Alerta

FORMULATION AND METHOD FOR TREATMENT OF ALZHEIMER'S DISEASE, STROKE AND DIABETIC RETINOPATHY

Resumen de: US2025345312A1

The invention discloses formulation for use in the treatment of diseases such as Alzheimer's Disease, Stroke, and Diabetic Retinopathy as well as a method for treating said diseases by administering said formulation. The formulation comprises a therapeutically effective amount of (1-H indazole-4yl-)methanol, ethanol, and Miglyol812N at a ratio of 1 drug, to 10 Ethanol to 90 Miglyol. The formulation helps in improving retention, bioavailability, and blood-brain barrier penetration of (1-H indazole-4yl-)methanol and was effective in suppressing inflammation by blocking hGMF-β phosphorylation and activity.

Methods for the administration of certain VMAT2 inhibitors

Resumen de: AU2025256123A1

Provided is a method of administering a vesicular monoamine transport 2 (VMAT2) inhibitor to a patient in need thereof, wherein the patient experiences one or more clinically significant parkinson-like signs or symptoms. Provided is a method of administering a vesicular monoamine transport 2 (VMAT2) inhibitor to a patient in need thereof, wherein the patient experiences one or more clinically significant parkinson-like signs or symptoms. ct c t

TREATMENT OF NEURONAL DISEASES

Resumen de: US2025345365A1

Methods and compositions for treating certain neurodegenerative diseases are provided. The method uses in vivo conversion of gilal cells to neurons by PTB and optionally nPTB knock down via CRISPR/Cas delivered by viral vectors (e.g., AAV vector). Examples of the neurodegenerative diseases include RGC loss-related degenerative disease and Parkinson's Disease,

POLYPEPTIDE AND USE THEREOF IN TREATMENT OF NERVOUS SYSTEM DISEASES

Resumen de: WO2025231617A1

Provided is a polypeptide which contains a polypeptide having at least 80%, 85%, 90%, 95%, or 99% identity to amino acid sequence MTYRPGYNPFG (SEQ ID NO: 41) or amino acid sequence AKGYYRPYGVPV (SEQ ID NO: 22), or a polypeptide having one or more amino acid substitutions, deletions and/or additions relative to the amino acid sequence as shown in SEQ ID NO: 41 or 22. The provided polypeptide can interfere with the binding of a Brag2 synaptic protein to the C terminus of an AMPA receptor, thereby inhibiting NMDA-mediated excessive endocytosis of an AMPA receptor and neuronal apoptosis, and playing a role in protecting neuronal cell activity. Therefore, the polypeptide has application prospects and potential development values as a drug for treating nervous system diseases such as strokes, depression, Alzheimer's disease and drug addiction.

METHODS OF TREATMENT AND DIAGNOSIS OF PARKINSON'S DISEASE ASSOCIATED WITH WILD-TYPE LRRK2

Resumen de: AU2024269632A1

The invention provides methods of treating patients with Parkinson's disease (PD) associated with wild-type LRRK2. The invention recognizes that analysis of genetic modifiers of LRRK2 in such patients allows identification of those patients who will respond to LRRK2 inhibitors. Thus, the invention provides methods of identifying PD patients who will respond to LRRK2 inhibitors and methods of treating such patients.

NOVEL KAPPA OPIOID LIGANDS

Resumen de: US2025346584A1

The invention provides novel ligands of Kappa (κ) opioid receptors, such as can be used to modulate a Kappa opioid receptor. Methods of synthesis and methods of use are also provided. Compounds of the invention can be used therapeutically in the treatment of dissociative disorders or pain, or to provide neuroprotection, or to induce diuresis, or to modulate the immune system, or for treatment of one or more of an affective disorders comprising depression or stress/anxiety; an addictive disorder; alcoholism, epilepsy; a cognition deficiency; schizophrenia; Alzheimer's disease; or pain.

LOW DOSE HUMAN INTERLEUKIN-2 FOR THE TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS

Resumen de: EP4647123A2

The present invention is in the field of amyotrophic lateral sclerosis (ALS) and relates to human interleukin-2 (IL-2) for use in the treatment of amyotrophic lateral sclerosis in a human subject, wherein each dose of human IL-2 administered to said subject is between 0.1 x10⁶ to 3x10⁶ international units (IU). Human IL-2 is preferably administered in cycles of 3 to 7 days of once-daily sub-cutaneous injection of 0.1 x10⁶ to 3x10⁶ IU human IL-2. The treatment does not comprise the administration of regulatory T cells to the subject, who is preferably also under riluzole treatment. The administered human IL-2 is preferably not complexed with anti-hIL-2 antibodies and the treatment also preferably does not comprise the administration of rapamycin or any other suppressive agent of effector T cells (Teffs) to the subject. The treatment permits to decrease plasma CCL2 concentration and to change the polarization of blood macrophages from an M1 inflammatory phenotype to an anti-inflammatory M2 phenotype involved in tissue repair.

