Follistatin in treating duchenne muscular dystrophy
AssigneeShire Human Genetic Therapies, Inc.
InventorRochelle Mineau
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Abstract
The present invention provides, among other things, methods and compositions for treating muscular dystrophy, in particular, Duchenne muscular dystrophy (DMD). In some embodiments, a method according to the present invention includes administering to an individual who is suffering from or susceptible to DMD an effective amount of a recombinant follistatin protein such that at least one symptom or feature of DMD is reduced in intensity, severity, or frequency, or has delayed onset.
Claims17
A method of treating Duchenne muscular dystrophy (DMD) comprising administering to an individual who is suffering from or susceptible to DMD an effective amount of a recombinant follistatin protein comprising an amino acid sequence of SEQ ID NO:
- 1 or 2 fused to an Fc domain via a peptide linker comprising 10 or more amino acids such that at least one symptom or feature of DMD is reduced in intensity, severity, or frequency, or has delayed onset, and wherein the peptide linker comprises the amino acid sequence of SEQ ID NO:
- 5, 6, or 7.
A recombinant follistatin fusion protein comprising
- a follistatin polypeptide;
- an Fc domain;
- and
- a linker that associates the follistatin polypeptide with the Fc domain,
- wherein the follistatin polypeptide comprises an amino acid sequence of SEQ ID NO.:
- 1 or 2;
- wherein the linker comprises the amino acid sequence of SEQ ID NO:
- 5, 6, or 7;
- and further wherein the recombinant follistatin fusion protein is capable of binding to activin, myostatin and/or GDF-11 and has an in vivo half-life ranging from about 0.5-10 days.
A method of treating Duchenne muscular dystrophy (DMD) comprising administering to an individual who is suffering from or susceptible to DMD an effective amount of a recombinant follistatin protein comprising an amino acid sequence SEQ ID NOs:
- 8, 9, 10 or 11, such that at least one symptom or feature of DMD is reduced in intensity, severity, or frequency, or has delayed onset.
Dependencies are inferred from the claim text.