Patent classifications
C07K14/76
AAV-ZYME AND USE FOR INFUSION REPLACEMENT THERAPY
The invention described herein provides methods and compositions for using recombinant AAV-based viral vectors to express any gene-of-interest (GOI), preferably from muscle and/or liver tissues, for secretion of the polypeptides encoded by the GOI into the bloodstream, thus serving as an alternative treatment regimen for diseases caused by or characterized by lack of functional GOI, or for disease caused by or characterized by a pathological antigen that can be neutralized by a neutralizing peptide encoded by the GOI, such as an immunoglobulin, antibody, or antigen-binding fragment thereof. The method/composition of the invention can be used as an alternative for enzyme replacement therapy (ERT), or for antibody-based therapy.
REGULATORY T CELL EPITOPES, COMPOSITIONS AND USES THEREOF
The invention is directed to T cell epitopes wherein said epitopes comprises a peptide or polypeptide chain comprising at least a portion of an immunoglobulin constant or variable region. The invention also relates to methods of using and methods of making the epitopes of the invention.
REGULATORY T CELL EPITOPES, COMPOSITIONS AND USES THEREOF
The invention is directed to T cell epitopes wherein said epitopes comprises a peptide or polypeptide chain comprising at least a portion of an immunoglobulin constant or variable region. The invention also relates to methods of using and methods of making the epitopes of the invention.
CONJUGATION OF LIPOPHILIC ALBUMIN-BINDING MOIETY TO RNA FOR IMPROVED CARRIER-FREE IN VIVO PHARMACOKINETICS AND GENE SILENCING
Provided herein are compounds and methods for gene silencing. The compound includes a RNA directly conjugated to an albumin-binding group. The method includes administering a compound comprising a RNA directly conjugated to an albumin-binding group to a subject in need thereof.
CONJUGATION OF LIPOPHILIC ALBUMIN-BINDING MOIETY TO RNA FOR IMPROVED CARRIER-FREE IN VIVO PHARMACOKINETICS AND GENE SILENCING
Provided herein are compounds and methods for gene silencing. The compound includes a RNA directly conjugated to an albumin-binding group. The method includes administering a compound comprising a RNA directly conjugated to an albumin-binding group to a subject in need thereof.
COMPOSITION AND METHOD FOR CRYOPRESERVATION OF CELLS
A cryopreservative composition includes a sugar component with a total concentration of sugar components in the composition of 300 mM or less; a sugar alcohol component, with a total concentration of sugar alcohol components in the composition of 2 M or less; and at least one of a polymer component and albumin, with the proviso that the composition includes less than a cryopreservative amount of dimethyl sulfoxide (DMSO). A method of cryopreserving a cell includes adding a cell to the cryopreservative composition; freezing the composition; storing the frozen composition; thawing the composition; removing the cell from the thawed composition; and culturing the cell under conditions effective for the cell to remain viable. The freezing may include cooling at a rate of 0.1° C./min to 5° C./min. The method may be performed without a washing step after thawing.
COMPOSITION AND METHOD FOR CRYOPRESERVATION OF CELLS
A cryopreservative composition includes a sugar component with a total concentration of sugar components in the composition of 300 mM or less; a sugar alcohol component, with a total concentration of sugar alcohol components in the composition of 2 M or less; and at least one of a polymer component and albumin, with the proviso that the composition includes less than a cryopreservative amount of dimethyl sulfoxide (DMSO). A method of cryopreserving a cell includes adding a cell to the cryopreservative composition; freezing the composition; storing the frozen composition; thawing the composition; removing the cell from the thawed composition; and culturing the cell under conditions effective for the cell to remain viable. The freezing may include cooling at a rate of 0.1° C./min to 5° C./min. The method may be performed without a washing step after thawing.
RECOMBINANT MILK PROTEINS AND COMPOSITIONS COMPRISING THE SAME
Provided are recombinant milk proteins with non-native PTMs, food products comprising the recombinant milk proteins, and methods of obtaining the recombinant milk proteins and for manufacturing the food products.
RECOMBINANT MILK PROTEINS AND COMPOSITIONS COMPRISING THE SAME
Provided are recombinant milk proteins with non-native PTMs, food products comprising the recombinant milk proteins, and methods of obtaining the recombinant milk proteins and for manufacturing the food products.
ACTIVATABLE FUSION PROTEIN AND USE THEREOF
Disclosed herein is an activatable fusion protein that includes, in an N- to C-terminal direction, an albumin, a matrix metalloproteinase-cleavable linker, and an immunoadhesin which includes a cytotoxic T lymphocyte-associated antigen-4 (CTLA4) having an N-terminal extracellular domain and an IgG Fc region, wherein the albumin is released from the activatable fusion protein in the presence of a matrix metalloproteinase that cleaves the cleavable linker, so that the N-terminal extracellular domain of the CTLA4 binds to CD80 or CD86. Also disclosed herein is use of the activatable fusion protein for suppressing a T cell-dependent immune response.