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.
Resumen de: EP4782452A1
A compound represented by formula (I) and a use thereof in the preparation of a drug. The invention relates to a novel compound having an IRAK4 degradation effect, which can effectively degrade IRAK4 or otherwise inhibit IRAK4 activity. The invention has very good prospects for application in IRAK4-mediated diseases, including immune diseases, tumors, Alzheimer's disease, and fibrotic diseases, and provides a new choice for clinical screening and/or preparation of drugs related to IRAK4 activity.
Resumen de: WO2025061918A1
The present invention relates to therapeutical uses of non-classical human major histocompatibility complex (MHC) molecules (also named MHC class Ib molecules) in combination with peptide antigens for the treatment of Parkinson's disease. The invention more specifically relates to recombinant polypeptides comprising peptide antigens and one or more domains of a non-classical MHC class Ib molecule. The invention also relates to methods of producing such recombinant polypeptides, pharmaceutical compositions comprising the same, as well as their uses for treating Parkinson's disease.
Resumen de: EP4782003A1
The present application relates to a novel therapeutic agent for treating Parkinson's disease and a method for treating Parkinson's disease using the same.
Resumen de: EP4782449A1
0001 The present invention provides a use of an SARM1 enzyme activity inhibitor in treating neurodegenerative diseases or neurological diseases or conditions, and specifically provides a compound of formula (I) acting as an SARM1 enzyme activity inhibitor, and a pharmaceutical composition thereof.
Resumen de: TW202530251A
The present invention relates to therapeutical uses of non-classical human major histocompatibility complex (MHC) molecules (also named MHC class Ib molecules) in combination with peptide antigens for the treatment of neuromyelitis optica (NMO). The invention more specifically relates to recombinant polypeptides comprising peptide antigens and one or more domains of a non-classical MHC class Ib molecule. The invention also relates to methods of producing such recombinant polypeptides, pharmaceutical compositions comprising the same, as well as their uses for treating neuromyelitis optica (NMO).
Resumen de: US20260209350A1
0000 The invention is generally directed to methods and compositions that include antibodies, e.g., monoclonal, chimeric, humanized antibodies, antibody fragments, etc., that specifically bind a TREM2 protein, e.g., a mammalian TREM2 and/or human TREM2. The methods provided herein find use in preventing, reducing risk, or treating an individual having dementia, frontotemporal dementia, Alzheimer's disease, Nasu-Hakola disease, or multiple sclerosis.
Resumen de: AU2025215148A1
The present disclosure provides for treating Parkinson´s disease (PD) comprising administering acetyl-leucine or a pharmaceutically acceptable salt thereof to a subject in need thereof.
Resumen de: WO2026152919A1
The present invention relates to the field of biopharmaceutical technology, and in particular, to a dual-drug nanoparticle for treating Alzheimer's disease, a method for preparing same, and use thereof. An Aβ inhibitor is connected to an amphiphilic polymer via a chemical bond. The amphiphilic polymer connected with the Aβ inhibitor is mixed with a STING inhibitor, and the mixture is slowly added dropwise into water under ultrasonication. After ultrasonication and filtration, the supernatant is taken to obtain the dual-drug nanoparticle. The dual-drug nanoparticle prepared by the present invention has good biocompatibility and stability, and can cross the blood-brain barrier and act on both the Aβ and microglial STING pathways, so as to depolymerize Aβ and promote Aβ clearance, inhibit the activation of the microglial STING pathway, and reduce the release of inflammatory factors that damage neurons. The dual-drug nanoparticle can significantly reduce Aβ deposition in the brain and significantly inhibit the activation of the microglial STING pathway in the brain, such that the memory ability and spatial recognition ability of mice are remarkably improved, and the inflammatory microenvironment in the brain is ameliorated.
Resumen de: WO2026156134A1
The present invention provides a compound of formula I or a pharmaceutically acceptable salt thereof T-L-B (I), useful for the modulation of TYK2 protein via degradation, their manufacture, pharmaceutical compositions containing them and their use as therapeutically active substances. The active compounds of the present invention are useful in the therapeutic and/or prophylactic treatment of autoimmune disorders like type 1 diabetes, ankylosing spondylitis, cutaneous lupus erythematosus, systemic lupus erythematosus, lupus nephritis, multiple sclerosis, systemic sclerosis, psoriasis, Crohn's disease, ulcerative colitis, proteasome-associated autoinflammatory syndromes (PRAAS), ISG15 deficiency, and inflammatory bowel disease; inflammatory disorders like rheumatoid arthritis, asthma, chronic obstructive pulmonary disease, psoriasis, Crohn's disease, ulcerative colitis, STING-associated vasculopathy with onset in infancy (SAVI). and inflammatory bowel disease; proliferative disorders like hematological cancer, polycythemia vera, myelofibrosis, essential thrombocythemia, and thrombocytosis; endocrine disorders like polycystic ovary' syndrome, Crouzon's syndrome, and type 1 diabetes; neurological disorders like Alzheimer's disease. Aicardi-Goiuteieres syndrome (AGS). Parkinson's disease, amyotrophic lateral sclerosis, Huntington's disease, cerebral ischemia, and neurodegenerative disease caused by traumatic injury, glutamate neurotoxicity and hypoxia: and/or disorders associated
Resumen de: WO2026156364A1
Provided herein is the identification of target proteins for determination of clinical aspects of Alzheimer's disease in a subject. Further provided are methods for diagnosing Alzheimer's disease, determining the progression or rate of memory decline of Alzheimer's disease, and predicting amyloid-tau status, brain amyloidosis status, and plasma p-tau217 status of a subject, using predictor value comparison to thresholds. Also provided are methods of selecting a subject for inclusion in a clinical trial, methods for treating the subject in need thereof, and kits providing the same.
