Biglycan mutants and related therapeutics and methods of use

Patent metadata

Field	Value
Publication number	US-9969785-B2
Application number	US-201113109558-A
Country	US
Kind code	B2
Filing date	May 17, 2011
Priority date	May 17, 2010
Publication date	May 15, 2018
Grant date	May 15, 2018

Abstract

The present disclosure provides compositions and methods for treating diseases or conditions associated with an abnormal level or activity of biglycan; disorders associated with an unstable cytoplasmic membrane, for example, due to an unstable dystrophin associated protein complex (DAPC); disorders associated with abnormal synapses or neuromuscular junctions, including those resulting from an abnormal MuSK activation or acetylcholine receptor (AChR) aggregation. Examples of diseases include muscular dystrophies, such as Duchenne's Muscular Dystrophy, Becker's Muscular Dystrophy, neuromuscular disorders and neurological disorders.

First claim

We claim:

1. A method for treating a muscular dystrophy in a subject, comprising administering to the subject an effective amount of a biglycan-related polypeptide comprising an amino acid sequence corresponding to amino acids 38-365 of SEQ ID NO: 11, which biglycan-related polypeptide comprises less than 1% total carbohydrate by weight.

2. The method of claim 1 , wherein the muscular dystrophy is selected from Duchenne's Muscular Dystrophy, Becker's Muscular Dystrophy, Congenital Muscular Dystrophy, Limb-girdle Muscular Dystrophy, and myotonic dystrophy.

3. A method for treating amyotrophic lateral sclerosis in a subject, comprising administering to the subject an effective amount of a biglycan-related polypeptide comprising an amino acid sequence corresponding to amino acids 38-365 of SEQ ID NO: 11, which biglycan-related polypeptide comprises less than 1% total carbohydrate by weight.

4. A method for treating Bethlem's myopathy, Ullrich Congenital Muscular Dystrophy, or Sorsby's fundus dystrophy, comprising administering to the subject an effective amount of a biglycan-related polypeptide comprising an amino acid sequence corresponding to amino acids 38-365 of SEQ ID NO: 11, which biglycan-related polypeptide comprises less than 1% total carbohydrate by weight.

Assignees

• Fallon Justin R
• Amenta Alison R
• Mckechnie Beth A
• Dechene Michelle
• Yilmaz Atilgan
• Univ Brown

Inventors

• Fallon Justin R
• Amenta Alison R
• Mckechnie Beth A
• Dechene Michelle
• Yilmaz Atilgan

Classifications

• A61P43/00

  Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00 · CPC title

• A61P25/02

  for peripheral neuropathies · CPC title

• A61K38/00

  Medicinal preparations containing peptides (peptides containing beta-lactam rings A61K31/00; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, A61K31/00; ergot alkaloids of the cyclic peptide type A61K31/48; containing macromolecular compounds having statistically distributed amino acid units A61K31/74; medicinal preparations containing antigens or antibodies A61K39/00; medicinal preparations characterised by the non-active ingredients, e.g. peptides as drug carriers, A61K47/00) · CPC title

• C07K14/4725 Primary

  Proteoglycans, e.g. aggreccan · CPC title

• A61P25/00

  Drugs for disorders of the nervous system · CPC title