A61K38/52

AGENT FOR USE IN THE CASE OF FRUCTOSE INTOLERANCE
20170319668 · 2017-11-09 ·

There is provided a method for treating or reducing the effects of fructose intolerance and health problems associated with excessive fructose intake by administration of glucose isomerase. Other embodiments are also disclosed.

METHODS FOR INDUCING SELECTIVE APOPTOSIS
20230270831 · 2023-08-31 ·

Provided herein are methods for cell therapy by modifying transfused cells to express an inducible caspase 9 protein, so that the cells may be selectively killed if the patient experiences dangerous side effects. Provided also within relates in part to methods for preventing or treating Graft versus Host Disease by modifying T cells before administration to a patient, so that they may be selectively killed if GvHD develops in the patient.

METHODS FOR INDUCING SELECTIVE APOPTOSIS
20230270831 · 2023-08-31 ·

Provided herein are methods for cell therapy by modifying transfused cells to express an inducible caspase 9 protein, so that the cells may be selectively killed if the patient experiences dangerous side effects. Provided also within relates in part to methods for preventing or treating Graft versus Host Disease by modifying T cells before administration to a patient, so that they may be selectively killed if GvHD develops in the patient.

POLYNUCLEOTIDES ENCODING METHYLMALONYL-CoA MUTASE

The disclosure relates to polynucleotides comprising an open reading frame of linked nucleosides encoding human methylmalonyl-CoA mutase precursor, human methylmalonyl-CoA mutase (MCM) mature form, or functional fragments thereof. In some embodiments, the disclosure includes methods of treating methylmalonic acidemia in a subject in need thereof comprising administering an mRNA encoding an MCM polypeptide.

POLYNUCLEOTIDES ENCODING METHYLMALONYL-CoA MUTASE

The disclosure relates to polynucleotides comprising an open reading frame of linked nucleosides encoding human methylmalonyl-CoA mutase precursor, human methylmalonyl-CoA mutase (MCM) mature form, or functional fragments thereof. In some embodiments, the disclosure includes methods of treating methylmalonic acidemia in a subject in need thereof comprising administering an mRNA encoding an MCM polypeptide.

DYNAMIC COVALENTLY LINKED HYDROGELS AS STABILIZATION NETWORK PLATFORMS

The present invention provides dynamically covalent polymeric hydrogel systems for encapsulating and stabilizing bioactive therapeutic agents (e.g., proteins, cells, viruses, and vaccines) from environmental stressors, obviating standard refrigeration requirements, and decreasing transportation and storage costs of temperature-sensitive biomolecules. Described herein are dynamic polymeric hydrogel compositions comprising a therapeutic agent and a combination of phenylboronic acid- and 1,2-diol-modified multi-arm polyethylene glycol (PEG) polymer backbones. Methods of encapsulating and stabilizing bioactive therapeutic agents within the dynamic polymeric hydrogel compositions are also provided. Also described are methods for releasing stabilized therapeutic agents from hydrogel encapsulation. The covalently adaptable hydrogel release systems allow for discretionary administration of temperature-sensitive therapeutic agents, as well as the parenteral administration of highly concentrated amounts of therapeutic agents.

DYNAMIC COVALENTLY LINKED HYDROGELS AS STABILIZATION NETWORK PLATFORMS

The present invention provides dynamically covalent polymeric hydrogel systems for encapsulating and stabilizing bioactive therapeutic agents (e.g., proteins, cells, viruses, and vaccines) from environmental stressors, obviating standard refrigeration requirements, and decreasing transportation and storage costs of temperature-sensitive biomolecules. Described herein are dynamic polymeric hydrogel compositions comprising a therapeutic agent and a combination of phenylboronic acid- and 1,2-diol-modified multi-arm polyethylene glycol (PEG) polymer backbones. Methods of encapsulating and stabilizing bioactive therapeutic agents within the dynamic polymeric hydrogel compositions are also provided. Also described are methods for releasing stabilized therapeutic agents from hydrogel encapsulation. The covalently adaptable hydrogel release systems allow for discretionary administration of temperature-sensitive therapeutic agents, as well as the parenteral administration of highly concentrated amounts of therapeutic agents.

Modified caspase-9 polypeptides and methods of use thereof

Provided herein are modified caspase-9 polypeptides, and chimeric caspase-9 proteins containing the modified caspase-9 polypeptides. The disclosure further provides polynucleotides encoding these proteins, engineered host cells containing these polynucleotides and proteins, including host cells that co-express a chimeric antigen receptor, and methods of making and using the same.

Modified caspase-9 polypeptides and methods of use thereof

Provided herein are modified caspase-9 polypeptides, and chimeric caspase-9 proteins containing the modified caspase-9 polypeptides. The disclosure further provides polynucleotides encoding these proteins, engineered host cells containing these polynucleotides and proteins, including host cells that co-express a chimeric antigen receptor, and methods of making and using the same.

METHODS FOR CONTROLLED ELIMINATION OF THERAPEUTIC CELLS

The technology relates in part to methods for controlling elimination of therapeutic cells, for example, cells that express a chimeric antigen receptor. The technology further relates to a two-step method of controlling destruction of therapeutic cells in a patient following an adverse event. The two-step system may include a rapamycin or rapamycin analog-based level of control and a second, rimiducid, level of control. The technology also relates in part to methods for cell therapy using cells that express the inducible caspase polypeptide and the rapamycin-sensitive polypeptide, where the proportion of therapeutic cells eliminated by apoptosis is related to the choice and amount of the administered ligand.