Resumen de: EP4748440A2
The present invention provides compounds of formula (I) that are prodrugs of catecholamine for use in treatment of neurodegenerative diseases and disorders. The present invention also provides pharmaceutical compositions comprising compounds of the invention and methods of treating neurodegenerative or neuropsychiatric diseases and disorders using the compounds of the invention, in particular Parkinson's disease.Accordingly, the present invention relates to compounds of formula (Id)whereinR1 is H and R2 is selected from one of the substituents (i) and (ii) below; orR1 is selected from one of the substituents (i) and (ii) below and R2 is H; orR1 and R2 are both represented by substituent (i) below; orR1 and R2 are both represented by substituent (ii) below; orR1 is substituent (i) and R2 is substituent (ii); orR1 is substituent (ii) and R2 is substituent (i);wherein * indicates the attachment point; andwherein the carbon atom at the attachment point on substituent (i) is in the S-configuration; or a pharmaceutically acceptable salt thereof.
Resumen de: EP4748443A2
The invention pertains to a solid composition suitable for treating adult animals that suffer from brain-related disorders, brain-related decline or brain-related diseases, preferably suitable for treating adult animals that suffer from brain-related disorders, the composition containing (i) a uridine source, wherein the amount of the sum of all uridine sources, calculated on the basis of the weight of the molar equivalent of uridine monophosphate (UMP), is 0.3-15 g per 100 g dry weight of the composition, (ii) a polyunsaturated fatty acid, preferably an omega-3 polyunsaturated fatty acid, (iii) choline bitartrate, (iv) a buffering salt component, and (v) a milk protein source, wherein the milk protein source is a milk powder, and the buffering salt component containing at least one food approved salt, with a buffering capacity between pH 5 - 8, the buffering salt component preferably containing a mixture of food-approved salts, or a food-approved salt and its conjugated acid or base.
Resumen de: AU2026203338A1
Abstract This disclosure provides methods for treating or preventing amyotrophic lateral sclerosis (ALS), methods for delaying the onset of neurological symptoms associated with ALS, increasing survival in subjects afflicted with ALS, and attenuating the decline of muscle strength associated with ALS in a subject in need thereof. This disclosure also provides methods for treating or preventing α-synucleinopathy or TDP-43 proteinopathy. The methods comprise administering to the subject an effective amount of a mitochondria- targeting peptidomimetic compound, such as (R)-2-amino-N-((S)-1-(((S)-5-amino-1-(3-benzyl-1,2,4-oxadiazol- 5-y l)pentyl)amino )-3-(4-hydroxy-2,6-dimethylphenyl)-1-oxopropan-2-yl)-5-guanidinopentanamide or a pharmaceutically acceptable salt, stereoisomer, tautomer, hydrate, and/or solvate thereof. Abstract
Resumen de: AU2024364256A1
This disclosure relates to vectors, compositions, pharmaceutical compositions, and kits that provide for brain cell-specific expression of reprogramming genes such as the Yamanaka factors Oct4, Sox2, Klf4 and c-Myc (OSKM). Also provided are methods and uses comprising the same for treating Alzheimer' s disease and progeria through brain cell-specific expression of reprogramming genes such as OSKM.
Resumen de: US20260137640A1
0000 A compound of formula (I) or a pharmaceutically acceptable salt thereof is disclosed for use as a medicament. Further, the compound of formula (I) for use in the therapy of amyotrophic lateral sclerosis. Still further, a method of treating amyotrophic lateral sclerosis comprising administering to a subject in need thereof an effective amount of the compound of formula (I) or a pharmaceutically acceptable salt thereof is disclosed.
Resumen de: AU2024372247A1
The present invention relates to the field of medicine, and specifically relates to a crystal form A and crystal form B of 20(S)-protopanaxadiol, a preparation method therefor and the use thereof. The crystal form A and crystal form B provided in the present invention are different from the existing known crystal forms of 20(S)-protopanaxadiol, each have a distinct profile, can be perfectly reproduced, have good bulk density, fluidity and stability, and good characteristics in terms of fighting depression, anxiety and cognitive disorder, preventing and/or treating Alzheimer's disease (AD), amyotrophic lateral sclerosis (ALS), and treating diseases such as tumors or cardiovascular diseases, and have broad application prospects in the drug development of 20(S)-protopanaxadiol.
