16 results about "Amyloidosis" patented technology

Based on co-drugs representing two different chemical entities and the co- and sequential administration of the two chemical entities, novel therapies for macular degeneration and TTR amyloidosis are provided. The first component (“selective TTR ligand”) is a chemical entity that binds to TTR in the RBP4 (retinol-binding protein 4)-TTR (transthyretin) complex, which participates in the delivery of retinol to the retina. This component reduces retinol transport from circulation to the retina and provides stabilization of the TTR tetramer. The second component (“C20-D3-visual chromophore-generating compound”) is a C20-D3 modified retinoid or carotenoid that, when metabolized in mammals, ultimately produces a C20-D3 visual chromophore, which is presented in the retina as C20-D3-9-cis-retinal or C20-D3-11-cis-retinal. Deuteration at C20 reduces the formation of lipofuscin biretinol, but other functions (such as providing a precursor for the synthesis of the visual chromophore 11-cis-retinaldehyde in vivo) are not reduced.

The present invention relates to peptides that have preventive, ameliorative, or therapeutic effects against amyloidosis and / or macular degeneration, are safe for living organisms, and have few side effects including abnormal reactions, as well as pharmaceutical compositions and health functional foods containing the same.

The present disclosure relates in some aspects to methods and compositions for detecting amyloid-related diseases in an individual.

Immunotherapy for musculoskeletal disorders and conditions associated with transthyretin amyloidosis is provided.

Aspects of the present invention disclose compounds that modulate the aggregation of amyloidogenic proteins or peptides. In some aspects, disclosed compounds modulate the aggregation of disease-associated proteins and natural β-amyloid peptides. In a preferred embodiment, the compounds can inhibit natural amyloid aggregation. Pharmaceutical compositions comprising the compounds of the embodiments, and diagnostic and treatment methods for diseases (e.g., amyloidogenic diseases) using the compounds, are also disclosed. In addition, there is provided an integrated bacterial platform for the discovery of rescuers of disease-associated protein misfolding.

The compounds are used for the detection and imaging of amyloid plaques or both of proteins or peptides, for screening or testing the efficacy of inhibitors against amyloidosis and / or fibrillary growth of proteins or peptides, and / or for the detection of RNA and nucleolar imaging. The compounds are d 8 or d 10 Metal complexes or salts thereof. The metal complexes of said compounds can bind to amyloid proteins or peptides, plaques or both and / or RNA, nucleoli or both. This binding induces the accumulation and supramolecular self-assembly of the metal complexes, thereby causing changes in the photophysical properties of the metal complexes.

This disclosure pertains to the field of biomedicine, specifically relating to double-stranded oligonucleotides targeting the APP gene and their applications. Specifically, it provides double-stranded oligonucleotide agents or their salts, conjugates, or compositions for inhibiting amyloid precursor protein (APP) expression, wherein the double-stranded oligonucleotide agent comprises a sense strand and an antisense strand forming a double-stranded region; wherein the antisense strand sequence comprises at least 15 consecutive nucleotides of any of the sequences shown in SEQ ID NO:1-154 with a difference of no more than 3 nucleotides, and / or the sense strand sequence comprises at least 15 consecutive nucleotides of any of the sequences shown in SEQ ID NO:155-308 with a difference of no more than 3 nucleotides. The double-stranded oligonucleotide agent or its salt for inhibiting APP expression disclosed in this application can significantly inhibit APP expression and can be used for the prevention and / or treatment of diseases or conditions mediated by the APP gene and / or associated with protein amyloidosis.

The present invention provides compositions used for the treatment and / or prevention of protein aggregation disorders. [Solution] Proteopathy encompasses a wide range of ailments, including neurodegenerative diseases (e.g., polyglutamine diseases such as huntingtin in Alzheimer's disease, Parkinson's disease, and Huntington's disease, and prion diseases); amyloidosis of other non-neuronal proteins (especially I1-antitrypsin, immunoglobulin light and heavy chains, lactadherin, apolipoprotein, gelzolin, lysozyme, fibrinogen, atrial natriuretic factor, keratin, lactoferrin, and β-2 microglobulin, etc.); sickle cell disease; cataracts; cystic fibrosis; retinitis pigmentosa; and nephrogenic diabetes insipidus. Administration of sulfatase inhibitors is generally suitable for treating and / or preventing protein toxicity associated with proteopathy. Therefore, the present invention provides compositions comprising sulfatase inhibitors for the treatment of proteopathy.

