A61P25/14

PYRROLIDINE DERIVATIVES AS OXYTOCIN/VASOPRESSIN V1A RECEPTORS ANTAGONISTS
20230097933 · 2023-03-30 ·

The present invention relates to a compound of formula (3Z,5S)-5-(hydroxymethyl)-1-[(2′-methyl-1,1′-biphenyl-4-yl)carbonyl]pyrrolidin-3-one O-meth19243yloxime, and/or an active metabolite thereof having antagonist action at the oxytocin receptor and/or vasopressin V1a receptor, to processes for their preparation, pharmaceutical compositions containing them and their use.

Inhibitors of histone deacetylase

The present invention relates to compounds of formula (I): ##STR00001##
or a pharmaceutically acceptable salt, hydrate, solvate, or prodrug thereof, wherein U, J, V, X, R.sup.2a, R.sup.2b, R.sup.2c, R.sup.5 and t are as described herein. The present invention relates generally to inhibitors of histone deacetylase and to methods of making and using them. These compounds are useful for promoting cognitive function and enhancing learning and memory formation. In addition, these compounds are useful for treating, alleviating, and/or preventing various conditions, including for example, neurological disorders, memory and cognitive function disorders/impairments, extinction learning disorders, fungal diseases and infections, inflammatory diseases, hematological diseases, and neoplastic diseases in humans and animals.

Solid forms of an sGC stimulator

The present disclosure relates to crystalline solid forms of a stimulator of soluble guanylate cyclase (sGC), Compound I: ##STR00001##
Also provided herein are methods for the preparation of these solid forms. The invention also relates to pharmaceutical formulations and dosage forms comprising these solid forms and their uses thereof, alone or in combination with one or more additional agents, for treating and/or preventing various diseases or disorders; these diseases or disorders are ones that may benefit from sGC stimulation or from an increase in the concentration of nitric oxide (NO) and/or cyclic guanosine monophosphate (cGMP).

METHODS AND PRODUCTS FOR TRANSFECTING CELLS
20230033888 · 2023-02-02 ·

The present invention relates in part to nucleic acids encoding proteins, nucleic acids containing non-canonical nucleotides, therapeutics comprising nucleic acids, methods, kits, and devices for inducing cells to express proteins, methods, kits, and devices for transfecting, gene editing, and reprogramming cells, and cells, organisms, and therapeutics produced using these methods, kits, and devices. Methods for inducing cells to express proteins and for reprogramming and gene-editing cells using RNA are disclosed. Methods for producing cells from patient samples, cells produced using these methods, and therapeutics comprising cells produced using these methods are also disclosed.

COMPOSITIONS AND METHODS FOR TREATING LYSOSOMAL DISORDERS
20230037062 · 2023-02-02 · ·

One aspect of the invention provides a method for treatment of a lysosomal storage disorder. The method may include administering to a subject in need of such treatment a composition including a therapeutically effective amount of an agent that mediates upregulation of Transcription Factor EB. In one embodiment, the composition includes a fibrate, such as gemfibrozil or fenofibrate. In another embodiment, the composition also includes all-trans retinoic acid or vitamin A.

Substituted imidazo[1,5-a]pyrazines for modulation of IRE1

Described herein, inter alia, are certain substituted imidazo[1,5-a]pyrazines of formula (I) and methods of using the same for modulating the activity of Ire1. ##STR00001##

COMPOSITION OF LACTIC ACID BACTERIUM FOR USE IN PREVENTING OR TREATING RETT SYNDROME
20230035928 · 2023-02-02 ·

The present disclosure provides a method of preventing or treating Rett syndrome in a subject in need thereof, including administering to the subject an effective amount of Lactobacillus plantarum subsp. plantarum PS128. Also provided is a composition comprising Lactobacillus plantarum subsp. plantarum PS128 and a carrier thereof for use in preventing or treating Rett syndrome in a subject in need thereof.

COMPOSITION OF LACTIC ACID BACTERIUM FOR USE IN PREVENTING OR TREATING RETT SYNDROME
20230035928 · 2023-02-02 ·

The present disclosure provides a method of preventing or treating Rett syndrome in a subject in need thereof, including administering to the subject an effective amount of Lactobacillus plantarum subsp. plantarum PS128. Also provided is a composition comprising Lactobacillus plantarum subsp. plantarum PS128 and a carrier thereof for use in preventing or treating Rett syndrome in a subject in need thereof.

Glycosidase inhibitors

Compounds of formula (I), wherein A, R, W, Q, n and m have the meaning according to the claims, can be employed, inter alia, for the treatment of tauopathies and Alzheimer's disease. ##STR00001##

Method for introducing hepatocyte growth factor and neurogenin 1 into mesenchymal stem cells

The present invention relates to an adult stem cell line introduced with an HGF gene and a neurogenic transcription factor gene of a bHLH family, a preparation method of the adult stem cell line, a composition for the prevention or treatment of neurological diseases comprising the adult stem cell line, and a method for treating neurological diseases comprising the step of administering the composition to a subject having neurological diseases or suspected of having neurological diseases. The adult stem cells according to the present invention, which are introduced with an HGF gene and a neurogenic transcription factor gene of a bHLH family, can be used to overcome chronic impairment caused by cell death following stroke. Thus, the adult stem cells can be developed as a novel therapeutic agent or widely used in clinical trial and research for cell replacement therapy and gene therapy that are applicable to neurological diseases including Parkinson's disease, Alzheimer disease, and spinal cord injury as well as stroke.