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LastUpdate Updated on 20/08/2026 [07:53:00]
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Solicitudes publicadas en los últimos 60 días / Last 60 days publications
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PHARMACEUTICAL FORMULATIONS OF GENE DELIVERY VEHICLES

Publication No.:  US20260224740A1 06/08/2026
Applicant: 
UNIQURE BIOPHARMA B V [NL]
UNIQURE BIOPHARMA B.V.
US_20260224740_A1

Absstract of: 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.

ALS treatment using induced regulatory T (IT) cells

Publication No.:  AU2026207969A1 06/08/2026
Applicant: 
RAPA THERAPEUTICS LLC
Rapa Therapeutics, LLC
AU_2026207969_A1

Absstract of: 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

NEGATIVE ALLOSTERIC MODULATORS OF METABOTROPIC GLUTAMATE RECEPTOR 2

Publication No.:  US20260226019A1 06/08/2026
Applicant: 
VANDERBILT UNIV [US]
Vanderbilt University
US_20260226019_A1

Absstract of: 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.

ALPHA-SYNUCLEIN BINDERS AND METHODS OF USE

Publication No.:  US20260224758A1 06/08/2026
Applicant: 
MERCK SHARP & DOHME LLC [US]
Merck Sharp & Dohme LLC
US_20260224758_A1

Absstract of: US20260224758A1

The invention is directed to compounds of Formula I (I) or their pharmaceutically acceptable salts, which may be suitable for imaging alpha-synuclein pathology and hence are useful in binding and imaging alpha-synuclein aggregates in patients with Parkinson's Disease. More specifically, this invention relates to a method of using the compounds of this invention as tracers in positron emission tomography (PET) imaging to study alpha-synuclein in brain in vivo to allow diagnosis of Parkinson's Disease and other neurodegenerative diseases Specific characterized by alpha-synuclein pathology. The invention further relates to a method of measuring clinical efficacy of therapeutic agents for Parkinson's Disease and other neurodegenerative diseases characterized by alpha-synuclein pathology.

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

Publication No.:  US20260224536A1 06/08/2026
Applicant: 
SHANGHAI SHENSHI WISE TECH CO LTD [CN]
SHANGHAI SHENSHI WISE TECHNOLOGY CO., LTD.
US_20260224536_A1

Absstract of: 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.

OLIGONUCLEOTIDES FOR MODULATING APOLIPOPROTEIN E (APOE) EXPRESSION AND METHODS OF USE THEREOF

Publication No.:  US20260226463A1 06/08/2026
Applicant: 
SCINEURO THERAPEUTICS INC [US]
SciNeuro Therapeutics Inc.
US_20260226463_A1

Absstract of: US20260226463A1

0000 The present disclosure relates to oligonucleotides, in particular antisense oligonucleotides (ASOs) and pharmaceutically acceptable salts thereof, that can hybridize and reduce the expression of APOE pre-mRNA or mRNA. ASOs disclosed herein can reduce translation of APOE protein in mammals (e.g., humans). The present disclosure further relates to methods of treating a disease or disorder in a subject in need thereof by administration of an antisense oligonucleotide disclosed herein. In particular, methods and ASOs described herein can be used for preventing and/or treating human diseases in which the reduction of APOE amount or its activity would be beneficial, including but not limited to neurodegenerative diseases such as Alzheimer's disease (AD), dementia with Lewy bodies (DLB), Parkinson disease dementia (PDD), and those broadly defined as tauopathies and synucleinopathies.

ANTIBODIES FOR TREATING NEUROLOGICAL AND NEUROVASCULAR DISORDERS

Publication No.:  US20260226177A1 06/08/2026
Applicant: 
LYS THERAPEUTICS [FR]
LYS THERAPEUTICS
US_20260226177_A1

Absstract of: US20260226177A1

0000 The present disclosure provides humanized anti-GluN1 receptor antibodies and antigen-binding fragments or derivatives thereof, which are effective in inhibiting the deleterious effects of tissue-type plasminogen activator (t-PA) mediated by N-methyl-D-aspartate (NMDA) receptors, as well as pharmaceutical compositions and medical uses thereof, particularly for the treatment of neurological, neurovascular or neurodegenerative disorders, such as stroke, multiple sclerosis, Parkinson's disease, and others.

