Resumen de: WO2026064802A1
Described herein is a gene therapy platform to restore GABAergic inhibition and improve cognition in neurodevelopmental disorders, epilepsies, sensory disorders, aging, and Alzheimer's disease, by increasing expression of the transcription factor Meis2 in parvalbumin-expressing inhibitory neurons (PV INs).
Resumen de: WO2026064542A1
The present disclosure describes crystalline forms of 2-(tert-butoxy)-4-(3-methyl-3-(5-(methylsulfonyl)isoindolin-2-yl)butyl)phenol fumarate salts and pharmaceutical compositions of same. Also described are methods of using the crystalline forms treating Alzheimer's Disease, Dementia with Lewy Bodies and Dry age-related macular degeneration in a subject in need thereof, comprising administering the crystalline form to the subject. Methods of making the solid forms are also described.
Resumen de: US20260083723A1
A therapeutic or preventive agent for amyotrophic lateral sclerosis has inhibitory action against ferroptosis. The therapeutic or preventive agent for amyotrophic lateral sclerosis contains a tetrahydroquinoline derivative or a pharmaceutically acceptable salt thereof as an active ingredient. Compositions for and methods of treating amyotrophic lateral sclerosis and inhibiting ferroptosis are also disclosed.
Resumen de: AU2023463541A1
The present invention relates to use of a novel compound for preventing, alleviating or treating Alzheimer's disease, and the novel compound exhibits an inhibitory effect on tau protein aggregation. In addition, it was identified that presenilin 1 was reduced by treatment using the novel compound. Therefore, the novel compound can be effectively used in the development of a therapeutic agent for Alzheimer's disease.
Resumen de: WO2026062257A1
New gelatinase inhibitors, processes for their preparation, pharmaceutical compositions comprising them, and their use in therapy and/or prophylaxis of conditions wherein inhibition of gelatinases is useful such as epilepsy, schizophrenia, Alzheimer disease, autism (in particular associated to fragile X syndrome), mental retardation, mood disorders such as bipolar disorders, depression, vascular diseases such as ischemic stroke and atherosclerosis, inflammatory diseases such as multiple sclerosis and rheumatoid arthritis, drug addiction, neuropathic pain, lung diseases such as asthma and chronic obstructive pulmonary disease, cancer and sepsis.
Resumen de: WO2026064441A1
The disclosure is related to methods of treating a subject with Alzheimer's Disease (AD) using anti-amyloid beta (Aβ) therapy. The patient may be treated based on the measurement and analysis of biomarker levels, such as plasma Aβ42/40 ratio, pTau181 and pTau217. The disclosure includes a systematic approach called CP Convert for converting biomarker measurements across different analytical platforms, enabling comparison of pTau217 data from LC-MS/MS, SIMOA, and chemiluminescent enzyme immunoassay platforms. Methods demonstrate that pTau217 has superior diagnostic accuracy compared to Aβ42/40 ratio and pTau181 for detecting brain amyloid positivity. Implementation of pTau217 prescreening can significantly reduce Aβ-PET testing, decreasing patient burden and clinical trial costs. The CP Convert methodology can be validated using independent cohorts, demonstrating robust cross-platform conversion for research applications.
Resumen de: US20260083740A1
The present disclosure relates to methods for treating a patient diagnosed with Parkinson's Disease (PD) by administering an escalating dose of tavapadon and monitoring efficacy of PD treatment.
Resumen de: US20260085036A1
The present invention relates to compounds according to formula (I), which are prodrugs of the psychoactive compound phenethylamine or its derivatives. The prodrugs provided herein exhibit improved pharmacokinetic properties during uptake as compared to phenethylamine (or the respective phenethylamine derivative), as well as reduced side effects resulting from the metabolites thus formed. Due to the affinity of the active phenethylamine compound, inter alia, for the 5-HT2a-receptor, these prodrugs are particularly advantageous for use in therapy, e.g., in the treatment of depression, posttraumatic stress disorder (PTSD), Alzheimer's disease or dementia.
