Patent classifications
C07K14/81
VECTORS ENCODING A GLUCOSE-6-PHOSPHATASE (G6PASE-A) FOR GENE THERAPY
The invention relates to an adeno-associated virus (AAV) vector comprising a nucleic acid construct for the expression of a glucose-6-phosphatase-a (G6Pase-a) in a cell, the construct comprising a nucleic acid sequence encoding the G6Pase-a, wherein the nucleic acid sequence encoding the G6Pase-a is operably linked to a human alpha-1 antitrypsin (hAAT) promoter, a cell transformed with the vector of the invention, a composition comprising the vector or the cell of the invention, and the use thereof.
NOVEL COMPOUNDS FOR THE TREATMENT OF CANCER
The technology provided herein relates to novel isolated polypeptides and peptides having a growth inhibitory effect on human cancer cells. Nucleic acid molecules encoding said polypeptides/peptides, vectors, host cells containing the nucleic acids and methods for preparation and producing said polypeptides/peptides. Compositions and methods for using such polypeptides/peptides for the prevention and treatment of cancer are also encompassed by the present disclosure.
METHODS FOR TREATING PULMONARY DISEASE USING INTER-ALPHA INHIBITOR PROTEINS
The invention features methods for treating or preventing pulmonary diseases, including acute respiratory distress syndrome (ARDS) and pneumonia, in a subject in need thereof that involve administering to the subject inter-alpha inhibitor proteins (IαIps), including, e.g., inter-alpha inhibitor (IαI) and/or pre-alpha inhibitor (PαI).
THERAPEUTIC VARIANT ALPHA-2-MACROGLOBULIN COMPOSTIONS
A2M polypeptide compositions containing a non-natural bait region are disclosed. Methods of producing wild-type and variant A2M polypeptides and polynucleotides containing a non-natural bait region are also disclosed. The bait regions of the variant A2M polypeptides demonstrate enhanced protease inhibitory characteristics compared to wild-type A2M. Variant A2M polypeptides that demonstrate longer half-lives upon administration to an organism compared to wild-type A2M are disclosed. The A2M compositions are useful in treating a number of diseases and conditions including inflammation, chronic wounds, and diseases with a pathology associated with proteases.
PEPTIDE LIBRARY AND USE THEREOF
Disclosed are compositions and method related to variants of SPINK2 that bind to targets other than an endogenous target of SPINK2. In one embodiment, a peptide is provided that comprises the amino acid sequence SEQ ID NO: 1. In further embodiments, an amino acid sequences encoded by nucleotide positions 4 to 42 and/or nucleotide positions 94 to 189 in the nucleotide sequence of SEQ ID NO: 14 flank the amino terminus and the carboxyl terminus, respectively, of the amino acid sequence. In another embodiment, a peptide is provided that comprises an amino acid sequence derived from the amino acid sequence of SEQ ID NO: 1 in which a conservative substitution, deletion, addition and/or insertion of 1 to 5 (inclusive) amino acids has occurred at amino acids other than the 1st Xaa to the 12th Xaa counting from the amino terminus.
O-Glycan Sialylated Recombinant Glycoproteins and Cell Lines for Producing the Same
The present invention relates to cell lines that are genetically modified to overexpress a β-galactoside α-2,3-sialyltransferase 1 (ST3Gal1), preferably human ST3Gal1, which can be used for the production of recombinant glycoproteins having highly or fully sialylated O-linked GalNAc glycans (GalNAc O-glycans), preferably core 1 GalNAc O-glycans, as well as to respective recombinant glycoproteins. Further, the present invention relates to respective methods of expressing recombinant glycoproteins, methods of increasing the degree of sialylation of recombinant glycoproteins, and methods of decreasing the micro-heterogeneity of GalNAc O-glycans. Finally, the present invention relates to respective uses of the above cell lines for the production of recombinant glycoproteins, for increasing the degree of sialylation of recombinant glycoproteins, and for decreasing the micro-heterogeneity of O-linked GalNAc glycans of recombinant glycoproteins.
Coiled coil immunoglobulin fusion proteins and compositions thereof
Disclosed herein are immunoglobulin fusion proteins comprising a first antibody region, a first therapeutic agent, and a first connecting peptide; wherein the first therapeutic agent is attached to the first antibody region by the connecting peptide; and wherein the connecting peptide does not comprise a region having beta strand secondary structure. The connecting peptide may comprise an extender peptide. The extender peptide may have an alpha helical secondary structure. The connecting peptide may comprise a linker peptide. The linker peptide may not comprise any secondary structure. Also disclosed herein are compositions comprising the immunoglobulin fusion proteins and methods for using the immunoglobulin fusion proteins for the treatment or prevention of a disease or condition in a subject.
CYSTINE KNOT SCAFFOLD PLATFORM
Provided are non-naturally occurring cystine knot peptides (CKPs) that bind to VEGF-A. Additionally, provided are methods of using non-naturally occurring CKPs that bind to VEGF-A, including diagnostic and therapeutic compositions and methods. Non-naturally CKPs that bind low density lipoprotein receptor-related protein 6 (LRP6) are also provided.
Compositions and methods for the diagnosis of rheumatoid arthritis
The present disclosure relates to the field of molecular biology and more specifically to methods for detecting anti-carbamylated protein (anti-CarP) antibodies in the serum of rheumatoid arthritis (RA) patients.
LIPID NANOPARTICLE COMPOSITIONS AND METHODS FOR MRNA DELIVERY
Disclosed herein are compositions and methods for modulatimi the production of a protein in a target cell, The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.