FULLERENE GABA DOPA AND METHODS

Publication No.:  US20260224713A1 06/08/2026
Applicant: 
CARBON 60 ZHEJIANG PHARMACEUTICAL TECH CO LTD [CN]
CARBON 60 (ZHEJIANG) PHARMACEUTICAL TECHNOLOGY CO., LTD.
US_20260224713_A1

Absstract of: 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.

Fused heterocyclic compounds and uses thereof

Publication No.:  GB2703562A 05/08/2026
Applicant: 
WISTA LABORATORIES LTD [SG]
WisTa Laboratories Ltd.

Absstract of: 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

Method

Publication No.:  GB2703561A 05/08/2026
Applicant: 
SANIA TX LTD [GB]
Sania TX Ltd

Absstract of: 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

PIPERIDINE SUBSTITUTED PYRAZOLOPYRIMIDINE DERIVATIVES AS INHIBITORS OF SGK1

Publication No.:  EP4784582A1 05/08/2026
Applicant: 
BRISTOL MYERS SQUIBB CO [US]
Bristol-Myers Squibb Company
WO_2025072438_PA

Absstract of: 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.

TRIAZOLOPYRIDINYL ETHER LINKED COMPOUNDS AS KINASE INHIBITORS

Publication No.:  EP4784577A1 05/08/2026
Applicant: 
BRISTOL MYERS SQUIBB CO [US]
Bristol-Myers Squibb Company
TW_202521108_PA

Absstract of: 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.

TRIAZOLOPYRIDINYL COMPOUNDS AS KINASE INHIBITORS

Publication No.:  EP4784245A1 05/08/2026
Applicant: 
BRISTOL MYERS SQUIBB CO [US]
Bristol-Myers Squibb Company
AU_2024353744_PA

Absstract of: 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.

PRODUCTS AND METHODS FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS

Publication No.:  CA3314724A1 05/08/2026
Applicant: 
RESEARCH INST AT NATIONWIDE CHILDRENS HOSPITAL [US]
LUDWIG INST FOR CANCER RESEARCH [CH]
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
LUDWIG INSTITUTE FOR CANCER RESEARCH
US_2021108209_A1

Absstract of: 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.

PYRIDINE DERIVATIVES WITH N-LINKED CYCLIC SUBSTITUENTS AS CGAS INHIBITORS

Publication No.:  EP4786463A2 05/08/2026
Applicant: 
BOEHRINGER INGELHEIM INT [DE]
Boehringer Ingelheim International GmbH
EP_4786463_PA

Absstract of: 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).

COMPOUND AS GLUTAMINE CYCLASE INHIBITOR

Publication No.:  EP4786464A1 05/08/2026
Applicant: 
SIMCERE PHARMACEUTICAL CO LTD [CN]
Simcere Pharmaceutical Co., Ltd.
EP_4786464_PA

Absstract of: 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.

PATHOGENIC FACTOR OF NEURODEGENERATIVE DISEASE AND USE THEREOF

Publication No.:  WO2026158729A2 30/07/2026
Applicant: 
THE FIRST AFFILIATED HOSPITAL OF ZHEJIANG UNIV SCHOOL OF MEDICINE THE FIRST HOSPITAL OF ZHEJIANG UNI [CN]
\u6D59\u6C5F\u5927\u5B66\u533B\u5B66\u9662\u9644\u5C5E\u7B2C\u4E00\u533B\u9662\uFF08\u6D59\u6C5F\u7701\u7B2C\u4E00\u533B\u9662\uFF09
WO_2026158729_A2

Absstract of: 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.

