohannes Nuijens, Henricus Van Veen, Frank Pieper, Joris Heus
Life of this patent
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Abstract
The invention provides transgenic nonhuman mammals expressing C1 inhibitor in their milk. The C1 inhibitor is useful in treating patients with hereditary angioedema or patients requiring immunosuppression.
Claims15
1-17. (canceled)
C1 inhibitor produced by
- recovering milk from a transgenic nonhuman mammal comprising:
- a DNA segment encoding a C1 inhibitor heterologous to the mammal operably linked to at least one regulatory sequence effective to promote expression of the DNA segment in mammary gland cells of the mammal and a segment encoding a signal peptide functional in mammary cells of the mammal:
- wherein the DNA segment encoding the C1 inhibitor can be expressed in the mammary gland cells of an adult form of the mammal to produce C1 inhibitor in the milk of the nonhuman mammal;
- and
- purifying the C1 inhibitor from the milk.
A method of treating a patient suffering from or susceptible to C1 inhibitor deficiency, comprising administering to the patient an effective dose of C1 inhibitor produced in accordance with claim 1 and a pharmaceutically acceptable composition.
A method of purifying human C1 inhibitor, comprising:
- loading a sample comprising human C1 inhibitor in milk from a nonhuman animal onto a cationic exchange column under conditions in which the human C1 inhibitor binds to the column;
- eluting the human C1 inhibitor from the cationic exchange column;
- loading the eluate on an anionic exchange column under conditions in which the human C1 inhibitor binds to the column;
- eluting the human C1 inhibitor from the anionic exchange column;
- loading the eluate onto a metal ion exchange column under conditions in which residual contaminating proteins bind to the column;
- collecting eluate containing the human C1 inhibitor from the metal ion exchange column.
A pharmaceutical composition for comprising human C1 inhibitor free of other human proteins.
A pharmaceutical composition comprising human C1 inhibitor that is at least 98% w/w pure.
31-32. (canceled)
Dependencies are inferred from the claim text.