Patent classifications
C12Y301/06
Antibody specific for CD22 and methods of use thereof
The present disclosure provides antibodies specific for an epitope present on CD22. The antibodies are useful in various treatment, diagnostic, and monitoring applications, which are also provided.
PRODUCTION OF CATALYTICALLY ACTIVE TYPE I SULFATASE
The present disclosure provides methods for producing activated type I sulfatases, or functional fragments thereof, using Formylglycine Generating Enzymes (FGEs). Also featured by the disclosure are recombinant fungal (e.g., Yarrowia lipolytica) cells expressing the FGE and, in some embodiments, type I sulfatases, or functional fragments thereof, and/or additional accessory enzymes. The disclosure also provides activated type I sulfatases or functional fragments thereof, made by the disclosed methods and therapeutic methods using the activated type I sulfatases or functional fragments thereof.
Cleavable lipids
Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent transfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.
Antibody specific for CD22 and methods of use thereof
The present disclosure provides antibodies specific for an epitope present on CD22. The antibodies are useful in various treatment, diagnostic, and monitoring applications, which are also provided.
Cleavable Lipids
Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent iransfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.
Aldehyde-tagged protein-based drug carriers and methods of use
The disclosure provides aldehyde-tagged protein carriers that can be covalently and site-specifically bound to drug to provide a drug-containing scaffold. The invention also encompasses methods of production of such drug-containing scaffolds and intermediates, as well as methods of use.
Method of co-expressing a sulfatase and a sulfatase modifying factor in a plant or plant cell
The subject invention concerns materials and methods for treating or preventing disease and conditions associated with various sulfatase enzymes that are defective or that are not properly expressed in a person or animal. In one embodiment, the disease is Sanfilippo A (MPS-IIIA) disease. The subject invention also concerns materials and methods for treating or preventing multiple sulfatase deficiency (MSD) in a person or animal. Compounds of the invention include a fusion protein comprising i) a mammalian sulfatase, or an enzymatically active fragment or variant thereof, and ii) a plant lectin or a binding subunit thereof. In a specific embodiment, the mammalian sulfatase is a human sulfatase, or an enzymatically active fragment or variant thereof. Polynucleotides encoding the fusion proteins are also contemplated for the subject invention. The subject invention also concerns materials and methods for producing proteins of the invention. The subject invention further provides a method of co-expressing sulfatase and sulfatase modifying factor in a plant or plant cell.
Lyophilized Pharmaceutical Compositions Comprising a Lipid Nanoparticle
Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent transfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.
T-Cell Modulatory Multimeric Polypeptide with Conjugation Sites and Methods of Use Thereof
The present disclosure provides T-cell modulatory multimeric polypeptides (T-Cell-MMPs) comprising an immunomodulatory polypeptide (MOD) that may be selected to exhibit reduced binding affinity to a cognate co-immunomodulatory polypeptide (Co-MOD) and a location for covalently attaching a molecule that can serve as an epitope, such as an epitope peptide. Once the epitope molecule is attached the resulting T-Cell-MMP-epitope conjugates are useful for modulating the activity of a T-cell by delivering immunomodulatory peptides, such as IL-2 or IL-2 variants that exhibit reduced binding affinity for IL-2R, to the T-cells in an epitope selective/specific manner, and accordingly, for modulating an immune response in an individual.
MATERIALS AND METHODS FOR TREATING DISORDERS ASSOCIATED WITH SULFATASE ENZYMES
The subject invention concerns materials and methods for treating or preventing disease and conditions associated with various sulfatase enzymes that are defective or that are not properly expressed in a person or animal. In one embodiment, the disease is Sanfilippo A (MPS-IIIA) disease. The subject invention also concerns materials and methods for treating or preventing multiple sulfatase deficiency (MSD) in a person or animal. Compounds of the invention include a fusion protein comprising i) a mammalian sulfatase, or an enzymatically active fragment or variant thereof, and ii) a plant lectin or a binding subunit thereof. In a specific embodiment, the mammalian sulfatase is a human sulfatase, or an enzymatically active fragment or variant thereof. Polynucleotides encoding the fusion proteins are also contemplated for the subject invention. The subject invention also concerns materials and methods for producing proteins of the invention.