Alerta

NEGATIVE ALLOSTERIC MODULATORS OF METABOTROPIC GLUTAMATE RECEPTOR 2

Resumen de: US20260226019A1

0000 Described are 6-aryl isoindolin-1-ones as negative allosteric modulators of metabotropic glutamate receptor 2 (mGlu<2>), pharmaceutical compositions including the compounds, and methods of using the compounds and compositions for treating depression, anxiety, obsessive-compulsive disorder, cognitive disorders, Alzheimer's disease, or autism spectrum disorders in a subject.

THIOUREA ANTIOXCOMPOUNDS WITH NEUROPROTECTIVE ACTIVITY

Resumen de: US20260226036A1

Provided herein are compounds of the formula wherein the variables are as defined herein. Pharmaceutical compositions of the compounds are also provided. In some aspects, the compounds or compositions of the present disclosure may be used for the treatment of diseases or disorders, such as an addiction or neurodegenerative disease including Alzheimer's disease or Parkinson's disease.

PHARMACEUTICAL FORMULATIONS OF GENE DELIVERY VEHICLES

Resumen de: US20260224740A1

The invention relates to formulations comprising miRNA that have improved stability for the treatment of diseases including neurodegenerative diseases such as Huntington's disease.

INHIBITORS OF CYCLIC ADP RIBOSE HYDROLASE AND METHODS OF USE THEREOF

Resumen de: US20260224561A1

0000 The disclosure provides compounds, in part, compounds of Formula I or Formula II and their use in treating medical diseases or disorders, such as neurodegenerative diseases, e.g., Parkinson's disease. Pharmaceutical compositions and methods of making compounds of the disclosure are provided. The compounds are contemplated to be modulators, e.g., inhibitors, of cyclic ADP ribose hydrolase (CD38).

ARYL HETEROCYCLIC KV1.3 INHIBITOR, PREPARATION METHOD THEREFOR, AND USE THEREOF

Resumen de: US20260224536A1

A novel Kv1.3 channel (or KV1.3) inhibitor, which can be used for preventing and/or treating diseases related to the Kv1.3 channel (or Kv1.3), including immune and inflammatory diseases, such as: multiple sclerosis, inflammatory bowel disease, ulcerative colitis, Crohn's disease, rheumatoid arthritis, type I diabetes, psoriasis and asthma, spondylitis, and periodontitis; as well as obesity, type 2 diabetes, renal fibrosis, Alzheimer's disease, ischemic stroke, etc.

FULLERENE GABA DOPA AND METHODS

Resumen de: US20260224713A1

0000 A novel dual neurotransmitter nanoparticle composition is provided to store and transport protons and cations into neural cell membranes and to disassemble salt-bridge stabilized toxic protein plaques. These properties function to mitigate cognitive deficits in neurological diseases such as Parkinson's disease and Alzheimer's disease, as well as to reduce the severity of Inflammatory Bowel Syndrome, and aging related reactive oxygen species damage by promoting the sequestration and termination of free radicals and reactive oxygen species. The composition comprises C60 bonded to one or more gamma amino butyric acid molecules and one or more molecules of either levodopa or dopamine. The composition can be produced at low temperatures through reactive shear milling. This composition therapeutically improves and prophylactically preserves cognitive performance, memory, and mental acuity on aging to promote mental performance and health-span improvement.

ALS treatment using induced regulatory T (IT) cells

Resumen de: AU2026207969A1

ALS TREATMENT USING INDUCED REGULATORY T (iTREG) CELLS The present disclosure provides methods for treating ALS using pentostatin and cyclophosphamide treatment followed by TREG and/or TREG/Th2 hybrid cells from dedifferentiated T cells. The present disclosure further provides methods for producing TREG and TREG/Th2 hybrid cells from de-differentiated T cells, said TREG and TREG/Th2 hybrid cells, populations thereof and compositions thereof. Methods for producing de-differentiated T cells, said de-differentiated T cells, populations thereof and compositions thereof are also provided. ul u l

COMPOUND AS GLUTAMINE CYCLASE INHIBITOR

Resumen de: EP4786464A1

Disclosed are a compound represented by formula (A-I) or a stereoisomer thereof or a pharmaceutically acceptable salt thereof as a glutamine cyclase inhibitor, a preparation method therefor, a pharmaceutical composition comprising the compound represented by formula (A-I) or the stereoisomer thereof or the pharmaceutically acceptable salt thereof, and a use of the compound represented by formula (A-I) or the stereoisomer thereof or the pharmaceutically acceptable salt thereof in preventing or treating diseases or conditions mediated by QPCT and/or QPCTL, including neurodegenerative diseases.