Resumen de: US20260083826A1
The present invention aims to provide a novel agent for treating Alzheimer's disease, a method for treating Alzheimer's disease, a method for screening for a candidate substance for a therapeutic drug for Alzheimer's disease, and the like. The present invention is a prophylactic and/or therapeutic agent for Alzheimer's disease comprising a peptide corresponding to dynamin 1. The peptide preferably corresponds to dynamin 1-pleckstrin-homology domain or dynamin 1-proline rich domain. In addition, the peptide is preferably encapsulated in nano-particles or linked to a peptide sequence that improve delivery of the peptide into the brain.
Resumen de: WO2026064401A1
The disclosure provides methods of treating neurodegenerative conditions, for example, amyotrophic lateral sclerosis, using a regimen of a chlorite salt, for example, sodium chlorite. The long-term survival of subjects receiving sodium chlorite and placebo are compared.
Resumen de: WO2026064194A1
Provided are methods for administering allogeneic induced pluripotent stem cells (iPSC)-derived midbrain dopaminergic progenitors to a subject for treatment of Parkinson's disease (PD). The midbrain dopaminergic progenitor cells are administered to 5 trajectories, each with 8 depositional sites, per putamen. Patients are operated on using a trans-frontal approach with an entry point near the coronal suture. Also provided are compositions and articles of manufacture for use in the methods.
Resumen de: US20260085066A1
The present disclosure relates to bifunctional compounds that cause the degradation of LRRK2; pharmaceutical compositions comprising the compounds; and methods of treating disorders associated with LRRK2, including Parkinson's Disease.
Resumen de: EP4714940A1
New gelatinase inhibitors, processes for their preparation, pharmaceutical compositions comprising them, and their use in therapy and/or prophylaxis of conditions wherein inhibition of gelatinases is useful such as epilepsy, schizophrenia, Alzheimer disease, autism (in particular associated to fragile X syndrome), mental retardation, mood disorders such as bipolar disorders, depression, vascular diseases such as ischemic stroke and atherosclerosis, inflammatory diseases such as multiple sclerosis and rheumatoid arthritis, drug addiction, neuropathic pain, lung diseases such as asthma and chronic obstructive pulmonary disease, cancer and sepsis.
Resumen de: MX2025013613A
Disclosed are methods of treating amyotrophic lateral sclerosis (ALS). Also disclosed are methods of treating C9orf72 amyotrophic lateral sclerosis (C9-ALS).
Resumen de: WO2026057822A1
Microglial spatial heterogeneity remains a crucial yet poorly studied question in light of potential cell-directed therapies for Alzheimer`s disease (AD). Little is known about the dynamics of spatially distinct microglia states, which are either adjacent or non-associated with the plaque site, and their selective contributions to neurodegeneration in vivo. So far, research has essentially focused on pathology-associated microglia. Here, we combined novel multicolor fluorescence fate mapping, single-cell transcriptional analysis, epigenetic profiling, advanced immunohistochemistry and computational modelling to comprehensively characterize the relation of plaque-associated and non-plaque- associated microglia during neurodegeneration. This approach enabled us to identify and characterize non-plaque-associated microglia as a unique and highly dynamic microglial state in a mouse model of AD. Non-plaque-associated microglia modulate network expansion, quickly adapt to environmental cues and their transition to plaque-associated microglia can be specifically modulated during disease, contrary to their reputation as a passive bystander subpopulation. This description of the dynamics of spatially segregated microglial states and their distinct molecular features may therefore open promising new avenues for state-specific therapeutic interventions during neurodegeneration.
Resumen de: WO2026057634A1
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 3.3, 10.0, 20.1, 21.7, and 25.0. 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.
Resumen de: US20260076927A1
The present invention features palatable pharmaceutical compositions including sodium phenylbutyrate and methods for the treatment of inborn errors of metabolism (e.g., Maple Syrup Urine Disease or Urea Cycle Disorders), neurodegenerative disorders such as Parkinson's disease, spinal muscular atrophy, dystonia, or inclusion-body myositis with such compositions.
Resumen de: US20260076931A1
The present invention generally provides methods and compositions for the treatment of Parkinson's disease and depression and/or anxiety. The invention relates to recombinant microorganisms, particularly gut-colonizing probiotics, modified to produce L-DOPA.