Resumen de: US20260137646A1
0000 Administration of pentadecanoylcarnitine or pentadecanoic acid is provided for prevention, management or treatment of aggression, allergies, allergic rhinitis, Alzheimer's disease, anxiety and anxiety disorders, amyotrophic lateral sclerosis, arthritis, asthma, atherosclerosis, attention-deficit hyperactivity disorder, bipoloar disorder, brain damage, cancer, cardiovascular disease, cholestatic pruritis, depression, chronic obstructive pulmonary disease (COPD), cocaine abuse, cough, dermatitis, depression, drug-seeking behavior, gastrointestinal disorders, facial erythema associated with rosacea, glaucoma, hepatic diseases, hyperactive bladder, hypersensitivity disorders, hypertension, impulsivity, inflammation, mental disorders and conditions, metabolic disorders, migraines, nasal and sinus congestion, nausea, neuropathic pain with and symptoms of multiple sclerosis, neurological and neuropsychiatric disorders, obesity, obsessive-compulsive disorders, opioid-induced respiratory depression, osteoarthritis, pain, Parkinson disease, pathological gambling, peptic ulcers, schizophrenia, sleep disorders, spinal cord injury, tardive dyskinesia, tics and behavioral problems with Tourette's syndrome; as well as for supporting appetite, cardiovascular health, hematologic health, memory, metabolic health, mood, prolonged REM sleep, renal health, sexuality, sociability and metabolic, hematological, renal, and weight loss.
Resumen de: US20260139017A1
Described herein are methods and compositions for treating Alzheimer's Disease (AD), as well as compositions comprising a reelin-derived peptide and methods of use thereof.
Resumen de: WO2026099248A1
The present invention relates to compounds of formula (I) as TMEM175 modulators for reducing alpha-synuclein aggregation for the treatment of Parkinson's disease.
Resumen de: WO2026099233A1
The present invention relates to compounds of formula (l) as TMEM175 modulateurs for reducing alapha-synuclein aggregation for the treatment of Parkinson's didease
Resumen de: WO2026101897A1
Described herein is a small molecule splicing modulator compound that modulates splicing of mRNA, such as pre-mRNA, encoded by genes, and methods of use of the small molecule splicing modulator compounds for modulating splicing and treating diseases and conditions.
Resumen de: WO2026098667A1
The present invention provides a compound having USP30 inhibitor activity, a use thereof, a preparation method therefor, and a composition containing the inhibitor. These inhibitors are useful in a variety of therapeutic fields, including diseases or conditions involving mitochondrial dysfunction, such as PD, cancer, and fibrosis.
Resumen de: US20260132401A1
Provided are antisense agents, pharmaceutical compositions, and methods of use for reducing the amount or activity of alpha-synuclein (SNCA) RNA in a cell or subject, and in certain instances reducing the amount of alpha-synuclein protein in a cell or subject. In certain embodiments, also provided herein are oligomeric compounds, oligomeric duplexes, and RNAi agents for reducing the amount or activity of SNCA RNA in a cell or subject, and in certain instances reducing the amount of SNCA protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such neurodegenerative diseases include synucleinopathies, e.g., Parkinson's disease, dementia with Lewy bodies, diffuse Lewy body disease, Parkinson's disease dementia, pure autonomic failure, multiple system atrophy, neuronopathic Gaucher's disease, and Alzheimer's disease.
Resumen de: US20260132123A1
The present invention concerns Amyloid Binding Alcohol Dehydrogenase (ABAD) inhibitors (I) or (II) that interact non-competitively with nicotinamide adenine dinucleotide+H (NADH). Inhibition of ABAD is useful in the treatment or prophylaxis of disease, including Alzheimer's disease (AD) and cancer. Accordingly, the present invention also concerns ABAD inhibitors for use as a medicament, specifically for use in the treatment of Alzheimer's disease and/or cancer.
Resumen de: AU2024367335A1
The present disclosure provides human Tau protein (Tau)-binding proteins comprising an antigen binding domain of an antibody, wherein the antigen binding domain binds specifically to an epitope of Tau that results in inhibition of Tau aggregate seeding, and could be used to inhibit spreading of Tau aggregates in vivo. Also disclosed herein are nucleic acids, expression vectors, and recombinant viruses encoding such Tau-binding proteins, as well as compositions comprising such Tau-binding proteins. Also disclosed are methods for use of such compositions in the diagnosis, prophylaxis, treatment, and prognosis of Tauopathies such as Alzheimer's Disease, frontotemporal dementia, Pick's disease, and progressive supranuclear palsy. Genetically modified cells for expression for the disclosed Tau-binding proteins, and their use for production of the Tau-binding proteins are also disclosed.
Resumen de: US20260130879A1
The present invention relates to methods of treating or ameliorating a neurodegenerative disease, such as Parkinson's disease or multiple sclerosis, with a compound according to Formula (I) to (V), or with a compound according to Formula (VI), or a pharmaceutically acceptable salt or solvate thereof, for example, vidofludimus, which acts as a Nurr1 agonist.
Resumen de: US20260132176A1
0000 Methods and compositions for treating or preventing a neurodegenerative disease, such as Alzheimer's Disease, in a subject by administering to a subject a therapeutic amount of a pharmaceutical composition comprising a recombinant SUMO2 analogue.
Resumen de: US20260132190A1
0000 The present disclosure relates to IL-34 antibodies, compositions comprising the same, and methods of using the antibodies and or compositions thereof for treating immune-mediated diseases such as neurodegenerative diseases, for example Alzheimer's Disease or a tauopathy disease.
