21 results about "Neuronal disease" patented technology

The present invention relates to sulphate salts of N-(3-(4-(3-(diisobutylamino)propyl)piperazin-1-yl)propyl)-1H-benzo[d]imidazol-2-amine and pharmaceutically acceptable solvates thereof, for use in the treatment and / or prevention of motor neuron diseases and neuromuscular junction disorders.

Disclosed herein are embodiments of a compound that inhibits c-Abl tyrosine kinase (also referred to herein as “c-Abl”). The compound embodiments described herein are novel c-Abl inhibitors that can bind to c-Abl at an allosteric site and inhibit its activity in various pathways. The compound embodiments also are capable of crossing the blood brain barrier and therefore are useful in inhibiting c-Abl activity as it affects pathways and / or proteins in the brain. The compound embodiments described herein are effective therapeutic agents for treating diseases involving c-Abl, such as cancers, motor neuron diseases, and neurodegenerative diseases. Also disclosed herein are embodiments of methods for making and using the c-Abl inhibitory compound embodiments.

Disclosed are compositions and / or methods of use of the compositions for patients with neuronal diseases such as AD, Parkinson's, Huntington's, multiple sclerosis, and ALS. In certain embodiments flavonoids alone, or in a pharmaceutical preparation, are administered through the nasal olfactory route. In certain embodiments the flavonoid is apigenin and the neural disease is Alzheimer's. In some embodiments a porosome complex is administered for reconstitution into a neural cell. In certain embodiments, a co-administered blood-brain barrier traversing peptide is configured as a mimic of a domain of ATP 1 A3 and / or Tubulin.

Antibodies that specifically bind to TrkB and methods for using the same are described. Neuroprotective agonist antibodies are described, as indicated by their effect on enhancing the survival of retinal ganglion cells in vitro, and these agonist antibodies may be used to treat eye disorders, such as glaucoma. Additionally, other neuronal diseases or disorders, including those characterized by partial neuronal damage, benefit from treatment using these agonist antibodies. In certain embodiments, the present invention comprises antibodies that bind to TrkB and mediate cell signaling. The antibodies of the present invention may be whole human, non-natural antibodies formulated as injectable excipients.

Antibodies that specifically bind to TrkB and methods of use thereof are disclosed. Agonist antibodies having neuroprotective effects, as shown by their ability to enhance retinal ganglion cell survival in vitro, are disclosed, and these agonist antibodies can be used in the treatment of conditions such as eye diseases, including glaucoma. In addition, other neuronal diseases or conditions can also benefit from treatment with these agonist antibodies, including those characterized in part by neuronal damage. In certain embodiments, the present invention includes antibodies that bind to TrkB and mediate cell signaling. The antibodies of the present invention can be fully human, non-naturally occurring antibodies formulated with an excipient for injection.

Presented herein are methods of identifying a subject who has, or is at risk of developing a motor neuron disease, specifically Amyotrophic Lateral Sclerosis (ALS), and / or Primary Lateral Sclerosis (PLS), that includes determining a presence or amount of two or more micro-RNAs (miRNAs) selected from miR-199a-3p, miR-4454, miR-10b-5p, miR-151a-5p, miR-199a-5p, miR-151a-3p, miR-146a-5p, and / or miR-29b-3p in a subject's circulating blood, without determining a presence or amount of the miRNAs from neural-derived exosomes. Also presented herein are methods of preventing, treating, or delaying the onset of a motor neuron disease, specifically ALS and / or PLS.

Disclosed are compositions and / or methods of use of the compositions for patients with neuronal diseases such as AD, Parkinson's, Huntington's, multiple sclerosis, and ALS. In certain embodiments flavonoids alone, or in a pharmaceutical preparation, are administered through the nasal olfactory route. In certain embodiments the flavonoid is apigenin and the neural disease is Alzheimer's. In some embodiments a porosome complex is administered for reconstitution into a neural cell. In certain embodiments, a co-administered blood-brain barrier traversing peptide is configured as a mimic of a domain of ATP1A3 and / or Tubulin.

Disclosed are compositions and / or methods of use of the compositions for patients with neuronal diseases such as AD, Parkinson's, Huntington's, multiple sclerosis, and ALS. In certain embodiments flavonoids alone, or in a pharmaceutical preparation, are administered through the nasal olfactory route. In certain embodiments the flavonoid is apigenin and the neural disease is Alzheimer's. Targeted dosages may affect mitochondrial function in brain neural cells thus ameliorating a neural disease. In some embodiments a porosome complex is administered for reconstitution into a neural cell. In certain embodiments, one or more macrocyclic or linear peptides are administered.

Presented herein are methods of identifying a subject who has, or is at risk of developing a motor neuron disease, specifically Amyotrophic Lateral Sclerosis (ALS), and / or Primary Lateral Sclerosis (PLS), that includes determining a presence or amount of two or more micro-RNAs (miRNAs) selected from miR-199a-3p, miR-4454, miR-10b-5p, miR-151a-5p, miR-199a-5p, miR-151a-3p, miR-146a-5p, and / or miR-29b-3p in a subject's circulating blood, without determining a presence or amount of the miRNAs from neural-derived exosomes. Also presented herein are methods of preventing, treating, or delaying the onset of a motor neuron disease, specifically ALS and / or PLS.

