Abstract

The disclosure provides novel conjugates comprising antisense oligonucleotides that hybridize to a DMPK transcript and a 3E10 antibody or binding fragment thereof. Also considered are these conjugates further comprising MBNLl polypeptides. Methods of treating myotonic dystrophy using these conjugates and kits comprising these conjugates are also considered. Wherein the conjugates are suitable for delivery to muscle cells.

IPC Classes  ?

• A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
• A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
• A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system

Abstract

The disclosure provides radiolabeled compounds suitable for in vivo imaging or as cytotoxic agents. These radiolabeled compounds can be used to image or treat cancer.

IPC Classes  ?

• A61K 51/10 - Antibodies or immunoglobulinsFragments thereof
• A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
• G01N 33/534 - Production of labelled immunochemicals with radioactive label
• G01N 33/53 - ImmunoassayBiospecific binding assayMaterials therefor
• A61K 103/00 - Radioactive metals
• A61K 101/00 - Radioactive non-metals
• A61P 35/00 - Antineoplastic agents
• A61K 101/02 - Halogens
• A61K 103/10 - TechnetiumRhenium

Abstract

The present invention provides chimeric polypeptides comprising myotubularin 1 (MTM1) polypeptides and an internalising moiety, wherein, the moiety can be an antibody, and is preferably monoclonal antibody 3E10, a functional variant or a fragment thereof. One aspect of the present invention provides compositions comprising these chimeric polypeptides together with a pharmaceutically acceptable carrier, and optionally, a further therapeutic agent. Another aspect of the present invention provides methods of treating Myotubular Myopathy comprising administering the polypeptides or compositions comprising the polypeptides to a subject in need.

IPC Classes  ?

• C07K 19/00 - Hybrid peptides
• A61K 38/16 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof
• A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
• A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
• C07H 21/00 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
• C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals

Abstract

A chimeric polypeptide comprising: (i) an MBNL (muscleblind-like) polypeptide and (ii) a targeting moiety which targets muscle cells (such as an antibody or a protein that acts via an equilibrative nucleoside transporter (ENT2) pathway). Methods of treating myotonic dystrophy using the chimeric polypeptide.

IPC Classes  ?

• C07K 19/00 - Hybrid peptides
• A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
• A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
• C12N 15/12 - Genes encoding animal proteins
• C12N 15/13 - Immunoglobulins
• C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals