Absstract of: WO2026032004A1
The present invention relates to a method for treating Parkinsonism or Parkinson's disease, and a pharmaceutical composition, and specifically relates to a pharmaceutical composition, the use and a method of a peripheral μ-opioid receptor antagonist for treating Parkinsonism or Parkinson's disease. The pharmaceutical composition, use and method can significantly ameliorate symptoms of Parkinsonism or Parkinson's disease, including constipation, particularly constipation that is unresponsive or refractory to treatment with general-purpose constipation drugs.
Absstract of: AU2024299217A1
The present invention relates to the use of bis(phenylmethylene)cycloalkanones and heterocyclic analogues thereof in human and veterinary medicine, for the treatment of diseases caused by the presence or elevated levels of metastable proteins in the cell, imbalances in protein homeostasis and proteotoxic stress, in general proteinopathy, in particular their use in the treatment of neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS), Parkinson's disease (PD), Alzheimer's disease (AD), Kennedy's disease (KD), Huntington's disease (HD), Creutzfeldt-Jakob disease (CJD), spinocerebellar ataxia (SCA), dentatorubral- pallidoluysian atrophy, transthyretin familial amyloid polyneuropathy, systemic amyloidosis, organ- confined amyloidosis, cystic fibrosis, and diabetes. Furthermore, the invention provides novel bis(phenylmethylene)cycloalkanones.
Absstract of: AU2024315761A1
Among other things, the present disclosure provides various technologies including chirally controlled oligonucleotide compositions and technologies for manufacturing and using such oligonucleotide compositions. In some embodiments, the present disclosure provides technologies useful for allele-specific knockdown of mutant Huntingtin transcripts. In some embodiments, the present disclosure provides technologies useful for reducing the expression, level, amount, and/or activity of mutant Huntingtin transcripts or products thereof. In some embodiments, the present disclosure provides methods for treating Huntington's disease.
Absstract of: WO2026036133A1
The invention provides binding agents targeted to the aggregated form of amyloid-beta (Aβ) peptide, nucleic acids comprising the inventive binding agents, vectors and cells comprising the inventive nucleic acids, and pharmaceutical compositions thereof. The invention also provides methods for treating or preventing a disease, disorder, or condition, in particular, Alzheimer's disease, in a mammal, by administering the binding agents or compositions thereof. The invention further provides methods for inducing clearance of an aggregated form of amyloid-beta (Aβ) peptide in a mammal, comprising administering the inventive binding agents and pharmaceutical compositions described herein.
Absstract of: AU2024343037A1
Provided is the new compositions and methods useful for the treatment, prevention, and potential cure of a genetic disease, such as familial Alzheimer's Disease, by disrupting the genomic sequence harboring one or more SNPs that are of high prevalence in a population but no relevance to the particular disease except for their genomic locations being in the same gene exon as a disease-relevant allele and upstream from such disease-relevant allele present in the genome of a treatment recipient.
Absstract of: WO2026036016A1
Methods and compositions described herein provide a solution for treating Huntington's disease and other neurodegenerative diseases by administering a recombinant polypeptide, such as a wild-type Huntingtin (HTT) fragment (e.g., SEQ ID NO:4), Brahma-related gene 1 (BRG1), Brahma (BRM), PNKP, Matrin 3 (MATRN3), or functional variants thereof, to a subject having, at risk of developing, or suspected of having a neurodegenerative disease characterized by persistent DNA double-strand breaks and impaired RNA processing. The polypeptides, delivered via mRNA, circular RNA, or AAV vectors, restore transcription-coupled non-homologous end-joining (TC-NHEJ) activity and RNA processing, ameliorating disease progression in neuronal and non-neuronal brain cells.
Absstract of: AU2024330245A1
Provided are a gene therapy vector for treating Parkinson's disease and a use thereof. Specifically, provided is an adeno-associated virus (AAV) vector for treating Parkinson's disease, which can simultaneously express functional tyrosine hydroxylase (TH), GTP-cyclohydrolase 1 (GCH1) and aromatic amino acid decarboxylase (AADC) to promote dopamine synthesis. Also provided are an AAV virus particle containing the AAV vector, a composition containing the AAV vector or the AAV virus particle, and uses of the AAV vector, the AAV virus particle and the composition in the preparation of drugs for preventing or treating Parkinson's disease.
Absstract of: WO2026032421A1
Provided herein are anti-overdosing compositions and uses thereof. Specifically, provided herein are anti-overdosing pharmaceutical compositions comprising a mitochondrial uncoupler and a GLP-1R agonist and uses thereof as well as methods for preventing overdosing of the uncoupler. Such pharmaceutical compositions can be useful for treating diseases, such as but not limited to NASH, overweight, obesity, medical complications related to overweight or obesity, type 2 diabetes (T2D), and Alzheimer's disease and related dementias (AD/ADRD).
Absstract of: US20260043791A1
A kit for diagnosing Alzheimer's disease and a pharmaceutical composition for treating Alzheimer's disease are disclosed, in which EDIL3 or a nucleic acid encoding EDIL3 is used as an index or target.
Absstract of: US20260042778A1
Novel compositions of N-desmethyl ruboxistaurin L-lactate salt and L-lactate salt polymorphs. The use of compositions of N-desmethyl ruboxistaurin L-lactate salt and polymorphs to modulate GSK-3 signaling is disclosed, as is the use of compositions of N-desmethyl ruboxistaurin L-lactate salt and polymorphs to inhibit protein kinase C. Methods are also disclosed of using compositions of N-desmethyl ruboxistaurin L-lactate salt and polymorphs in the treatment of subjects having a neurological disease and/or psychiatric disorder, including Alzheimer's disease, bipolar disorder, depression, schizophrenia, Parkinson's disease, or neuroinflammation, as well as methods of using compositions of N-desmethyl ruboxistaurin L-lactate salt and polymorphs in treating conditions associated with diabetes mellitus or its complications, or ischemia, inflammation, pulmonary hypertension, congestive heart failure, cardiovascular disease, dermatological disease, or cancer. In addition, compositions of N-desmethyl ruboxistaurin L-lactate salt and polymorphs administered in combination with lithium or other treatments for bipolar disorder are also disclosed.
Absstract of: US20260041670A1
The present disclosure discloses drugs for preventing and/or treating Alzheimer's disease (AD). A CF3CN derivative provided by present disclosure has any one of structural formulas 1-4 shown below. All four CF3CN derivatives have TrkB agonist activities; and specifically, the CF3CN derivative shown in formula 2 serves as an optimal derivative. In vivo PK studies reveal that the CF3CN derivative shown in the formula 2 is capable of improving a B/P Ratio of CF3CN, and overcoming the limitations of CF3CN. Nanoparticles are prepared by encapsulating the CF3CN derivatives with zein and lactoferrin, which may further enhance an oral bioavailability and a brain drug concentration, thereby improving AD treatment effects. By further improving the formulation and administration route, a liposome is employed to encapsulate the CF3CN derivative for both oral and intranasal administration, which effectively solves the problems of low bioavailability and low brain drug concentration of the derivative.
Absstract of: US20260042801A1
Embodiments of the disclosure include methods and compositions related to use of compound comprising a particular peptide linked to a dye. In specific embodiments, the peptide DIRG is linked to the BODIPY dye. In specific embodiments, the compositions are utilized for treatment of neurological conditions and secondary pathologies related therewith, such as spinal cord injury and Alzheimer's Disease, as examples.
Absstract of: EP4691472A1
For dementia, particularly Alzheimer's disease, there is currently no effective agent for ameliorating or treating it. The object of the present invention is to find a drug for ameliorating or treating dementia thereby providing a novel agent for ameliorating or treating dementia. The present invention provides an agent for ameliorating or treating dementia, which comprises cyclic phosphatidic acid, carbacyclic phosphatidic acid, thiacyclic phosphatidic acid, or carba-lysophosphatidic acid, or a salt thereof.
Absstract of: US20260034233A1
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).
Absstract of: WO2026028165A1
The present disclosure encompasses engineered meganucleases that bind and cleave recognition sequences within a C9Orf72 gene. The present disclosure also encompasses methods of using such engineered meganucleases to make genetically modified cells. Further, the disclosure encompasses pharmaceutical compositions comprising engineered meganuclease proteins, or polynucleotides encoding engineered meganucleases of the disclosure, and the use of such compositions for the modification of a C9Orf72 gene in a subject, or for treatment of amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD).
Absstract of: WO2026028189A1
The present invention relates to a mixture, compositions comprising said mixture, and their use in the treatment and/or prevention of neurodegenerative diseases affecting motor neurons and/or due to demyelination processes, such as Amyotrophic Lateral Sclerosis (ALS) and Multiple Sclerosis (MS). In greater detail, the present invention relates to a mixture comprising or, alternatively, consisting of: i) an extract of chamomile; ii) an extract of turmeric; iii) L-acetyl carnitine or a salt thereof; iv) a blueberry extract; and v) lipoic acid, or a salt thereof, and/or a bergamot extract.
Absstract of: WO2026030341A1
The present disclosure provides a method of treating or inducing cognitive preservation in a patient with Alzheimer's disease, comprising administering an effective amount of CT1812. The effective amount of CT1812 is 100 mg or 300 mg administered orally once daily. The method demonstrates improvements in cognitive outcomes compared to placebo across multiple measures including ADAS-Cog11, MMSE, ADCS-ADL, and ADCS-CGIC. The method demonstrates improvements in cognitive outcomes in patients with low plasma levels of phosphorylated tau 217 prior to administration.