Resumen de: US20260076939A1
The present invention relates to: a method for preparing a chiral gamma lactam derivative or a pharmaceutically acceptable salt thereof by using a chiral organocatalytic compound; and a composition for preventing, alleviating or treating muscle diseases, mental diseases, or neurodegenerative diseases, comprising the derivative or the pharmaceutically acceptable salt thereof. The chiral gamma lactam derivative or the pharmaceutically acceptable salt thereof, of the present invention, has an effect of inhibiting MAO-B and MSTN, targets D1-mClu5, and can be used in the prevention, alleviation, or treatment of muscle diseases including sarcopenia, mental diseases including depression, neurodegenerative diseases including Parkinson's disease, and the like.
Resumen de: WO2026055750A1
The present disclosure relates to the field of oligonucleotides. More particularly, the present disclosure relates to antisense oligonucleotides for the specific binding and inhibition of translation of Chitinase (CHIT1) encoding mRNA. Additionally, this disclosure relates to methods of using such oligonucleotides in preventing or treating diseases, disorders or conditions associated with CHIT1, such as neurodegenerative or neuroinflammatory diseases, disorders or conditions like ALS.
Resumen de: WO2026056131A1
The use of a TRPM7 activator in preparing a therapeutic product for treating Alzheimer's disease is provided. The TRPM7 activator can be a regulator of an extracellular divalent cation or a metal salt containing a divalent cation. The TRPM7 activator can retain memory and cognitive functions, protect synaptic density and reduce Aβ plaque accumulation in a mouse model of Alzheimer's disease, and the morphology and organ weight analysis of heart, liver and pancreas show that the administration of the TRPM7 activator is safe. In addition, a TRPM7 gene or a protein encoded by the TRPM7 gene can serve as a therapeutic target for treating Alzheimer's disease. A high throughput method for screening potential drug targets for treating Alzheimer's disease by screening TRPM7 activators via TRPM7 proteins broadens idea and provides a new direction for discovery and development of new agents for treating Alzheimer's disease.
Resumen de: WO2026056971A1
Provided in the present invention is an anti-prothrombin/phosphatidylserine antibody, comprising a heavy chain variable region and a light chain variable region; the heavy chain variable region comprises HCDR1 to HCDR3 having amino acid sequences as shown in SEQ ID NOs: 1-3, respectively, and the light chain variable region comprises LCDR1 to LCDR3 having amino acid sequences as shown in SEQ ID NOs: 4-6, respectively. The anti-prothrombin/phosphatidylserine antibody of the present invention can bind to a prothrombin/phosphatidylserine complex with high affinity, thereby targeting and binding to phosphatidylserine, and has a potential therapeutic effect with respect to tumors, infections, atherosclerosis, ischemia-reperfusion injury, inflammatory bowel disease, Alzheimer's disease, Parkinson's disease, etc.
Resumen de: WO2026059890A1
The present invention is directed to a pharmaceutical composition and method for treating a subj ect diagnosed amyotrophic lateral sclerosis (ALS) with a pharmaceutical composition containing dissolved or dispersed therein a SOD1 and/or TDP-43 aggregation- inhibiting amount of a rose bengal (RB) compound that is a pharmaceutically acceptable salt of RB, RB lactone, a RB amide, an aromatic RB derivative, wherein the aromatic derivative is an ester or amide formed from an alcohol or monosubstituted amine having a 5 - or 6 -membered aromatic ring, or a 5, 6 - or 6, 6 - fused aromatic ring system that contains 0, 1, or 2 hetero ring atoms that are independently nitrogen, oxygen or sulfur. This treatment method is typically repeated a plurality of times or until the subj ect no longer needs it.
Resumen de: CN121285374A
The present invention provides a method of treating a patient suffering from Parkinson's disease (PD) associated with wild type LRRK2, and a method of treating a patient suffering from Parkinson's disease (PD) associated with wild type LRRK2. The present invention recognizes that the analysis of genetic modification factors of LRRK2 in such patients allows for the identification of patients who will respond to LRRK2 inhibitors. Accordingly, the invention provides methods of identifying PD patients who will respond to LRRK2 inhibitors and methods of treating such patients.
Nº publicación: EP4709398A2 18/03/2026
Solicitante:
MCLEAN HOSPITAL CORP [US]
The McLean Hospital Corporation
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.