SYNAPTIC CONNECTOR, NUCLEIC ACID, DOPAMINE SYNAPSE FORMATION PROMOTER, PHARMACEUTICAL COMPOSITION, METHOD FOR PROMOTING FORMATION OF DOPAMINE SYNAPSE, METHOD FOR TREATING PARKINSON'S DISEASE, USE OF SYNAPTIC CONNECTOR, FUNCTIONAL VARIANT OF CBLN4, COMPLEX, AND METHOD FOR PRODUCING ARTIFICIAL SYNAPTIC CONNECTOR

Publication No.:  WO2026159574A1 30/07/2026
Applicant: 
KEIO UNIV [JP]
THE UNIV OF TOKYO [JP]
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\u56FD\u7ACB\u5927\u5B66\u6CD5\u4EBA\u6771\u4EAC\u5927\u5B66
WO_2026159574_A1

Absstract of: WO2026159574A1

A synaptic connector that promotes the formation of dopamine synapse, the synaptic connector being a molecule capable of connecting a deleted in colorectal cancer (DCC) protein present on the surface of the front part of dopamine synapse and a delta-type glutamate receptor (GluD) protein present on the surface of the rear part of dopamine synapse.

BERBERINE TAUROURSODEOXYCHOLATE COMPOSITIONS AND METHODS THEREOF

Publication No.:  US20260217699A1 30/07/2026
Applicant: 
SHENZHEN HIGHTIDE BIOPHARMACEUTICAL LTD [CN]
SHENZHEN HIGHTIDE BIOPHARMACEUTICAL LTD.
US_20260217699_A1

Absstract of: US20260217699A1

0000 The invention provides berberine tauroursodeoxycholate (BTUDC), pharmaceutical compositions and methods of use thereof for the treatment, reduction and/or prevention of Parkinson's disease, or an associated disease and disorder, as monotherapy or in combination with other agents or as an adjuvant. The invention further provides pharmaceutical compositions and methods of use of berberine (BBR) and tauroursodeoxycholic acid (TUDCA) for the treatment, reduction and/or prevention of Parkinson's disease, or an associated disease and disorder, as monotherapy or in combination with other agents or as an adjuvant.

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

Publication No.:  US20260216372A1 30/07/2026
Applicant: 
VOYAGER THERAPEUTICS INC [US]
VOYAGER THERAPEUTICS, INC.
US_20260216372_A1

Absstract of: 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.

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

Publication No.:  US20260219283A1 30/07/2026
Applicant: 
THE REGENTS OF THE UNIV OF CALIFORNIA [US]
The Regents of the University of California
US_20260219283_A1

Absstract of: 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.

INHIBITORS OF LRRK2

Publication No.:  US20260217678A1 30/07/2026
Applicant: 
VANDERBILT UNIV [US]
Vanderbilt University
US_20260217678_A1

Absstract of: US20260217678A1

Indazole- and azaindazole-substituted octahydrocyclopentacpyrrole and hexahydrocyclopentacpyrrole compounds are inhibitors of LRRK2. The compounds and their pharmaceutical compositions may be used in the treatment of CNS disorders such as familial/genetic and/or sporadic Parkinson's disease, a tauopathy, or Alzheimer's disease. Indazole- and azaindazole-substituted octahydrocyclopentacpyrrole and hexahydrocyclopentacpyrrole compounds of formula (I) are inhibitors of LRRK2. The compounds and their pharmaceutical compositions may be used in the treatment of CNS disorders such as familial/genetic and/or sporadic Parkinson's disease, a tauopathy, or Alzheimer's disease.

METHOD FOR TREATING NEUROLOGICAL DISEASES THROUGH GLIAL PRUNING REGULATION

Publication No.:  US20260216139A1 30/07/2026
Applicant: 
FRED HUTCHINSON CANCER CENTER [US]
Fred Hutchinson Cancer Center
US_20260216139_A1

Absstract of: 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.

Treatment Methods for ALS Patients

Publication No.:  US20260216237A1 30/07/2026
Applicant: 
NEUVIVO INC [US]
THE REGENTS OF THE UNIV OF CALIFORNIA [US]
Neuvivo, Inc.
The Regents of the University of California
US_20260216237_A1

Absstract of: 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.

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

Nº publicación: WO2026158331A1 30/07/2026

Applicant:

GUIZHOU JINQIANGUO BIOTECHNOLOGY CO LTD [CN]
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WO_2026158331_A1

Absstract of: 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.

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