TRIAZOLOPYRIDINYL COMPOUNDS AS KINASE INHIBITORS

Resumen de: AU2024353744A1

Compounds having formula (I), and enantiomers, and diastereomers, stereoisomers, pharmaceutically acceptable salts thereof, (I) are useful as kinase modulators, including RIPK1 modulation. All the variables are as defined herein.

TRIAZOLOPYRIDINYL ETHER LINKED COMPOUNDS AS KINASE INHIBITORS

Resumen de: TW202521108A

Compounds having formula (I), and enantiomers, and diastereomers, stereoisomers, pharmaceutically-acceptable salts thereof, are useful as kinase modulators, including RIPK1 modulation. All the variables are as defined herein.

PIPERIDINE SUBSTITUTED PYRAZOLOPYRIMIDINE DERIVATIVES AS INHIBITORS OF SGK1

Resumen de: WO2025072438A1

The present invention provides compounds of Formula (I): or stereoisomers, tautomers, or pharmaceutically acceptable salts thereof, wherein all the variables are as defined herein. These compounds are selective SGK1 inhibitors. This invention also relates to pharmaceutical compositions comprising these compounds and methods of treating disorders associated with serum- and glucocorticoid-regulated kinase 1 (SGK1) activity, such as cardiovascular disorders, fibrotic diseases, metabolic diseases, immune and inflammatory diseases, neurological disorders, and cancer, by using the compounds and pharmaceutical compositions.

PYRIDINE DERIVATIVES WITH N-LINKED CYCLIC SUBSTITUENTS AS CGAS INHIBITORS

Resumen de: EP4786463A2

0001 The invention relates to new proline derivatives of formula (I) as cGAS inhibitors, wherein wherein R1, R2, R3, R4, R5, R6, R7, R8, R9, R10, R11 and G are defined as in claim 1, and prodrugs or pharmaceutically acceptable salts of these compounds for the treatment of diseases such as systemic lupus erythematosus, systemic sclerosis (SSc), non-alcoholic steatohepatitis (NASH), interstitial lung disease (ILD) and idiopathic pulmonary fibrosis (IPF).

Method

Resumen de: GB2703561A

The present invention relates to a method of introducing a vector comprising a nucleotide sequence encoding a Kv7.3 ion channel subunit to a lower urinary tract (LUT) neuron and expressing or overexpressing the Kv7.3 ion channel subunit, such that it forms functional ion channels. The method can additionally include a step of administering a neuromodulatory drug. Also disclosed is a gene therapy vector, comprising a nucleotide sequence encoding a Kv7.3 ion channel subunit, wherein the vector selectively transduces the target LUT neuron. Further disclosed is an AAV viral particle gene therapy vector comprising an AAV capsid and a nucleotide sequence encoding a Kv7.3 ion channel subunit. The method can be used to treat a neurological disorder, selected from; spasticity, Parkinson's disease, multiple sclerosis, stroke, traumatic brain injury, spinal cord injury, motor neuron disease, spina bifida, transverse myelitis, HTLV-1 associated neurological conditions, and cerebral palsy. The neuromodulatory drug is selected from; Retigabine/Ezogabine and derivatives thereof, BHV-7000 and Xen496, Xen 1101, flupirtine, diclofenac, BMS-204352, meclofenamic acid, ETX-123 and linopirdine. A use of a neuromodulatory drug in the treatment of bladder musculature dysfunction (BMD), such as detrusor sphincter dyssynergia (DSD) or OAB-NC is also disclosed. Fig. 1