The instant invention provides methods and compositions related to discovery of Free Fatty Acid Receptor 1 (FFA1) as a therapeutic target for treatment or prevention of diseases or disorders of neurons that are characterized by angiogenesis, or of vascular diseases of the eye, retinal degeneration and / or tumors more generally. Therapeutic and / or prophylactic uses and compositions of known FFA1 inhibitors, including small molecules and nucleic acid agents, are described. Methods for identification of novel FFA1 inhibitors are also provided.

Disclosed are compositions and / or methods of use of the compositions for patients with neuronal diseases such as AD, Parkinson's, Huntington's, multiple sclerosis, and ALS. In certain embodiments flavonoids alone, or in a pharmaceutical preparation, are administered through the nasal olfactory route. In certain embodiments the flavonoid is apigenin and the neural disease is Alzheimer's. Targeted dosages may affect mitochondrial function in brain neural cells thus ameliorating a neural disease. In some embodiments a porosome complex is administered for reconstitution into a neural cell. In certain embodiments, one or more macrocyclic or linear peptides are administered.

Disclosed herein are methods for treating a motor neuron disease such as amyotrophic lateral sclerosis (ALS) comprising administering mEphA4-Fc with an interval of greater than every week, for example administering mEphA4-Fc every two weeks, every three weeks, or every four weeks, and at a concentration of about 10 to about 40 mg / kg body weight of a subject.

Disclosed herein are stable peptide antagonists based on the consensus yc-subunit binding site to inhibit the activity of yc-cytokines. Such peptide antagonists are capable of inhibiting the activity of multiple yc-cytokine family members. The yc-family cytokines are associated with important human diseases, such as leukemia, autoimmune diseases, collagen diseases, diabetes mellitus, skin diseases, degenerative neuronal diseases and graft-versus-host disease (GvHD). Thus, inhibitors of yc-cytokine activity are valuable therapeutic and cosmetic agents as well as research tools.

The present application relates to pyrimidopyridone and pteridinone compounds of formula I, processes for their preparation and compositions comprising them. More particularly, the present application relates to compounds of formula I having activity as inhibitors of GCN2 kinase and their use in the treatment of diseases, disorders or conditions treatable by inhibition of GCN2 kinase, such as cancer and neuronal diseases. (I).

The present disclosure provides compositions and methods using C9ORF72-mediated genes and expression products thereof for diagnosis, treatment and prevention of amyotrophic lateral sclerosis, frontotemporal dementia, or both, in carriers of a C9ORF72 hexanucleotide expansion. The present invention also relates to a method of identifying therapeutic agents to treat and diagnose amyotrophic lateral sclerosis, frontotemporal dementia, or both, in carriers of a C9ORF72 hexanucleotide expansion based on C9ORF72-mediated genes.

The present invention discloses compositions, means and kits thereof for treating neuronal clinical indications in a mammalian subject. The composition comprises, inter alia, a synergistic combination of an anti-inflammatory drug and a DICER activator. The present invention further discloses methods for treating neuronal diseases including Motor neuron diseases (MNDs), ALS, FTD (Frontotemporal Dementia), macular degeneration (AMD) autism, and neurodegenerative diseases such as Parkinson's disease and Alzheimer's disease.

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,

Antibodies that specifically bind to TrkB and methods of use thereof are disclosed. The present invention discloses agonist antibodies having neuroprotective effects as shown in their effect of enhancing retinal ganglion cell survival in vitro, and these agonist antibodies are useful in the treatment of such as eye diseases, including glaucoma. In addition, other neuronal diseases or disorders can also be benefited by treatment with these agonist antibodies, including those which are partially characterized by neuronal impairment. In certain embodiments, the invention includes antibodies that bind to TrkB and mediate cellular signaling. The antibodies of the invention may be fully human non-naturally occurring antibodies formulated with excipients for injection.

Methods for preventing or treating brain neuronal diseases are provided. The methods include administering mesenchymal stem cells having increased agouti-related peptide (AgRP) expression or a conditioned medium thereof to a subject in need thereof. In some embodiments, the conditioned medium contains AgRP. The methods may further comprise administering ghrelin. The brain neuronal diseases include Alzheimer's disease, frontotemporal dementia, tauopathy, Parkinson's disease, amyotrophic lateral sclerosis, Huntington's disease, multiple sclerosis, stroke, thrombosis, embolism, head trauma, cerebral circulatory or metabolic disorders, brain dysfunction, and dementia.

The present application relates to triazolopyridopyrimidine and dihydroimidazopyridopyrimidine compounds having activity as inhibitors of generally regulated repressor protein 2 (GCN2) kinase, processes for their preparation, compositions comprising them and their use, for example in therapy. More specifically, the present application relates to compounds useful in the treatment of diseases, disorders or conditions treatable by inhibition of GCN2 kinase, such as cancer and neuronal diseases. (I)