A CYNANCHUM AURICULATUM EXOSOME, ITS EXTRACTION METHOD, AND APPLICATION

Resumen de: NL4000209A

The present invention discloses a Polygonum multiflorum exosome and its extraction method and application. The specific extraction method of the Polygonum multifiorum exosome comprises the following steps: removing the rootlets of fresh Polygonum multifiorum fruits, cutting them into pieces, juicing, collecting the filtrate, subjecting the filtrate to continuous centrifugation and differential centrifugation to obtain the supernatant, continuing to perform ultrahigh-speed centrifugation on the supernatant to collect the precipitate, and resuspending the precipitate, which is the Polygonum multifiorum exosome extract. The Polygonum multiflorum exosome obtained by the present invention is applied in the preparation of drugs for the prevention and treatment of Alzheimer’s disease. By using 3XTgAD transgenic homozygous mice, the extracted Polygonum multiflorum exosome is injected into AD mice via tail vein injection, and small animal behavioral methods, such as open field test, novel object recognition, and water maze, are employed to observe changes in the cognitive behavior of the mice, thereby verifying the improvement of cognitive impairment in AD mice by the Polygonum multiflorum exosome.

PHARMACEUTICAL COMBINATION OF DONEPEZIL AND CEVIMELINE

Resumen de: WO2025229537A1

The present invention relates to combination of donepezil or a pharmaceutically acceptable salt thereof and cevimeline or a pharmaceutically acceptable salt thereof for use in the treatment of Alzheimer ́s disease. Furthermore, a pharmaceutical formulation containing donepezil or a pharmaceutically acceptable salt thereof, cevimeline or a pharmaceutically acceptable salt thereof, and at least one pharmaceutically acceptable excipient is provided.

CRYSTALLINE FORM OF A PYRIDAZINE NLRP3 INHIBITOR

Resumen de: WO2025229146A1

The present disclosure relates to a compound of formula (I) which is in crystalline Form 1, characterized by having a powder X-ray diffractogram displaying peaks expressed as degree 2-Theta angles at about 3.4, 6.8, 10.3, 13.7, and 20.5. The present disclosure also relates to processes for its preparation, as well as a medicament and a pharmaceutical composition comprising it. The present disclosure further concerns the crystalline Form 1 of compound of formula (I) for use as a medicine and more particularly in the prevention and/or in the treatment of Parkinson's disease, frontotemporal dementia, multiple system atrophy, Alzheimer's disease, multiple sclerosis, amyotrophic lateral sclerosis, or brain injury.

PHOSPHO-TAU AGGREGATION-BASED BIOMARKERS FOR ALZHEIMER'S DISEASE DIAGNOSIS, DIFFERENTIATION, AND TREATMENT

Resumen de: WO2025231348A1

Provided are methods of phospho-tau aggregation-based biomarker discovery, and new utilities for discovered biomarkers in Alzheimer's disease (AD) diagnosis, differentiation, treatment, and identification of the presence of pretangles in a subject. Novel p-tau sites, p-tau198, p-tauS356, p-tau396, and p-tau422, identified through such methods showed comparable or superior characteristics with established p-tau biomarkers, and identified biomarkers were capable of differentiating AD or mild cognitive impairment (MCI) from cognitively normal controls.

METHODS FOR PREVENTING, TREATING, AND/OR DIAGNOSING NEURODEGENERATIVE DISEASES

Resumen de: WO2025229644A1

The present invention relates to pharmaceutical compositions and diagnostic methods for neurodegenerative diseases, particularly Parkinson's disease. The invention includes compositions comprising anti-Clostridium tetani agents such as tetanus vaccines, anti-tetanus immunoglobulins, and antibiotics. Methods for diagnosis and monitoring Parkinson's disease comprising detecting C. tetani-derived nucleic acids, proteins, or analogues thereof, in biological specimens, are further provided.

OLIGONUCLEOTIDE TARGETING AMYLOID PRECURSOR PROTEIN GENE AND USE THEREOF

Resumen de: WO2025228429A1

An RNAi agent targeting an amyloid precursor protein, such as a double-stranded small interfering RNA (siRNA) agent. A method for inhibiting the expression of the APP gene by using the RNAi agent and a method for preventing and treating APP-related diseases, such as cerebral amyloid angiopathy (CAA) or Alzheimer's disease (AD), including early-onset familial Alzheimer's disease (EOFAD). The siRNA significantly inhibits the expression level of the APP gene and has a long-lasting drug effect.

