Alerta

MUTATION-INDEPENDENT ALLELE-SPECIFIC CRISPR TARGETING STRATEGY FOR TREATING GENETIC DISEASES

Resumen de: 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.

GENE THERAPY VECTOR FOR TREATING PARKINSON'S DISEASE AND USE THEREOF

Resumen de: 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.

COMPOSITIONS AND METHODS FOR DYE-BOUND CYCLIZED PEPTIDES FOR MEDICAL USE

Resumen de: 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.

DRUGS FOR PREVENTING AND/OR TREATING ALZHEIMER'S DISEASE

Resumen de: 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.

Polymorphs of N-Desmethyl Ruboxistaurin and Salts Thereof

Resumen de: 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.

KIT FOR DIAGNOSING ALZHEIMER'S DISEASE AND PHARMACEUTICAL COMPOSITION FOR TREATING ALZHEIMER'S DISEASE

Resumen de: 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.

ANTI-OVERDOSING COMPOSITIONS AND USES THEREOF

Resumen de: 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).

METHOD FOR TREATING PARKINSONISM OR PARKINSON'S DISEASE, AND PHARMACEUTICAL COMPOSITION

Resumen de: 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.

POTENT, AGGREGATE-SELECTIVE ANTI-Aβ ANTIBODIES AND USES THEREOF

Resumen de: 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.

METHODS AND COMPOSITIONS FOR TREATING NEUROLOGIC DISORDERS

Resumen de: 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.

COMBINATORIAL THERAPY FOR ALZHEIMER'S DISEASE

Resumen de: WO2026035942A1

The present invention provides novel compositions and methods for treating diseases and conditions associated with amyloid beta in a subject, said method comprising administering to the subject (i) an anti-amyloid beta antibody or an antigen binding fragment thereof and (ii) a regulatory T cell (Treg) inducing or activating agent.

METHODS TO EVALUATE EARLY-STAGE PRE-TANGLE TAU AGGREGATES AND TREATMENT OF ALZHEIMER'S DISEASE

Resumen de: WO2026033422A1

Provided herein is a method of identifying a pre-stage neurofibrillary tangle (NFT) in a patient sample, including obtaining a sample from a patient suspected of having or at risk of developing a tauopathy, incubating the sample with a composition comprising a first binding reagent, wherein the first binding reagent is specific to Ser262 and/or Ser356 of a tau protein, and detecting binding between the first binding reagent and the tau protein, wherein detecting binding between the first binding reagent and the tau protein indicates the presence of a pre-stage NFT in the patient sample.

IMPROVING/THERAPEUTIC DRUG FOR DEMENTIA, PARTICULARLY ALZHEIMER-TYPE DEMENTIA

Resumen de: 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.

ANTI-GALECTIN 3 ANTIBODIES AND THEIR USE IN EPILEPSY AND RELATED DISEASES

Resumen de: 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).

LEVODOPA DOSING REGIMEN

Resumen de: 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.

AGENTS, COMPOSITIONS AND METHODS FOR TREATING AND PREVENTING ALZHEIMER'S DISEASE

Resumen de: US20260034143A1

Compositions of Allopregnanolone (Allo), and methods of use thereof for treating and preventing Alzheimer's Disease (AD) or dementia have been developed. In some embodiments, the amount of Allo effective to treat AD or dementia is between about 2 mg and about 10 mg, preferably 4 mg per dose. Methods for identifying subjects for treatment of AD or dementia are also provided. The methods include selecting a subject having one or more Apo E4 gene alleles. Methods of treating a human subject having AD or at risk of AD OR DEMENTIA are provided. The methods include administering a dosage of from 2 mg to 6 mg to the subject once within a 24 hour period. The dosing is repeated every seven days, or less frequently. The methods stimulate mitosis of neural progenitor cells, stimulate neurite growth and organization, protect against neural loss, or one or more of these neural processes.

METHOD OF TREATING AMYOTROPHIC LATERAL SCLEROSIS WITH PRIDOPIDINE

Resumen de: US20260034109A1

Provided herein is a method for treating a human subject afflicted with ALS by administering to the subject a therapeutically effective amount of pridopidine or pharmaceutically acceptable salt thereof.

COMPOSITIONS AND METHODS FOR ALPHA-SYNUCLEIN FIBRIL GROWTH INHIBITION

Resumen de: 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.

COMPOUNDS FOR TREATING HUNTINGTON'S DISEASE

Resumen de: US20260035387A1

The present disclosure provides a compound of Formula (I′), or a pharmaceutically acceptable salt thereof and its use in, e.g. treating a condition, disease, or disorder in which lowering mutant huntingtin protein (“mHTT”) in a subject is of therapeutic benefit, specifically in treating Huntington disease (“HD”). This disclosure also features a composition containing the same as well as methods of using and making the same.

COMPOSITIONS FOR ALZHEIMER'S DISEASE VACCINES

Resumen de: 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.

ISOTOPE-ENRICHED 3-AMINO-1-PROPANESULFONIC ACID DERIVATIVES AND USES THEREOF

Resumen de: 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.

SULFOPROPANOIC ACID DERIVATIVES FOR TREATING NEURODEGENERATIVE DISORDERS

Resumen de: 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.

HERV-K (HML-2) ENV ANALOG FUSION PROTEINS FOR ANTIGEN SPECIFIC IMMUNOTHERAPY AND METHODS OF USE

Resumen de: 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.

ZERVIMESINE FOR TREATING NEURODEGENERATIVE DISEASE

Resumen de: 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.

HERV-K (HML-2) ENV ANALOG FUSION PROTEINS FOR ANTIGEN SPECIFIC IMMUNOTHERAPY AND METHODS OF USE

Nº publicación: WO2026030590A1 05/02/2026

Solicitante:

TWILIGHT BIOSCIENCE INC [US]
TWILIGHT BIOSCIENCE, INC

Resumen de: 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.