Absstract of: WO2026030590A1
The present disclosure provides recombinantly manufactured fusion proteins comprising a HERV-K (HML-2) Env protein fragment or an analog thereof linked to a human Fc fragment. Embodiments include the administration of the fusion proteins to patients having a disease or a disorder with the intention of mitigating and/or reducing the duration of symptoms associated with the condition or disease (for example but not limited to muscular weakness, paralysis and respiratory failure), and/or preventing symptoms associated with the condition or disease, for example, by preventing motor neuron degeneration and cell death in ALS patients associated with the condition or disease. Accordingly, "treatment" generally means both therapeutic treatment and prophylactic or preventative measures. Improvement after treatment may be manifested as a decrease or elimination of such symptoms, e.g., by a decrease or elimination of symptoms associated with ALS, and/or by a decrease in the duration of such symptoms.
Absstract of: WO2026030596A1
The present disclosure provides recombinantly manufactured fusion proteins comprising a HERV-K (HML-2) Env protein fragment or an analog thereof linked to a human Fc fragment. Embodiments include the administration of the fusion proteins to patients having a disease or a disorder with the intention of mitigating and/or reducing the duration of symptoms associated with the condition or disease (for example but not limited to muscular weakness, paralysis and respiratory failure), and/or preventing symptoms associated with the condition or disease, for example, by preventing motor neuron degeneration and cell death in ALS patients associated with the condition or disease. Accordingly, "treatment" generally means both therapeutic treatment and prophylactic or preventative measures. Improvement after treatment may be manifested as a decrease or elimination of such symptoms, e.g., by a decrease or elimination of symptoms associated with ALS, and/or by a decrease in the duration of such symptoms.
Absstract of: US20260035342A1
There are provided isotope-enriched compounds of Formula (I) and pharmaceutically acceptable salts or esters thereof, as well as pharmaceutical compositions thereof and methods of use thereof for prevention and treatment and amyloid-β related diseases, such as Alzheimer's disease.
Absstract of: WO2026030249A1
Described herein is an active Alzheimer's Disease (AD) immunotherapy based on a nanoparticle vaccine comprising a plurality of Aβ peptides and/or a plurality of tau peptides. These peptides may correspond to both soluble and aggregated targets and are displayed on the surface of immunogenic liposomes in an orientation that maintains reactivity with epitope-specific monoclonal antibodies. Also provided are methods of making and using same.
Absstract of: US20260034085A1
The invention is a method for treating patients with Parkinson's disease by orally administering a controlled release levodopa formulation and the method provides an improvement of a patient's total post-dose “Off” time, total post dose “On” time and total post dose “Good On” time compared to post-dose of treatment regimens with oral immediate release levodopa tablets.
Absstract of: US20260034088A1
Provided herein is the use of a compound of Formula I:or a pharmaceutically acceptable salt thereof, for treating a disease characterized by amyloid and amyloid-like aggregates, e.g., Alzheimer's disease.
Absstract of: WO2026027658A1
The invention refers to the field of neurodegenerative diseases including Alzheimer's Disease and a treatment thereof with a Benzoxazepine compound. Benzoxazepine compounds are e.g. known in the treatment of breast cancer and according to the use of the invention are a first therapeutic and prophylactic treatment for neurodegenerative diseases, including Alzheimer's Disease. The invention further relates to test systems for identifying, mapping, elaborating and evaluating said and further therapeutic and prophylactic uses of compounds of interest and/or other pharmaceutical compositions for the treatment of neurodegenerative diseases, including Alzheimer's Disease. (AD)
Nº publicación: US20260034121A1 05/02/2026
Applicant:
HWANG HELEN [US]
KOTZBAUER PAUL [US]
DHAVALE DHRUVA [US]
WASHINGTON UNIV [US]
Hwang Helen,
Kotzbauer Paul,
Dhavale Dhruva,
Washington University
Absstract of: US20260034121A1
Provided herein are compounds, compositions, and methods for inhibiting fibril growth. Compositions include at least one alpha-Synuclein (aSyn) inhibiting agent in the form of a compound including a dimethyoxyphenyl piperazine group. Methods include inhibiting aSyn fibril growth and treating a neurodegenerative disease in a subject in need thereof, including Parkinson's Disease and Lewy Body disease (LBD). Methods including administering a composition of the present disclosure. Further provided is a system for detecting aSyn fibril growth in a subject in need thereof, the system including a fluorescence screening assay of the present disclosure.