Resumen de: US20260130880A1
0000 Provided is a method of digital therapeutics for modulating one or more Parkinson's disease-related markers, symptoms, or disease progression in an individual, on a standalone basis or by combining them with medicinal agents, particularly dopaminergic agents. Also provided are corresponding systems for implementing, delivering, and adapting such therapeutic interventions.
Resumen de: US20260130947A1
0000 Disclosed herein are methods of treating ALS in a subject by administering a therapeutically effective amount of a composition comprising EVs, e.g., derived from neural cells, e.g., neural progenitor cells, to the subject. The EVs can be administered distally or peripherally to the CNS, such that the EVs cross the blood brain barrier and exert their therapeutic function in the CNS. The methods can reduce inflammation (e.g., NLRP3 inflammatory pathway signaling), reduce disease activity or progression, and/or improve motor or neurological performance, signs or symptoms associated with ALS, or survival in the ALS subject as compared to a control ALS subject. The methods of inhibiting necroptosis in a cell by contacting the cell with a therapeutically effective amount of a composition comprising EVs are also provided.
Resumen de: US20260130894A1
0000 The present invention relates to a pharmaceutical composition for the prevention or treatment of Parkinson's disease. Leading to the present invention, an isoquinoline derivative, identified through screening based on mitophagy activation, was found to exhibit an excellent effect in promoting mitophagy and proved to be a fundamental therapeutic agent for Parkinson's disease. Specifically, the isoquinoline derivative according to the present invention not only reduces mitochondrial dysfunction closely associated with Parkinson's disease in a Parkinson' disease animal model but also ameliorates motor function impairment in the Parkinson's disease animal model. Particularly, in the Parkinson's disease animal model treated with the isoquinoline derivative according to the present invention, the demise of dopaminergic neurons, a primary cause of Parkinson's disease, was effectively inhibited. Thus, the isoquinoline derivative according to the present invention is expected to be advantageously utilized as a fundamental therapeutic agent in the field of prevention, alleviation, and/or treatment of Parkinson's disease.
Resumen de: US20260131140A1
0000 The present disclosure relates to the treatment of patients with early-stage Parkinson's Disease using subthalamic nucleus deep brain stimulation (STN-DBS) to a target a defined region of the brain. In particular, by positioning and/or programming the DBS electrode to stimulate cortical input fibers from the supplementary motor area (but not the pre-SMA), improved therapeutic benefits are obtained.
Resumen de: US20260132177A1
0000 Changes in Reelin levels as well as Reelin signaling alter cognitive function. This can be accomplished by administering a therapeutically effective amount of a repeat fragment of Reelin, or a construct formed from fragment repeats of Reelin to a patient or subject. Changes to Reelin levels can be used to treat various neurodegenerative diseases, neuronal insults, or stroke, such as fragile X syndrome, William's syndrome, Rett syndrome, Down's syndrome, Angelman syndrome, autism, ischemia, hypoxia, Alzheimer's disease, and schizophrenia: Reelin can also be used to alter dendritic spine density, diminished long-term potentiation, and diminished synaptic plasticity and associative learning deficits. Constructs formed from repeat region 3 of full length Reelin and repeat region 5 of full length Reeling or repeat region 3 of full length Reelin and repeat region 6 of full length Reelin have been found particularly useful.
Resumen de: EP4741506A1
The present disclosure discloses a plurality of CpG-containing oligodeoxynucleotides (ODNs) with immunomodulatory properties, and use thereof. The oligodeoxynucleotides provided in present disclosure differ in sequence and structure from the design concept of conventional Type A CpG ODNs, reflecting their own complex drug-forming principles and overcoming the problems of conventional Type A CpG in drug development. The oligodeoxynucleotides exhibit excellent immunomodulatory activity both in vitro and in vivo, and they can stimulate the production of type I, II, and III interferons, upregulate Th1-type immune responses, and inhibit Th2-type and Th17-type inflammatory responses. Therefore, the oligodeoxynucleotides can: prevent and treat allergic diseases caused by Th2-type immune responses, including allergic rhinitis, atopic dermatitis, asthma, chronic obstructive pulmonary disease, eosinophilia, and Th17-related diseases; can prevent and treat infections and transmission of respiratory or non-respiratory pathogens, including viruses, bacteria, fungi, and parasites; can improve the immune levels of the elderly population and individuals with immunodeficiency; can transform "cold tumors" into "hot tumors" and improve the effectiveness of cancer immunotherapy; can prevent and treat central nervous system diseases related to immune dysfunction, such as Alzheimer's disease; and can be applied to other diseases related to immune dysregulation.
Nº publicación: EP4739676A1 13/05/2026
Solicitante:
SITALA BIO LTD [GB]
Sitala Bio Ltd
Resumen de: WO2025008453A1
The present invention relates to compounds (I) possessing an indole moiety linked to an isoindoline moiety, to compounds having similar core structures, and to associated salts, solvates, prodrugs and pharmaceutical compositions. The present invention further relates to methods of synthesising such compounds, and to the use of such compounds in the treatment and prevention of medical disorders and diseases, most especially by Factor B inhibition.