15 results about "Transthyretin" 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.

Disclosed is a method for diagnosing a cancer health state, or a change in cancer health state in a patient, or for diagnosing a risk of the change or presence of a cancer in a patient, comprising determining, in a plasma sample from said patient, one or more biomarker values that correspond to transthyretin-containing complex structures, and assigning the patient as having or not having cancer, or having or not having a change in cancer health state, or having or not having a risk of cancer based on said biomarker values, wherein said cancer is preferably lung cancer (LC).

The disclosure relates to RNAi constructs, such as siRNA, for reducing expression of the TTR gene. Methods of using such RNAi constructs to treat or prevent cardiac disease, such as transthyretin-associated cardiomyopathy (ATTR-CM), are also described.

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

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.

Disclosures herein are directed to polypeptide probes that may be used to detect transthyretin (TTR) oligomers or fibrils in patient samples obtained from subjects with wildtype and mutant TTR alleles. Also provided are methods of using the provided probes to diagnose subjects with a TTR-associated disease or condition or for monitoring the efficacy of a therapeutic administered to treat a TTR-associated disease or condition.

Based on co-drugs representing two different chemical entities, and the co-administration and sequential administration of said two chemical entities, novel therapies are provided for macular degeneration, non-alcoholic fatty liver disease (NAFLD), and gouty arthritis (gout). The first component (“Selective RBP4 Antagonist”) is a chemical entity that interacts with RBP4 (retinol-binding protein 4), part of the RBP4-TTR (transthyretin) complex that delivers retinol to the retina. This component reduces the transport of retinol from circulation to the retina. The second component (“C20-D3-Visual Chromosome-Generating Compound”) is a C20-D3-modified retinoid or carotenoid that, when metabolized in mammals, ultimately produces a C20-D3 visual chromophore in the retina representing either C20-D3-9-cis-retinal or C20-D3-11-cis-retinal. Deuteration at the C20 position reduces the formation of lipofuscin biretinol, while other functions (such as providing a precursor for the in vivo synthesis of visual chromophore 11-cis-retinaldehyde) 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.

An RNAi agent and composition that inhibits expression of transthyretin (TTR) for use in the treatment and / or prevention of ATTR-associated diseases, including ATTR-PN (transthyretin amyloidosis polyneuropathy), ATTR-CM (transthyretin amyloidosis cardiomyopathy), senile systemic amyloidosis disease, leptomeningeal / central nervous system amyloidosis disease.

Compositions and regimens useful in treating Wilson's Disease are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human ATP7B.

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

Disclosed is a method for diagnosing a cancer health state, or a change in cancer health state in a patient, or for diagnosing a risk of the change or presence of a cancer in a patient, comprising determining, in a plasma sample from said patient, one or more biomarker values that correspond to transthyretin-containing complex structures, and assigning the patient as having or not having cancer, or having or not having a change in cancer health state, or having or not having a risk of cancer based on said biomarker values, wherein said cancer is preferably lung cancer (LC).

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.