Alerta

RECONSTITUTED LIPOPROTEIN PARTICLES TO RESCUE LIPID DEFECTS IN THE ALZHEIMER'S BRAIN

Absstract of: WO2025117721A1

The present disclosure provides products and methods for facilitating brain lipid transport in a subject. In some aspects the products are useful for slowing the progression of or preventing the development of Alzheimer's Disease or for treating Alzheimer's disease. The products include reconstituted lipoprotein particles (rLPs). Libraries and screening of libraries to identify additional rLPs are also disclosed.

PHARMACEUTICAL PREPARATION FOR DIAGNOSING AND TREATING ALZHEIMER'S DISEASE

Absstract of: EP4818330A1

The present invention relates to the field of biomedicine. Provided are a marker that can be used for early diagnosis of Alzheimer's disease, and the use thereof. Specifically provided is a marker for early diagnosis of Alzheimer's disease. The marker is a Maf1 gene or the protein rhereof. High expression of the Maf1 gene or the protein thereof in neuronal cells indicates that a subject is at high risk of developing Alzheimer's disease. The method provides a new diagnostic and therapeutic target for AD.

POLYCYCLIC COMPOUND AND USE THEREOF

Absstract of: EP4818163A1

The present disclosure belongs to the technical field of medicine, and particularly discloses a polycyclic compound as represented by formula I and use thereof. The polycyclic compound of the present disclosure has a structure as represented by formula I, and definitions of each group and substituent are as described in the specification. The compound of the present disclosure can be used for treating or preventing neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, progressive multiple sclerosis, and amyotrophic lateral sclerosis.

CRYSTAL FORMS OF 20(S)-PROTOPANAXADIOL, PREPARATION METHOD THEREFOR AND USE THEREOF

Absstract of: EP4818185A1

The present application 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 application 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.

ANTI-Aβ1-40 ANTIBODY, PREPARATION METHOD THEREFOR, AND USE THEREOF

Absstract of: WO2026199817A1

The present application provides a method for preparing a humanized Aβ1-40 antibody and use thereof. Specifically, the antibody of the present application combines a murine antibody variable region and a human antibody framework region; on the basis of specifically binding to an Aβ peptide segment, the antibody reduces immunogenicity due to its humanized framework region. The antibody has the advantages of high affinity, high specificity, low immunogenicity, and scalability for large-scale production. The antibody of the present application can be used for detecting an Aβ1-40 antigen, eliminating Aβ deposits, or treating related diseases thereof, such as Alzheimer's disease.

ANTI-Aβ1-42 ANTIBODY, PREPARATION METHOD THEREFOR, AND USE THEREOF

Absstract of: WO2026199818A1

The present application provides a method for preparing a humanized Aβ1-42 antibody and use thereof. Specifically, the antibody of the present application combines a murine antibody variable region and a human antibody framework region; on the basis of specifically binding to an Aβ peptide segment, the antibody reduces immunogenicity due to its humanized framework region. The antibody has the advantages of high affinity, high specificity, low immunogenicity, and scalability for large-scale production. The antibody of the present application can be used for detecting an Aβ1-42 antigen, eliminating Aβ deposits, or treating related diseases thereof, such as Alzheimer's disease.

BIPARATOPIC ANTIBODIES TARGETING TAU PROTEIN PATHOLOGY: COMPOSITION AND METHODS FOR ENHANCED THERAPEUTIC EFFICACY

Absstract of: WO2026202374A1

The present invention relates to anti-Tau biparatopic bispecific antibodies (referred as Tau biparatopic κλ-bodies) or fragments thereof, or a mixture, to be used for the prevention, and treatment of tauopathies including Alzheimer's disease. The antibodies could also be used for diagnosis purposes before or after symptoms occur. Lastly, it could be used to monitor the treatment effect.

Antibody

Absstract of: WO2026202512A1

The invention relates to antibodies, and specifically to anti-amyloid-beta (Aβ) antibodies. The invention extends to compositions comprising the antibodies, including pharmaceutical compositions and kits. The invention also extends to methods of making and using the antibodies, for example in therapy and diagnosis of Aβ-mediated 5 conditions, such as Alzheimer's disease.

