Resumen de: WO2025056018A1
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.
Resumen de: US2025092064A1
The present invention aims to provide a medicament capable of treating and/or preventing diseases associated with oxidative stress by inhibiting the protein-protein interaction between Keap1 and Nrf2 and activating Nrf2. The present invention relates to a compound represented by the following formula (1):wherein each symbol is as described in the DESCRIPTION, or a pharmaceutically acceptable salt thereof. In addition, the present invention also relates to a medicament containing the aforementioned compound, for the prophylaxis and/or treatment of diseases involving oxidative stress selected from the group consisting of chronic kidney disease, non-alcoholic steatohepatitis, chronic obstructive pulmonary disease, radiation skin disorder, radiation mucosal disorder, cardiac failure, pulmonary arterial hypertension, Parkinson's disease, Friedreich's ataxia, multiple sclerosis, age-related macular degeneration, retinitis pigmentosa and glaucoma.
Resumen de: US2025090505A1
Provided herein are methods for preventing biomineralization in brain tissue or neuronal tissue in vivo or in vitro by inhibiting the deposition of hydroxyapatite therein. Also provided are methods for delaying the progression or onset of Alzheimer's disease in a subject and for preventing the development of Alzheimer's disease in a post-traumatic brain injury subject. Contacting the brain tissue or neuronal tissue with or administering to a subject the drugs Levamisole and/or Foscarnet and/or a bisphosphonate inhibits or decreases levels of alkaline phosphatase and calcium and counteracts alpha-glycerophosphate therein to inhibit the activation of spontaneous biomineralization within a brain tissue to decrease or prevent ectopic biomineralization.
Resumen de: WO2025056518A1
The present invention relates to the field of medicinal chemistry and more specifically to 5,6- disubstituted 1H- and 2H- indazoles as pharmaceutically active compounds. The compounds of the present invention are dual inhibitors of butyrylcholinesterase and p38α mitogen-activated protein kinase, and thus are particularly useful for the treatment of various diseases which may be therapeutically modified by altering the activity of butyrylcholinesterase (BChE) and/or p38α MAP kinase such as dementias including Alzheimer's disease (AD) and other conditions involving chronic inflammation.
Resumen de: WO2025058089A1
The purpose of the present invention is to provide an agent for suppressing amyloid β aggregation/deposition and an agent for suppressing/improving cognitive decline of a subject both of which are expected to have the effect of treating and/or preventing Alzheimer's disease. Specifically, the present invention pertains to an agent for suppressing amyloid β aggregation/deposition or an agent for suppressing/improving cognitive decline of a subject, the agent comprising an extract of a plant belonging to Myrtaceae.
Resumen de: WO2025058473A1
The present invention relates to: a pharmaceutical composition for selectively preventing or treating neurodegenerative diseases including Parkinson's disease, comprising as an active ingredient exosomes into which has been delivered a degenerative brain disease-therapeutic protein via a photocleavable protein; and a method for preparing same. Parkinson's disease therapeutic proteins, such as Parkin protein, effectively inhibit the death of damaged dopaminergic neurons, and have protective effects against neurotoxic substances, and thus have an effect of preventing damage to neurons.
Resumen de: US2025090571A1
Provided herein are methods and compositions to enhance or activate protective microglial activities (such as phagocytosis of neurotoxic material) by activating CARD9.
Resumen de: AU2025201530A1
The present disclosure is in the field of modulation of genes involved in rare diseases including for diagnostics and therapeutics for rare diseases such as Angelman's Syndrome, 5 Facioscapulohumeral Muscular Dystrophy (FHMD), Amyotrophic Lateral Sclerosis (ALS), Frontotemporal dementia (FTD) and Spinal Muscular Atrophy (SMA).
Resumen de: AU2023348297A1
The present invention relates to compounds which are suitable for imaging TDP-43 (Transactive response (TAR) DNA binding protein 43 kDa) aggregates. The compounds can be used, for example, for diagnosing a disease, disorder or abnormality associated with TDP-43 aggregates or a TDP-43 proteinopathy, such as amyotrophic lateral sclerosis (ALS), Alzheimer's disease (AD), Frontotemporal dementia (FTD) and limbic-predominant age-related TDP-43 encephalopathy (LATE).
Resumen de: AU2025201237A1
METHODS FOR TREATING ALZHEIMER'S DISEASE A method for treatment of a human patient for Alzheimer's disease (AD) comprises sequentially administering multiple doses of a recombinant, fully human, anti-amyloid beta monoclonal antibody to the patient. In preferred embodiments, the antibody is administered in increasing amounts over a period of time. In preferred embodiments, the susceptibility of the patient to amyloid related imaging abnormalities (ARIA) is thereby reduced.
Resumen de: US2025090541A1
Disclosed are methods for treating or inhibiting development of a disorder selected from the group consisting of a frontotemporal dementia (FTD), Pick's disease, progressive supranuclear palsy, Huntington's disease, Parkinson's disease, corticobasal degeneration, amyotrophic lateral sclerosis (ALS), Lewy body disease, and hippocampal sclerosis (HS), by administering a compound of formula I.
Resumen de: WO2023218187A1
Disclosed are heterobifunctional compounds that can induce the degradation of leucine-rich repeat kinase 2 (LRRK2) and PDE6D. These compounds can engage LRRK2 on one end and bind to an ubiquitin E3 ligase (e.g. cereblon, Von Hippel-Lindau, or Cellular Inhibitor of Apoptosis (cIAP)) on the other end and therefore bring LRRK2 in close proximity to the E3 ligase and induce the ubiquitination and degradation of the LRRK2 protein. Also disclosed are pharmaceutical composition comprising the heterobifunctional compounds and methods of using the compounds to treat LRRK2 and PDE6D- related diseases and disorders.