Fused heterocyclic compounds and uses thereof

Resumen de: GB2703562A

A phenoxazine, chromenothiazole, xanthene or thioxanthone compound of formula I or a pharmaceutically acceptable salt, hydrate or solvate thereof for use in a method of treatment or prophylaxis of a tauopathy or a disease of tau protein aggregation: wherein: R1 is -H, C1-4alkyl, halogenated C1-4alkyl, ORO1, C(=O)ORO2 or C(=O)NRN1RN2; R2 is H, halo, C1-4alkyl, NO2, OH or OC1-4alkyl; or R1 and R2, together with the atoms to which they are bound, form a fused phenylene group; R3 is H, halo, -ORO4, or -NRN3RN4; RN3 and RN4 are H, C1-4alkyl or -C(=O)R’, where R’ is C1-4alkyl; R4 is selected from H, halo, C1-4alkyl, halogenated C1-4alkyl, and -ORO5; RO1, RO2, RO4, RO5, RN1 and RN2 are H or C1-4alkyl; X is O; Y is N, N-oxide, or C-R5; and ring ‘A’ is a fused phenylene of formula A1 or a fused aminothiazole of formula A2; or X is S; Y is C=O; and ring A is a fused phenylene of formula A3: wherein W is O or N+ substituted with H or C1-4alkyl; and the remaining groups are as defined herein. The compounds may be useful in the treatment of Alzheimer’s disease. Compounds prepared include N-ethyl-N-2-(piperidin-1-yl)-6H-chromeno2,3-dthiazol-6-ylideneethanaminium perchlorate. See generic formula I at page 5

PRODUCTS AND METHODS FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS

Resumen de: US2021108209A1

0001 The present invention relates to RNA-based methods for inhibiting the expression of the superoxide dismutase 1 (SOD-1) gene. Recombinant adeno-associated viruses of the invention deliver DNAs encoding RNAs that knock down the expression of SOD-1. The methods have application in the treatment of amyotrophic lateral sclerosis.

Improved brain architecture and biomarkers in alzheimer's disease with mesenchymal stem cells

Resumen de: WO2025137077A1

Compositions and methods are disclosed herein for the treatment of Alzheimer's disease with allogeneic mesenchymal stem cells. The methods of treatment involve the administration of a composition of allogeneic mesenchymal stem cells to a subject in need thereof, wherein the efficacy of the treatment methods can be determined through the measurement of specific biomarkers and improved cognitive function and/or quality of life.

PATHOGENIC FACTOR OF NEURODEGENERATIVE DISEASE AND USE THEREOF

Resumen de: WO2026158729A2

Provided in the present application are a pathogenic factor of neurodegenerative diseases and the use thereof. The pathogenic factor of neurodegenerative diseases is a PSAP-GPR37-IL-6 signaling axis. The present application specifies for the first time the action mechanism of the PSAP-GPR37-IL-6 signaling axis as a pathogenic factor of neurodegenerative diseases (especially Parkinson's disease), and reveals that oligodendrocytes regulate the molecular pathway of neuroinflammation and neurodegenerative changes via the signaling axis, thus filling the research gap in the prior art regarding the participation of oligodendrocytes in the pathogenesis of PD.

TRADITIONAL CHINESE MEDICINE FOR PREVENTING AND/OR TREATING PARKINSON'S DISEASE AND METHOD FOR PREPARING SAME

Resumen de: WO2026158331A1

Provided are a traditional Chinese medicine for preventing and/or treating Parkinson's disease and a method for preparing same. The traditional Chinese medicine comprises the following raw materials in parts by weight: 10-15 parts of Gastrodiae Rhizoma, 5-10 parts of Juglandis Semen, 30-40 parts of honey, 30 parts of Rosa roxburghii, and 5-8 parts of lemon. The traditional Chinese medicine has significant effects on the prevention and/or treatment of Parkinson's disease, and features simple composition, mild adverse effects, and good drug stability.