AROMATIC HETEROCYCLE-FUSED CYCLOHEXYL AMINOALKYL PIPERIDINE DERIVATIVE, PREPARATION METHOD AND USE THEREOF

Absstract of: US20260297079A1

0000 The present invention relates to an aromatic heterocycle-fused cyclohexyl aminoalkyl piperidine derivative, a preparation method and use thereof. Specifically, the present invention provides a compound of general formula (I), a stereoisomer thereof, a tautomer thereof, or a pharmaceutically acceptable salt thereof, and a preparation method therefor, use thereof for activating the activities of a 5-HT<1A >receptor and dopamine D<2 >and D<3 >receptors, and use thereof in the preparation of a medicament for Parkinson's disease. 0000

ANTI-TAU ANTIBODIES

Absstract of: AU2025239656A1

The present disclosure provides anti-tau antibodies, including compositions and methods of using such antibodies for treating Alzheimer's disease.

USES OF REGULATORY T CELLS FOR TREATING AMYOTROPHIC LATERAL SCLEROSIS

Absstract of: AU2025239644A1

Provided herein are medical uses of CD11a-enriched T regulatory cells for treating amyotrophic lateral sclerosis (ALS) or ameliorating a symptom of ALS. Such uses may include longitudinal analysis of biomarkers, such as inflammatory cytokines.

1 H-PYRROLO2,3-BPYRIDIN-4-YL-2-OXOPYRROLIDINE-3-CARBONITRILE DERIVATIVES AS TYROSINE KINASE 2 (TYK2) INHIBITORS FOR THE TREATMENT OF INFLAMMATORY DISEASES

Absstract of: US20260297085A1

This disclosure relates to compounds of Formula (I-1) or (I-2): The compounds of the present disclosure are capable of inhibiting the activity of tyrosine kinase 2 (TYK2) and are useful for the treatment of diseases or disorders, such as e.g. inflammation, autoimmune disease, neuroinflammation, arthritis, rheumatoid arthritis, spondyloarthropathies, systemic lupus erythematous, lupus nephritis, arthritis, osteoarthritis, gouty arthritis, pain, fever, pulmonary sarcoisosis, silicosis, cardiovascular disease, atherosclerosis, myocardial infarction, thrombosis, congestive heart failure and cardiac reperfusion injury, cardiomyopathy, stroke, ischaemia, reperfusion injury, brain edema, brain trauma, neurodegeneration, liver disease, inflammatory bowel disease, Crohn's disease, ulcerative colitis, nephritis, retinitis, retinopathy, macular degeneration, glaucoma, diabetes (type 1 and type 2), diabetic neuropathy, viral and bacterial infection, myalgia, endotoxic shock, toxic shock syndrome, autoimmune disease, osteoporosis, multiple sclerosis, endometriosis, menstrual cramps, vaginitis, candidiasis, cancer, fibrosis, obesity, muscular dystrophy, polymyositis, dermatomyositis, autoimmune hepatitis, primary biliary cirrhosis, primary sclerosing cholangitis, vitiligo, alopecia, Alzheimer's disease, skin flushing, eczema, psoriasis, atopic dermatitis and sunburn. The disclosure further provides methods of preparing the compounds.

METHODS OF TREATING OXIDIZED PHOSPHATIDYLCHOLINE-ASSOCIATED DISEASES

Absstract of: US20260297217A1

0000 Provided herein are methods of treating diseases and disorders related to TDP-43 aggregation (e.g., ALS) with an antibody that specifically binds to OxPC or a polynucleotide encoding an antibody that specifically binds to OxPC.