Resumen de: AU2023269025A1
Disclosed are therapeutic methods for reducing the rate of functionality loss in patients suffering from amyotrophic lateral sclerosis (ALS) during the accelerated stages of this disease.
Resumen de: AU2023267136A1
This application includes methods for reducing axonal tau protein. Those methods include inhibiting the binding between MAPT mRNA and hnRNP R.
Resumen de: KR20250038283A
본 발명은 트롤록스를 유효성분으로 포함하는 파킨슨병의 예방, 개선 또는 치료용 조성물에 관한 것으로, 상기 트롤록스는 PD 마우스를 이용한 행동분석에서 운동기능 장애를 개선하는 효과 있으며, PD 마우스의 선조체 및 SNpc에서 α-시누클레인의 발현량을 감소시키고, 도파민 합성 및 수송을 담당하는 도파민성 신경 세포 마커(TH, VMAT2 및 DAT)의 발현량을 증가시키며, 산화스트레스 관련 단백질 Nrf2 및 HO-1의 발현량을 증가시킬뿐만 아니라, ROS 및 LPO 함량을 감소시키는 효과가 있고, 성상세포 및 신경아교세포의 활성 마커인 GFAP 및 Iba1의 발현량을 감소시키는 효과가 있으며, 신경염증을 유발하는 전염증성 사이토카인(NF-κB, TNF-α 및 IL-1β)을 감소시키는 효과가 있으므로, 파킨슨병의 예방, 개선 또는 치료를 위한 의약품, 건강기능 식품 또는 사료 첨가제로 유용하게 사용할 수 있다.
Resumen de: KR20250037364A
본 발명은 바닐릭산 유도체 화합물에 관한 것으로서, 바닐릭산과 카테콜아민 간의 탈수축합반응에 의해 형성될 수 있다. 본 발명에 따른 바닐릭산 유도체 화합물은 알츠하이머병 및 신경염증 치료제제로 사용할 수 있다는 점에 특징이 있다. 또한 본 발명은 상기 화합물을 포함하는 알츠하이머 치료제 조성물 및 신경염증 치료제 조성물에 관한 것이다.
Resumen de: AU2023316187A1
Provided is a drug for treating Alzheimer's disease, the drug enabling retention of cognitive function amelioration and nerve quality improvement for a specific time even after treatment ends. This drug for causal treatment of Alzheimer's disease (disease-modifying drug) contains hydrogen gas as an active ingredient.
Resumen de: AU2023341169A1
The present disclosure provides compounds of Formula I: (I), or an N- oxide thereof, or a pharmaceutically acceptable salt of the compound or the N-oxide, wherein: A, Y, m, n, p, R1, R2, R3, R3a, R4, R5, R6, R7, and Z are as described herein; processes for the preparation of; intermediates used in the preparation of; and compositions containing such compounds, N-oxides, or salts, and their uses for treating M4-mediated (or M4-associated) disorders including, e.g., Alzheimer's Disease, Parkinson's Disease, schizophrenia (e.g., its cognitive and negative symptoms), pain, addiction, and a sleep disorder.
Resumen de: AU2023347307A1
The disclosure relates to lemborexant, a dual orexin receptor antagonist, and compositions and methods for use in treatment of Alzheimer's disease (AD), e.g., in a subject who has AD or who is at risk for developing AD.
Resumen de: US2025084135A1
Fusion protein Tau-4R can be recombined into Escherichia coli to achieve the fusion expression. It is confirmed by means of experiments that the tau protein prepared by the strain may be used in the treatment research of Alzheimer's disease.
Resumen de: US2025084435A1
The present invention relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and/or canine degenerative myelopathy (DM).
Resumen de: US2025082598A1
The invention concerns a compound of formula (I) R1=H or acyl group, preferably R1=H; R2=halogen atom selected in the group consisting of: F, Cl, Br, I, preferably R2=Cl or a pharmaceutically acceptable isomer, salt and/or solvate thereof, for use in preventing and/or treating circadian rhythm sleep disorders and/or circadian rhythm sleep neurological diseases and/or any medical condition associated due to sleep deprivation.
Resumen de: US2025082686A1
The present disclosure provides adeno-associated vector (AAV)-based methods of increasing gene expression of a GBA1 in floor plate midbrain progenitor cells, determined dopamine (DA) neuron progenitor cells, and/or DA neurons, or glial cells, including those differentiated from pluripotent stem cells, and methods of lineage specific differentiation of the same. Also provided are compositions and uses thereof, such as for treating neurodegenerative diseases and conditions, including Parkinson's disease.
Resumen de: US2025082647A1
The present invention relates in a first aspect to a compound of formula (I) for use in the treatment and/or in prevention of Alzheimer's dementia, wherein the compound of formula (I) is administered in a dosage<20 mg. In a second aspect, the invention relates to a pharmaceutical composition comprising the compound according to the first aspect.
Nº publicación: US2025082762A1 13/03/2025
Solicitante:
KOREA RESEARCH INSTITUTE OF CHEMICAL TECH [KR]
UBIX THERAPEUTICS INC [KR]
KOREA RESEARCH INSTITUTE OF CHEMICAL TECHNOLOGY,
UBIX THERAPEUTICS, INC
Resumen de: US2025082762A1
The present invention relates to a novel heterocyclic compound and a composition, for preventing or treating a cancer, an autoimmune disease, and an inflammatory disease, comprising same. The novel heterocyclic compound of the present invention is a bifunctional compound having a Bruton's tyrosine kinase (BTK) degradation function via a ubiquitin proteasome pathway, and may be utilized as a composition for preventing or treating a cancer, an autoimmune disease, and Parkinson's disease.