4-AMINOPYRROLO2,1-F1,2,4TRIAZINES AND PREPARATION AND USES THEREOF

Resumen de: US2025340575A1

4-Aminopyrrolo2,1-f1,2,4triazine compounds for treating various diseases and pathologies are disclosed. More particularly, the present disclosure concerns the use of 4-aminopyrrolo2,1-f1,2,4triazine compounds or analogs thereof, in the treatment of disorders characterized by overexpression of DYRK1A (e.g., cancer, Down syndrome, Alzheimer's disease, diabetes, and osteoarthritis).

IMIDAZOLONE DERIVATIVES AS INHIBITORS OF PROTEIN KINASES IN PARTICULAR DYRK1A, CLK1 AND/OR CLK4

Resumen de: US2025340538A1

The present invention relates to a compound of formula (I) wherein A, B, C, D and E are selected from the group consisting of ═CH— and —N═, R2 is selected from a hydrogen atom, a (C1-C4)alkyl group and a (C3-C6)cycloalkyl group, R1 represents a (C4-C6)alkyl group, a (C3-C8)cycloalkyl group, a bridged (C6-C10)cycloalkyl group, a fused phenyl group, a substituted phenyl group, a R′-L- group, wherein L is either a single bond or a (C1-C3)alkanediyl group, and R′ represents, a (C3-C8)heterocycloalkyl group, or a (C3-C8)heteroaryl group, or a R′-L- group wherein L is a (C1-C3)alkanediyl group, and R′ is a an optionally substituted phenyl group or any of its pharmaceutically acceptable salt. The present invention further relates to a composition comprising a compound of formula (I) and a process for manufacturing said compound as well as its synthesis intermediates. It also relates to said compound for use as a medicament, in particular in the treatment and/or prevention of cognitive deficits and neuroinflammation associated with Down syndrome, Alzheimer's disease, dementia and/or tauopathies; Parkinson's disease; CDKL5 Deficiency Disorder; Phelan-McDermid syndrome; autism; type 1 and type 2 diabetes; abnormal folate and methionine metabolism; tendinopathy and osteoarthritis; Duchenne muscular dystrophy; several cancers; neuroinflammation, anemia, infections and for regulating body temperature.

MIVELSIRAN COMPOSITIONS AND METHODS OF USE THEREOF

Resumen de: AU2024260719A1

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.

INTRA-STRIATAL CO-TRANSPLANTATION OF AUTOLOGOUS TREG AND MDA CELLS IN PARKINSON'S DISEASE CELL THERAPY

Resumen de: WO2024233788A2

Described herein, inter alia, are compositions and methods of use (e.g., treating Parkinson's Disease and/or reducing the immune response due to needle trauma during cell transplantation) for administering a population of regulatory T (TREG) cells and/or a population of midbrain dopamine (mDA) cells to the brain of a subject.

IMIDAZOLONE DERIVATIVES AS INHIBITORS OF PROTEIN KINASES IN PARTICULAR DYRK1A, CLK1, AND/OR CLK4

Resumen de: US2025340539A1

The present invention relates to a compound of formula (I) wherein R1 represents a (C3-C8)cycloalkyl group, a bridged (C6-C10)cycloalkyl group, a fused phenyl group, a substituted phenyl group, a R′-L- group, wherein L is either a single bond or a (C1-C3)alkanediyl group, and R′ represents, a (C3-C8)heterocycloalkyl group, or a (C3-C8)heteroaryl u group, or a R″-L- group wherein L is a (C1-C3)alkanediyl group, and R″ is an optionally substituted phenyl group; R2 is selected from the group consisting of a hydrogen atom and a (C1-C3)alkyl group; R5 represents a hydrogen atom, a (C1-C4)alkyl group or a (C3-C6)cycloalkyl group or any of its pharmaceutically acceptable salt. The present invention further relates to a composition comprising a compound of formula (I) and a process for manufacturing said compound as well as its synthesis intermediates. It also relates to said compound for use as a medicament, in particular in the treatment and/or prevention of cognitive deficits and neuroinflammation associated with Down syndrome; Alzheimer's disease; dementia; tauopathies; Parkinson's disease; CDKL5 Deficiency Disorder; Phelan-McDermid syndrome; autism; type 1 and type 2 diabetes; abnormal folate and methionine metabolism; osteoarthritis and tendinopathy; Duchenne muscular dystrophy; cancers and leukemias; neuroinflammation, anemia, infections caused by unicellular parasites, viral infections and for regulating body temperature.

COMPOUNDS AND METHODS FOR REDUCING TAU EXPRESSION

Resumen de: US2025340872A1

Provided are RNAi agents, methods, and pharmaceutical compositions for reducing the amount or activity of tau RNA in a cell or animal, and in certain instances reducing the amount of tau protein in a cell or animal. Such RNAi agents, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease, including a tauopathy, Alzheimer's disease, fronto-temporal dementia (FTD), FTDP-17, progressive supranuclear palsy (PSP), chronic traumatic encephalopathy (CTE), corticobasal ganglionic degeneration (CBD), epilepsy, or Dravet's Syndrome.