Inhibitors of JUN N-Terminal Kinases (JNK1, JNK2, and/or JNK3) and Mitogen-Activated Protein Kinases (MAPK8, MAPK9, and/or MAPK10) and Methods of Using Same

Absstract of: US20260294922A1

The present disclosure relates, in part, to compounds of Formula (I) and (II), which selectively inhibit JUN N-Terminal Kinases (JNK1, JNK2, and/or JNK3; also known as MAPK8, MAPK9, and/or MAPK10), pharmaceutical compositions thereof, and methods of using the same for the treatment, prevention, and/or amelioration of one or more diseases and/or disorders in a subject. In certain embodiments, the inflammatory disease or disorder is endometriosis, arthritis, pulmonary fibrosis, cancer, type 1 and/or 2 diabetes, Alzheimer's disease, Parkinson's disease, or amyotrophic lateral sclerosis. In certain embodiments, the methods described herein further comprise detecting the disease and/or disorder in the subject with a suitable diagnostic method.

AGENT FOR TREATING OR PREVENTING AMYOTROPHIC LATERAL SCLEROSIS

Absstract of: US20260294875A1

An agent for treating or preventing amyotrophic lateral sclerosis, wherein an active component of the agent essentially consists of at least one selected from the group consisting of cycloserine, terizidone, and salts thereof. The active component may consist of at least one selected from the group consisting of cycloserine, terizidone, and salts thereof. The cycloserine may be D-cycloserine. The cycloserine may be L-cycloserine.

DUAL VECTOR SELF-INACTIVATING CRISPR/CAS9 SYSTEM

Absstract of: AU2025224578A1

A self-inactivating CRISPR/Cas9 delivery system utilizing a dual vector system may be provided. A first viral vector includes an expression unit for expression of a Cas9 nuclease and a nucleotide sequence encoding an sgRNA targeting a specific genomic locus. The second viral vector includes a nucleotide sequence encoding an sgRNA targeting expression of the Cas9 nuclease by the expression unit. The self-inactivating CRISPR/Cas9 dual vector delivery system can be used for treating a genetic gene-associated disease/disorder and/or a sporadic gene-associated disease/disorder. In embodiments, the disease and/or disorder may be a neurological disease and/or disorder. In embodiments, neurological disease and/or disorder may be Huntington's disease. Methods of treatment, medicaments, and pharmaceutical compositions may be provided.

METHODS FOR DECREASING NEURONAL DEATH, INFLAMMATION, AND DEGENERATION

Absstract of: US20260297571A1

0000 The present disclosure provides compositions and methods for treating ALS and/or FTD and for reducing neuroinflammation and neurodegeneration, e.g., as caused by dipeptide repeat (DPR) toxicity. Particularly, the disclosure provides methods for improving neuron survival, decreasing neuroinflammation and treating ALS and/or FTD using PTPσ inhibitors.

USE OF MIMETIC PEPTIDE CONTAINING FIBRINOGEN RGD MOTIF IN PREPARATION OF MEDICATIONS FOR PREVENTING AND/OR TREATING PARKINSON'S DISEASE

Absstract of: US20260295017A1

This disclosure belongs to the technical field of biomedicine, and discloses use of a mimetic peptide containing a fibrinogen RGD motif in preparation of medications for preventing and/or treating Parkinson's disease. A sequence of the mimetic peptide containing the fibrinogen RGD motif of this disclosure is: Gly-Arg-Gly-Asp-Ser-Pro-Leu-Ala-Pro-Ser-Cys. The applicant of this disclosure has found through research that there is abnormal accumulation of FG in substantia nigra pars compacta of PD mice, and that excessively accumulated FG in the brain may promote abnormal aggregation of α-syn in dopaminergic neurons under mediation of αvβ3 integrin receptor. The mimetic peptide provided by this disclosure can effectively inhibit the abnormal aggregation of α-syn in the dopaminergic neurons and death of the dopaminergic neurons caused by the abnormally accumulated FG in the brain, improve a motion ability of MPTP-modeled PD mice, and provide a new target and idea for PD treatment.