Resumen de: WO2026155574A1
The present invention relates to a papillomavirus virus-like particle-based anti-amyloid beta Alzheimer's disease vaccine and to a vaccine for the purpose of preventing and treating amyloid beta-associated diseases in animals. Specifically, the present invention relates to an Alzheimer's disease vaccine in which an amyloid beta antigenic group is loaded onto virus-like particles of human papillomavirus (HPV). According to the present invention, an amyloid beta peptide fragment is linked to HPV VLPs having excellent structural and immunological properties, thereby greatly enhancing the effect of inducing antibodies against amyloid beta. In addition, it was confirmed that the vaccine prepared according to the present invention exhibits superior immunogenicity and seroconversion rate compared to conventional VLP-based Alzheimer's disease vaccines.
Resumen de: WO2026156335A1
Small molecule mimetics of Carboxypeptidase E (CPE) and related compositions, methods, and systems for providing cytoprotection. Compounds of Formula I, which encompasses specific sub-sets including hydrazone-based (Formula II, III, IV), urea-based (Formula V), and amide-based (Formula VI) scaffolds are configured to bind selectively to the 5-Hydroxytryptamine Receptor 1E (5-HTR1E) to serve as functional mimetics of the neurotrophic protein CPE. Upon binding, the compounds trigger a specific biased signaling pathway that involves the recruitment of beta-arrestin, phosphorylation of ERK, and upregulation of the anti-apoptotic protein BCL-2. The compounds exhibit nanomolar binding affinity and significant cytoprotective activity against oxidative stress in human neurons and peripheral tissues. Also methods for identifying such mimetics using a hybrid virtual screening protocol, synthetic methods comprising a Betti reaction sequence, and therapeutic methods for treating conditions associated with oxidative stress, such as neurodegenerative disorders (e.g., Alzheimer's Disease) and peripheral organ injury, via intranasal or systemic administration.
Resumen de: WO2026154410A1
Described herein are novel compounds capable of binding to the protein synapsin III and reducing the fibrillar aggregates of said protein with alpha-synuclein. The novel compounds of the invention are therefore suitable for use in the therapeutic treatment of neurodegenerative diseases such as, in particular, Parkinson's disease, Lewy body dementia, multiple system atrophy, and the Lewy body variant of Alzheimer's disease. A pharmaceutical composition comprising the compounds according to the invention in a pharmaceutically acceptable carrier, as well as in combination with optional adjuvants, stabilizers and/or preservatives, is also described.
Resumen de: US20260209695A1
0000 Disclosed are novel strategies for the treatment of patients with Parkinson's disease and other secondary Parkinsonian disorders. Disclosed are DA neuronal cells that have been modified in vitro with a genetic insertion of GDNF. The GDNF coding sequence is inserted under the transcriptional control of a promotor such that secreted proteins are made and taken up by the endogenous cells after the administered engineered cells have matured to a Neuronal Mature Cell Type to promote survival of endogenous neurons. Also disclosed are DA neuronal cells that have been modified with a genetic insertion of GBA in vitro and are hemizygous null for SNCA. The GBA coding sequence is inserted under the transcriptional control of a ubiquitous promotor and the secreted proteins are made and taken up by the graft and endogenous cells immediately upon transplantation to promote long-term graft integrity.
Resumen de: WO2026152239A1
The present disclosure provides oral film compositions for transmucosal delivery of xanomeline and trospium chloride for treating schizophrenia and Alzheimer's disease. The compositions comprise xanomeline or a pharmaceutically acceptable salt thereof, trospium or a pharmaceutically acceptable salt thereof, at least one film-forming polymer, and d-alpha- tocopheryl polyethylene glycol succinate (TPGS) as a permeation enhancer, wherein the composition has a pH of from about 4.5 to about 7. The formulations address challenges including poor aqueous solubility, pH-dependent stability, and permeability limitations. Buccal administration enables transmucosal absorption bypassing first-pass hepatic metabolism, thereby mitigating gastrointestinal side effects and food effects compared to conventional oral capsule formulations. The disclosure demonstrates a synergistic effect wherein co-formulation of trospium chloride with xanomeline tartrate unexpectedly enhances transmucosal permeation of trospium chloride compared to films containing trospium chloride alone. Methods of treatment and manufacturing processes are also provided.