METHOD AND COMPOSITION FOR TREATING NEURODEGENERATIVE DISORDER

Resumen de: US2025340593A1

Inventors have shown evidence of VEGF accumulation in extracellular Aβ plaques in the post-mortem brain of patients with Alzheimer's disease (AD) and of the APP/PS1 mouse model of AD. They identified specific binding domains involved in the direct interaction between A0o and VEGF and engineered a peptide that blocks this interaction. The designed peptide binds to Aβ oligomers with high affinity and inhibits the process of Aβ self-aggregation, leading to the blockade of fibrillar aggregation. Furthermore, the peptide prevents soluble Aβ-derived toxins to target synapses in hippocampal neuron cultures and restores long-term potentiation in the hippocampus of the APP/PS1 mouse model of Alzheimer's disease. Thus, these findings have broad implications for preventing and treating diseases with Aβ neurotoxicity such as Alzheimer's disease. Accordingly, the invention relates to a peptide comprising the amino acid sequence KRKKSRYKSWSVYVG (SEQ ID NO: 1).

LOW-DOSE DOPAMINERGIC DRUGS TO DELAY THE PROGRESSION OF SPINOCEREBELLAR ATAXIA TYPE 3 AND ALZHEIMER'S DISEASE

Resumen de: WO2025230432A1

The present invention refers to dopaminergic drugs such as dopamine precursors and aromatic L-amino acid decarboxylase (AADC) inhibitors for use in a treatment to delay the progression of pathologies in which abnormal amyloid deposits spread to different brain regions, consisting of Spinocerebellar Ataxia Type 3 and Alzheimer's Disease, administered in levodopa-equivalent doses below 300 mg/day. The present invention further refers to pharmaceutical formulations comprising the said dopaminergic drugs. The present invention's compounds pharmaceutical formulations and uses may be advantageously employed in a treatment to prevent further accumulation of amyloid deposits in neurons, and minimize adverse effects associated with the prolonged use of dopamine precursors and/or their peripheral degradation.

USE OF SUBSTITUTED 1,4 BENZOQUINONES TO TREAT ALPHA-SYNUCLEINOPATHIES

Resumen de: WO2025231245A1

The present disclosure provides methods, compounds, compositions, formulations, or medicaments for treating, preventing, inhibiting, ameliorating, or delaying the onset of a- synucleinopathies (e.g., Parkinson's disease (PD), PD with dementia (PDD), dementia with Lewy bodies (LBD), or Multiple System Atrophy (MSA)) as well as methods for ameliorating, inhibiting, or delaying the onset of signs or symptoms of an a-synucleinopathy in a subject. The disclosed methods, compounds, compositions, formulations, or medicaments are also useful for addressing the related signs and symptoms of a- synucleinopathies. The methods comprise administering to the subject the compounds, mixtures of compounds, or compositions, formulations, or medicaments derived from said compounds or mixtures thereof to thereby produce the aforementioned therapeutically beneficial effect(s).

4-AMINOPYRROLO2,L-FL,2,4TRIAZINES AND PREPARATION AND USES THEREOF

Resumen de: WO2025231425A1

4-Aminopyrrolo2,l-fl,2,4triazine compounds for treating various diseases and pathologies are disclosed. More particularly, the present disclosure concerns the use of 4-aminopyrrolo2,l-fl,2,4triazine compounds or analogs thereof, in the treatment of disorders characterized by overexpression of DYRK1A (e.g., cancer, Down syndrome, Alzheimer's disease, diabetes, and osteoarthritis).

COMPOSITIONS AND METHODS FOR TREATING PARKINSON'S DISEASE

Resumen de: CN120435551A

Provided herein are compounds, compositions, uses, and methods for increasing cell viability of dopaminergic neurons, or for preventing or treating dopaminergic neuronal death. In certain examples, methods are provided for reducing symptoms and/or for preventing or treating Parkinson's disease in an individual in need thereof, which can include the step of treatment with a GDP-binding form of Rab1a (Rab1aGDP), one or more manifestation nucleic acids encoding Rab1aGDP, or a combination thereof.

PRIDOPIDINE FOR TREATING JUVENILE HUNTINGTON'S DISEASE

Nº publicación: EP4642457A1 05/11/2025

Solicitante:

PRILENIA NEUROTHERAPEUTICS LTD [IL]
Prilenia Neurotherapeutics Ltd

Resumen de: CN120569199A

Provided herein is a method of treating teenager Huntington's disease in a subject in need thereof, the method comprising orally administering a pharmaceutical composition comprising pridopidine and/or an analog thereof or a pharmaceutically acceptable salt thereof.