Use of C-type natriuretic peptide variants to treat skeletal dysplasia
AssigneeBiomarin Pharmaceutical Inc.
InventorsSherry Bullens, Stuart Bunting, Tianwei Chou, Augustus O. Okhamafe, Christopher P. Price, Daniel J. Wendt, Clarence Yap
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Abstract
The present disclosure provides for use of variants of C-type natriuretic peptide (CNP), and novel pharmaceutical compositions and formulations comprising CNP variant peptides for the treatment of skeletal dysplasias, one or more symptoms of skeletal dysplasias, such as long bone growth or growth velocity, and other disorders having a skeletal dysplasia and/or CNP-associated symptom or component.
Claims13
A method of treating skeletal dysplasia in a subject comprising the step of administering to said subject a composition comprising a CNP variant peptide in an amount of at least 7.5 μg/kg of said CNP variant peptide, wherein the CNP variant peptide is selected from the group consisting of:
- [CNP-37(M32N);
- SEQ ID NO:
- 1]
- QEHPNARKYKGANKKGLSKGCFGLKLDRIGSNSGLGC;
- (Met-CNP-37;
- SEQ ID NO:
- 2)
- MQEHPNARKYKGANKKGLSKGCFGLKLDRIGSMSGLGC;
- (Pro-CNP-37;
- SEQ ID NO:
- 3)
- PQEHPNARKYKGANKKGLSKGCFGLKLDRIGSMSGLGC;
- [Gly-CNP 37(M32N);
- SEQ ID NO:
- 4]
- GQEHPNARKYKGANKKGLSKGCFGLKLDRIGSNSGLGC;
- (Pro-Gly-CNP-37;
- SEQ ID NO:
- 5)
- PGQEHPNARKYKGANKKGLSKGCFGLKLDRIGSMSGLGC;
- (Met-Gly-CNP-37;
- SEQ ID NO:
- 6)
- MGQEHPNARKYKGANKKGLSKGCFGLKLDRIGSMSGLGC;
- and
- (Gly-CNP-37:
- SEQ ID NO:
- 7)
- GQEHPNARKYKGANKKGLSKGCFGLKLDRIGSMSGLGC,
- wherein said composition comprises a formulation comprising citric acid monohydrate, sodium citrate dihydrate, trehalose dihydrate, D-mannitol, L-methionine and polysorbate 80, and wherein the step of administering treats said skeletal dysplasia.
Dependencies are inferred from the claim text.