A61K38/1825

Fibroblast growth factor 21 (FGF21) gene therapy for central nervous system disorders

Described herein is a gene construct comprising a nucleotide sequence encoding a fibroblast growth factor 21 (FGF21), for use in the treatment and/or prevention of a central nervous system (CNS) disorder or disease, or a condition associated therewith.

METHODS FOR TREATING AND PREVENTING CARDIOMYOPATHY

Methods for treating patients having a cardiomyopathy are provided. Additionally, methods for prophylactically treating patients at risk of developing a cardiomyopathy are provided. Methods for treating patients having, or at risk of developing, a cardiomyopathy may comprise administering a fusion protein including a tafazzin peptide and a cellular permeability peptide to the patient. Further, the tafazzin peptide may be coupled to the cellular permeability peptide by a polypeptide linker.

METHODS OF USING FIBROBLAST GROWTH FACTOR (FGF) 1 WITH MUTATION IN THE HEPARIN BINDING DOMAIN AND METHODS TO REDUCE BLOOD GLUCOSE

The present disclosure provides FGF1 mutant proteins having one or more mutations in the heparin binding domain. Such mutants may also have an N-terminal deletion, point mutation(s), or combinations thereof. In some examples, the mutant FGF1 proteins have reduced mitogenic activity. Also provided are nucleic acid molecules that encode such proteins, and vectors and cells that include such nucleic acids. The disclosed FGF1 mutants can reduce blood glucose in a mammal, and in some examples are used to treat a metabolic disorder.

METHODS OF USING FIBROBLAST GROWTH FACTOR (FGF) 1 WITH MUTATION IN THE HEPARIN BINDING DOMAIN AND METHODS TO REDUCE BLOOD GLUCOSE

The present disclosure provides FGF1 mutant proteins having one or more mutations in the heparin binding domain. Such mutants may also have an N-terminal deletion, point mutation(s), or combinations thereof. In some examples, the mutant FGF1 proteins have reduced mitogenic activity. Also provided are nucleic acid molecules that encode such proteins, and vectors and cells that include such nucleic acids. The disclosed FGF1 mutants can reduce blood glucose in a mammal, and in some examples are used to treat a metabolic disorder.

EXTRACELLULAR MATRIX SHEET STRUCTURES
20250228999 · 2025-07-17 · ·

An implantable medical product and method of use for substantially reducing or eliminating harsh biological responses associated with conventionally implanted medical devices, including inflammation, infection and thrombogenesis, when implanted in in a body of a warm blooded mammal. The bioremodelable pouch structure is configured and sized to receive, encase and retain an electrical medical device therein and to allow such device to be inserted into the internal region or cavity of the pouch structure; with the pouch structure formed from either: (a) first and second sheets, or (b) a single sheet having first and second sheet portions. After receiving the electrical device, the edges around the opening are closed by suturing or stapling. The medical device encased by the bioremodelable pouch structure effectively improves biological functions by promoting tissue regeneration, modulated healing of adjacent tissue or growth of new tissue when implanted in the body of the mammal.

Stabilized pharmaceutical formulations of insulin analogues and/or insulin derivatives

Stabilized pharmaceutical formulations of insulin analogs and/or insulin derivatives are disclosed.

Stabilized pharmaceutical formulations of insulin analogues and/or insulin derivatives

Stabilized pharmaceutical formulations of insulin analogs and/or insulin derivatives are disclosed.

FGF21 variant, fusion protein and application thereof

A fibroblast growth factor 21 (FGF21) variant, further to a FGF21 variant fusion protein, a protein multimer, and use thereof can significantly improve the binding ability with the target and can be used to treat metabolic diseases.

COMBINATION THERAPIES FOR INNER EAR SENSORY HAIR CELL REGENERATION/REPLACEMENT
20170348346 · 2017-12-07 · ·

The present disclosure relates to compositions and methods for the regeneration and/or restoration of hair cells utilizing a composition or an agent that decreases expression of a gene in a tissue of the inner ear and a second agent.

Adeno-associated virus virion for gene transfer to nervous system cells
11674156 · 2023-06-13 · ·

The present invention provides a means for transferring a therapeutic gene of interest into a nervous system cell by a highly-efficient and simpler means. More specifically, the present invention provides a recombinant vector that uses an adeno-associated virus (AAV), a method for manufacturing the recombinant vector, and a method for using the recombinant vector. More specifically, recombinant adeno-associated virus virions, which are capable of passing through the brain-brain barrier, for transferring a therapeutic genes of interest into a nervous system cell in a highly-efficient manner, a drug composition containing the recombinant adeno-associated virus virions, a method for manufacturing the recombinant adeno-associated virus virions, and a kit or the like are provided.