Based on co-drugs representing two different chemical entities and the co- and sequential administration of the two chemical entities, novel therapies for macular degeneration and TTR amyloidosis are provided. The first component (“Bispecific RBP4 / TTR ligand”) is a chemical entity that binds to both TTR and RBP4 in the RBP4 (retinol-binding protein 4)-TTR (transthyretin) complex, which participates in the delivery of retinol to the retina. This component reduces retinol transport from circulation to the retina and provides stabilization of the TTR tetramer. The second component (“C20-D3-visual chromophore-generating compound”) is a C20-D3 modified retinoid or carotenoid that, when metabolized in mammals, ultimately produces a C20-D3 visual chromophore, which is presented in the retina as C20-D3-9-cis-retinal or C20-D3-11-cis-retinal. Deuteration at C20 reduces the formation of lipofuscin biretinol, but other functions (such as providing a precursor for the synthesis of the visual chromophore 11-cis-retinaldehyde in vivo) are not reduced.

Anti-transthyretin (TTR) antibodies, corresponding polynucleotides, and expression vectors, as well as compositions (e.g., pharmaceutical compositions) containing anti-TTR antibodies as drugs and related manufactured articles are provided herein. Methods for treating or preventing transthyretin-mediated amyloidosis (ATTR) in subjects requiring such treatment or prevention using the pharmaceutical compositions described herein are also provided herein. Additionally, methods for verifying, identifying, and screening amyloid-depleting drugs using high-resolution live cell imaging are provided herein, as well as methods for producing pharmaceutical compositions of amyloid-depleting drugs and kits suitable for use in said methods.

This disclosure provides a method for treating a subject with cancer or light chain amyloidosis, the method comprising administering to the subject a combination therapy containing an effective amount of nirostat dihydrobromide form A and B cell maturation antigen (BCMA) targeted therapy and providing the use of the combination therapy.

To provide an ATP production promoter useful for preventing or treating diseases associated with decreased ATP production, particularly renal impairment. [Solution] The present invention comprises an ATP production promoter containing a component having URAT1 inhibitory activity. Preferably, the component having URAT1 inhibitory activity is at least one selected from the group consisting of benzbromarone, probenecid, dotinurad, recinurad, and berinurad. The present invention is expected to be useful as a preventive and therapeutic agent for diseases of decreased ATP production, and is particularly useful as a therapeutic and / or preventive agent for nephrotic syndrome, chronic glomerulonephritis, chronic tubulointerstitial nephritis, chronic pyelonephritis, amyloid kidney, familial juvenile hyperuricemia nephropathy, autosomal dominant tubulointerstitial kidney disease, nephronoplasia, renovascular hypertension, renal vein thrombosis, renal arteriovenous fistula, renal tubular acidosis, Gitelman syndrome, Bartter syndrome, Fanconi syndrome, Lowe syndrome, Alport syndrome, kidney and ureteral stones, and chronic renal failure.

This document provides anti-thyroxine transporter (TTR) antibodies, corresponding polynucleotides and expression vectors, and compositions (e.g., pharmaceutical compositions) containing anti-TTR antibodies as drugs, and related articles. This document also provides methods for treating or preventing thyroxine transporter-mediated amyloidosis (ATTR) in subjects of need using pharmaceutical compositions. Additionally, this document provides methods for validating, identifying, and screening amyloid depletion drugs using high-resolution live-cell imaging, methods for generating pharmaceutical compositions of amyloid depletion drugs, and kits suitable for said methods.

Peptide-based vaccines are provided to treat or prevent amyloid-transthyretin amyloidosis (ATTR).

Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene are provided. Compositions and methods for treating subjects having amyloidosis associated with transthyretin (ATTR), are provided.

The present invention provides a method for reducing or suppressing the increase of the Neuropathy Impairment Score (NIS) or Revised NIS (mNIS+7) in human subjects. [Solution] A method for reducing the neuropathic impairment score (NIS) or revised NIS (mNIS+7) of a human subject with TTR-related disorder, comprising the step of administering an effective amount of a transthyretin (TTR) inhibitor composition to a human subject.