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US 11,590,204 B2Granted
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.
Claims33
A formulation comprising:
- (a) a C-type natriuretic peptide (CNP) variant 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;
- and
- (b) citric acid monohydrate, sodium citrate dihydrate, trehalose dihydrate, D-mannitol, L-methionine and polysorbate 80, wherein the formulation comprises less than 2.0 mg/ml of said CNP peptide variant.
29 more elements
A formulation comprising:
- (a) a C-type natriuretic peptide (CNP) variant 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;
- and
- (b) citric acid monohydrate, sodium citrate dihydrate, trehalose dihydrate, D-mannitol, L-methionine and polysorbate 80, wherein the formulation comprises about 0.5 to about 2.0 mg/ml of said CNP peptide variant.
29 more elements
A formulation comprising:
- (a) a C-type natriuretic peptide (CNP) variant 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;
- and
- (b) citric acid monohydrate, sodium citrate dihydrate, trehalose dihydrate, D-mannitol, L-methionine and polysorbate 80, wherein the formulation comprises 10.0 mg/ml±9.9 mg/ml of said CNP peptide variant.
29 more elements
Dependencies are inferred from the claim text.