CORRECTION OF ALZHEIMER'S DISEASE PATHOLOGY

Resumen de: US20260216351A1

Disclosed are compositions and methods of use for treating neuronal diseases such as Alzheimer's Disease, Parkinson's Disease, Huntington's Disease, multiple sclerosis, and amyotrophic lateral sclerosis (ALS). In certain embodiments, a flavonoid, such as apigenin, is administered alone or in a pharmaceutical preparation via the nasal olfactory route. In some embodiments, the composition further comprises a porosome complex for reconstitution into neural cells. In additional embodiments, a synthetic blood-brain barrier traversing peptide is included, wherein the peptide is engineered to mimic a domain of ATP1A3 and/or Tubulin, thereby enhancing therapeutic efficacy in restoring secretory and metabolic function in neural tissue.

HISTONE ACETYLATION BLOOD BIOMARKER FOR ALZHEIMER'S DISEASE

Resumen de: WO2026161742A1

Methods and materials for identifying mammals (e.g., humans) as having, or being likely to have or to develop, Alzheimer's disease are provided herein. In some cases, methods and materials for stratifying mammals with Alzheimer's disease are provided herein.

Treatment Methods for ALS Patients

Resumen de: US20260216237A1

0000 Provided herein are methods and compositions for the treatment of ALS using chlorite or a pharmaceutical composition comprising sodium chlorite, for a target ALS patient population. Also provided herein are methods for identifying a target ALS patient population likely to be responsive to treatment with chlorite or a pharmaceutical composition comprising sodium chlorite, the methods including identifying ALS patients with elevated plasma C-reactive protein (CRP) levels and/or patients age 40-65, sporadic ALS pathology, or combinations thereof.

METHOD FOR TREATING NEUROLOGICAL DISEASES THROUGH GLIAL PRUNING REGULATION

Resumen de: US20260216139A1

Methods for regulating aberrant glia-directed engulfment and active pruning of Neuronal Receptive Endings (NRE) are provided. Such methods comprise contacting a neuronal and/or glia cell with an effective amount of a composition which is effective in modulating at least one gene and/or protein associated with glia-directed engulfment and active pruning of Neuronal Receptive Endings. Such methods are useful in treating neurological disorders such as Parkinson's disease.

METHODS FOR DIAGNOSING ALS

Resumen de: WO2026161463A1

Disclosed is a method of diagnosing a subject with ALS, the method including measuring the amount of OxPL on apolipoprotein E, E2, E3 and/or E4.

A DUPLEX ELECTROCHEMILUMINESCENCE IMMUNOASSAY FOR TOTAL A-SYNUCLEIN AND PS129-A-SYNUCLEIN

Resumen de: US20260219283A1

0000 Synucleinopathies are a group of neurodegenerative diseases including Parkinson's disease (PD), dementia with Lewy bodies (DLB), and multiple system atrophy (MSA). These diseases are characterized by the aggregation and deposition of α-synuclein (α-syn) in Lewy bodies (LBs) in PD and DLB or as glial cytoplasmic inclusions in MSA. In healthy brains, only ~4% of α-syn is phosphorylated at Ser129 (pS129-α-syn), whereas >90% pS129-α-syn may be found in LBs. Embodiments of the invention include a duplex assay for both total α-synuclein and pS129-α-synuclein, which allows measuring both analytes in the same sample, leading to substantial saving in sample volume. The assays can be widely used in methods for detecting pS129-α-syn in biomedical studies including when only a limited volume of sample is available and high sensitivity is required, offering new opportunities for diagnostic biomarkers, monitoring disease progression, and quantifying outcome measures in clinical trials.

COMPOSITIONS AND METHODS FOR THE TREATMENT OF NEUROLOGICAL DISORDERS RELATED TO GLUCOSYLCERAMIDASE BETA 1 DEFICIENCY

Nº publicación: US20260216372A1 30/07/2026

Solicitante:

VOYAGER THERAPEUTICS INC [US]
VOYAGER THERAPEUTICS, INC.

Resumen de: US20260216372A1

The disclosure relates to compositions and methods for altering, e.g., enhancing, the expression of GCase proteins, whether in vitro and/or in vivo. Such compositions include delivery of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Parkinson's Disease (PD), Gaucher Disease (GD), Dementia with Lewy Bodies (DLB), or related condition resulting from a deficiency in the quantity and/or function of GBA1 gene product or associated with decreased expression or protein levels of GCase protein.