A61K38/443

COMPOUND DECREASING THE CONCENTRATION OF 2-HYDROXY-GLUTARATE

The present invention relates to a compound decreasing the concentration of 2-hydroxy-glutarate (2HG) in a subject for use in treating, preventing, and/or preventing progression of cardiac remodeling, in particular cardiomyopathy and/or heart failure and to viral particles, compositions, uses and methods related thereto.

Nanoparticles for Controlled Release of Anti-Biofilm Agents and Methods of Use
20210330599 · 2021-10-28 ·

The present invention relates to compositions and methods to treat and/or prevent biofilms and biofilm related diseases. The invention comprises a nanoparticle carrier (NPC) and at least one therapeutic agent therein. The NPC binds within biofilm and to surfaces at risk for biofilm formation and accumulation while providing local, sustained, enhanced and controlled delivery of the therapeutic agent, when triggered for release. In one embodiment, the NPC comprises pH-responsive elements that allows for specific delivery of the therapeutic agent when the local environment dictates that the agent should be delivered precisely when it is most needed.

Use of Lentivector-Transduced T-RAPA Cells for Amelioration of Lysosomal Storage Disorders

The present disclosure provides methods of treating lysosomal storage disorders. The method comprises producing vector-transduced T-Rapa cells that express a transgene of interest and administering the cells to a patient in need thereof.

Therapeutic compositions including frataxin, lactoferrin, and mitochondrial energy generating enzymes, and uses thereof
11141456 · 2021-10-12 · ·

Disclosed herein are methods and compositions for the treatment and/or prevention of diseases or conditions comprising administration of a therapeutic biological molecule, and/or naturally or artificially occurring derivatives, analogues, or pharmaceutically acceptable salts thereof, alone or in combination with one or more active agents (e.g., an aromatic-cationic peptide). The present technology provides compositions related to aromatic-cationic peptides linked to a therapeutic biological molecule and uses of the same. In some embodiments, the aromatic-cationic peptide comprises 2′,6′-dimethyl-Tyr-D-Arg-Phe-Lys-NH.sub.2, Phe-D-Arg-Phe-Lys-NH.sub.2, or D-Arg-2′,6′-Dmt-Lys-Phe-NH.sub.2.

VIRAL VECTOR FOR TREATING AUTOIMMUNE DISEASE AND DIABETES AND CONSTRUCTION METHOD AND APPLICATION THEREOF
20210269824 · 2021-09-02 ·

Provided are a viral vector for treating autoimmune disease and diabetes and a construction method and an application thereof. The viral vector is a lentiviral expression plasmid or an adeno-associated viral expression plasmid cloned with mfat-1 gene, and the mfat-1 gene is as shown in SEQ ID NO: 1.

COMPOSITIONS AND METHODS FOR GENERATING PHYSIOLOGICAL X CHROMOSOME INACTIVATION

The present invention features compositions and methods for recapitulating physiological X-chromosome inactivation (XCI) in a cell, including a cell of any embryo generated by Somatic Cell Nuclear Transfer (SCNT). In one aspect, the invention features a method for generating physiological X chromosome inactivation in an embryo generated by SCNT, the method comprising injecting the embryo generated via SCNT with an H3K27me3-specific demethylase polypeptide or a polynucleotide encoding said demethylase. Disclosed herein are methods, compositions, and kits comprising an agent which increases the expression of genes encoding an H3K27me3-specific demethylase, or increases the activity of human H3K27me3-specific demethylase.

Methods and compositions for treating mucosal tissue disorders

The present invention provides compositions and formulations comprising glutathione with or without thiocyanate and methods of use thereof to treat diseases and disorders in mucosal/epithelial tissue.

Methods and compositions for treating mucosal tissue disorders

The present invention provides compositions and formulations comprising glutathione with or without thiocyanate and methods of use thereof to treat diseases and disorders in mucosal/epithelial tissue.

METAL-ORGANIC FRAMEWORK MEDIATED MACROMOLECULE DELIVERY SYSTEM

Articles and methods related to the administration of biomolecules to subjects using metal-organic frameworks are generally described. In some embodiments, an article is described, the article comprising a substrate comprising an adhesive layer, a support layer disposed on at least a portion of the substrate, and an active material disposed on and/or in at least a portion of the support layer, wherein the active material comprises a composite comprising a metal-organic framework and a biomolecule. In certain embodiments, a method of treating a subject with a biomolecule is described, the method comprising administering an article to a subject.

COMPOSITIONS AND METHODS FOR TREATING SOLID CANCER
20230398190 · 2023-12-14 ·

Compositions and methods for treating solid cancer are provided. Specifically, the disclosure provides compositions comprising haloperoxidases, and methods comprising administering such compositions, for treating solid cancer.