Resumen de: AU2025231111A1
Provided herein are antibodies that target Galectin-3. Such antibodies are used in methods of treating epilepsy and related neurological disorders, such as Alzheimer's disease (AD) and Parkinson's disease (PD).
Resumen de: AU2026205314A1
Abstract Dosage regimens for SOD1-targeting antisense oligonucleotides, and salts thereof, are provided. These dosage regimens find use in the treatment of subjects having or at risk of developing amyotrophic lateral sclerosis. Abstract ul b s t r a c t u l
Resumen de: US20260207794A1
The present invention relates to novel compounds of formula (I), or a detectably labelled compound, stereoisomer, racemic mixture, pharmaceutically acceptable salt, hydrate, or solvate thereof, that can be employed in the imaging of alpha-synuclein aggregates and determining an amount thereof. Furthermore, the compounds can be used for diagnosing a disease, disorder or abnormality associated with an alpha-synuclein aggregates (such as Parkinson's disease or such as multiple system atrophy (MSA)) determining a predisposition to such a disease, disorder or abnormality, prognosing such a disease, disorder or abnormality, monitoring the evolution of the disease in a patient suffering from such a disease, disorder or abnormality, monitoring the progression of such a disease, disorder or abnormality and predicting responsiveness of a patient suffering from such a disease, disorder or abnormality to a treatment thereof.
Resumen de: US20260209725A1
0000 Compounds, compositions, uses, and methods for increasing cell viability of a dopaminergic neuron, or for preventing or treating dopaminergic neuronal death, are provided herein. In certain examples, methods for reducing symptoms and/or for preventing or treating Parkinson's disease in a subject in need thereof are provided which may include a step of treatment with a GDP-bound form of Rab1a (Rab1a
Resumen de: US20260207575A1
Provided herein a method of treating Juvenile Huntington disease in a subject in need thereof comprising orally administering a pharmaceutical composition comprising pridopidine and/or its analog or a pharmaceutically acceptable salt thereof.
Resumen de: US20260209771A1
The present disclosure relates to an antisense oligonucleotide suitable for editing an adenosine in a target RNA nucleic acid sequence to inosine (A-to-I editing), wherein the antisense oligonucleotide comprises a sequence that is substantially complementary to the target RNA nucleic acid sequence: 5′-A C U A C A G C A U U G C-3′ (SEQ ID NO: 1), or complementary to a sequence that has at least 80% sequence identity to SEQ ID NO: 1, wherein A is the target adenosine to be edited in the target nucleic acid; and the antisense oligonucleotide can be represented by: 3′-X−5 X−4 X−3 X−2 X−1 X0 X+1 X+2 X+3 X+4 X+5 X+6 X+7-5′, wherein X represents a nucleic acid, X0 represents cytosine (C), deoxycytosine (dC), or uracil (U) directly opposite to the target adenosine to be edited, and wherein there is a mismatch between the antisense oligonucleotide and the target nucleic acid at one or more of the following position(s): a) −5 (X−5); b)+3 (X+3); c)+7 (X+7); d)+7 (X+7) and +2 (X+2); e)+7 (X+7) and −5 (X−5); or f)+6 (X+6) and −5 (X−5).
Resumen de: US20260207625A1
0000 The present invention is directed to the compound golexanolone for use in the treatment of Parkinson's Disease (PD) or for use in the treatment of L-dopa Induced Dyskinesia (LID) in Parkinson's Disease (PD) patients. Further, the present invention is directed to the compound golexanolone or a pharmaceutically acceptable salt thereof, for use in the treatment of Parkinson's Diseases (PD) patients, in particular PD patients exhibiting a L-dopa Induced Dyskinesia (LID).
Resumen de: EP4778582A2
The present invention relates to antisense oligonucleotides that are complimentary to SOD1, leading to decreased expression of SOD1. Reduced expression of SOD1 is beneficial in medical disorders such as Amyotrophic Lateral Sclerosis.
Nº publicación: EP4778518A2 22/07/2026
Solicitante:
GOLDEN GAIT BIOTECH S L [ES]
Golden Gait Biotech, S.L.
Resumen de: EP4778518A2
0001 The present disclosure provides methods and compositions for treating fall-related symptoms in patients with neurodegenerative diseases such as Parkinson's disease or Parkinson-related diseases. In some embodiments, the disclosure utilizes nicotine or a salt thereof in combination of dopaminergic agent treatments for reducing fall-related symptoms such as reducing frequency of fall, reducing injuries related to fall, reducing severity of injuries related to fall, freezing of gait, improving posture stability, improving locomotion ability, improving balance and gait. In some embodiments, the methods predict fall frequency and tendency of recurrent falls in patients with Parkinson's disease, in particular, patients with typical Parkinson's disease.