2-AZASPIRO3.4OCTANE DERIVATIVES AS M4 AGONISTS

Absstract of: EP4813390A2

Provided herein are compounds according to Formula (I)or a pharmaceutically acceptable salt thereof, wherein R¹, R², R³, R⁵, and R⁷ are defined herein. Also provided herein are pharmaceutical compositions comprising a compound of Formula (I) as well as the use of such compounds as M4 receptor agonists.

IMIDAZO 1,2-B PYRIDAZINE-BASED NOVEL COMPOUND AS CDK INHIBITOR AND USE THEREOF

Absstract of: EP4813991A1

Provided are a novel compound, a use thereof for inhibiting the activity of CDK12 and/or CDK13 and degrading cyclin K, a pharmaceutical composition comprising the same, and a use thereof for the treatment of diseases associated with CDK12 and/or CDK13 and/or cyclin K, such as cancer. According to the present invention, the compounds exhibit excellent inhibitory activity and selectivity against CDK12 and/or CDK13, and induce degradation of cyclin K, and thus can be useful for the treatment of cancers such as breast cancer or gastric cancer.

PREPARATION OF QUINAZOLINONE DERIVATIVE AS KINASE INHIBITOR, AND USE THEREOF

Absstract of: EP4813985A1

Provided are a compound represented by formula I, a stereoisomer, a deuterated substance, or a pharmaceutically acceptable salt thereof, or a pharmaceutical composition containing same, as well as the use thereof as a BRAF regulator in the preparation of a drug for treating related diseases. Each group in formula (I) is as defined in the description.

METHODS OF MODIFYING NEURONS IN VIVO TO TREAT AND/OR PREVENT AMYOTROPHIC LATERAL SCLEROSIS (ALS)

Absstract of: US20260284230A1

0000 Provided herein are compositions and methods of treating and/or preventing amyotrophic lateral sclerosis (ALS) in a subject in need thereof, the methods comprising administering an agent wherein the agent modifies neurons via changes in Kcnn1 protein expression or activity, which in turn modifies neuron firing and/or increases the clearance or protection from toxicity of an ALS-causing protein.

Use of CFTR Modulators For Treating Cerebrovascular Conditions

Absstract of: US20260284023A1

Methods of use of CFTR corrector compounds, particularly lumacaftor and tezacaftor, to treat reduced cerebral perfusion associated with heart failure, sudden sensoneurinal hearing loss, vascular dementia, arterial hypertension, ischemic stroke, hemorrhagic stroke, heart disease, diabetes and Alzheimer's disease by treatment with an amount sufficient to provide a proteostatic effect on cystic fibrosis transmembrane conductance regulator (CFTR) protein expression in cerebral artery smooth muscle cells and an increase in cerebrovascular perfusion in the patient.

ANTI-TDP-43 BINDING MOLECULES AND USES THEREOF

Absstract of: US20260285956A1

0000 The present invention is in the field of transactive response DNA binding protein with a molecular weight of 43 kDa (TARDB or also TDP-43). The invention relates to TDP-43 specific binding molecules, in particular to anti-TDP-43 antibodies or antigen-binding fragment or a derivative thereof and uses thereof. The present invention provides means and methods to diagnose, prevent, alleviate and/or treat a disease, disorder and/or abnormality associated with TDP-43 aggregates including but not limited to Frontotemporal dementia (FTD), amyotrophic lateral sclerosis (ALS), Alzheimer's disease (AD), Parkinson's disease (PD), Chronic Traumatic Encephalopathy (CTE), and limbic-predominant age-related TDP-43 encephalopathy (LATE).

Mivelsiran Compositions and Methods of Use Thereof

Nº publicación: US20260284097A1 24/09/2026

Applicant:

ALNYLAM PHARMACEUTICALS INC [US]
ALNYLAM PHARMACEUTICALS, INC.

Absstract of: US20260284097A1

0000 The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting the APP gene, as well as methods of inhibiting expression of an APP gene and methods of treating subjects having an APP-associated disease or disorder, such as Alzheimer's disease (e.g., early onset Alzheimer's disease), using such dsRNAi agents and compositions.