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6 results about "Trypsin deficiency" patented technology
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Also called AAT deficiency, alpha-1 antitrypsin deficiency is a genetic disease, which means it's passed down from your parents. It can cause serious lung disease that makes it hard to breathe. It can also cause liver disease that leads to jaundice, which makes your skin look yellowish.
RNAi agent for inhibiting the expression of an alpha-1 antitrypsin (AAT) gene, composition comprising the same and uses thereof to inhibit AAT expression and treat alpha-1 antitrypsin deficiency.
This refers to RNAI agents for inhibiting the expression of the alpha-1 antitrypsin (aat) gene, compositions including Aat RNAI agents, and methods of use. Pharmaceutical compositions including one or more Aat RNAI agents together with one or more excipients capable of delivering the RNAI agents to a liver cell in vivo are also disclosed. Delivery of Aat RNAI agents to liver cells in vivo inhibits Aat gene expression and treats diseases associated with Aat deficiency such as chronic hepatitis, cirrhosis, hepatocellular carcinoma, transaminitis, cholestasis, fibrosis, and fulminant hepatic failure.
Owner:ARROWHEAD PHARMACEUTICALS INC
Processes for preparing modulators of alpha-1 antitrypsin
This disclosure provides large-scale processes for preparing a modulator of alpha-1 antitrypsin (AAT) activity that may be useful for treating alpha-1 antitrypsin deficiency (AATD), such as 4-(5-(4-fluorophenyl)-6-(tetrahydro-2H-pyran-4-yl)-1,5-dihydropyrrolo[2,3-f]indazol-7-yl)benzoic acid (Compound 1), 3-[5-(4-fluorophenyl)-6-isopropyl-1H-pyrrolo[2,3-f]indazol-7-yl]propanoic acid (Compound 2), or a pharmaceutically acceptable salt of any of the foregoing.
Owner:VERTEX PHARMACEUTICALS INC
7- or 8-hydroxy-isoquinoline and 7- or 8-hydroxy-quinoline derivatives as alpha-1-antitrypsin modulators for treating alpha-1-antitrypsin deficiency (AATD)
7- or 8-hydroxy-isoquinoline and 7- or 8-hydroxy-quinoline derivatives as alpha-1-antitrypsin modulators for treating alpha-1-antitrypsin deficiency (AATD).
Owner:VERTEX PHARMACEUTICALS INC
Compositions and their use for the treatment of A1-antitrypsin deficiency
The invention relates to pharmaceutical compositions comprising (S)-1-(2-(trifluoromethyl)phenyl)sulfonyl)piperidine-3-carboxylic acid and their medical use, for example for use in the treatment of α1-antitrypsin deficiency (AATD).
Owner:CENTESSA PHARMACEUTICALS (UK) LIMITED
Methods and compositions for treating alpha-1 antitrypsin deficiency
Compositions and methods for editing deleterious mutations associated with Alpha-1 Antitrypsin Deficiency (A1AD). In particular embodiments, the invention provides methods for treating A1AD using a modified adenosine base editor with improved on-target editing and decreased off-target editing to correct mutations associated with A1AD.
Owner:BEAM THERAPEUTICS INC
Treatment for alpha-1 antitrypsin deficiency (AATD)
To provide methods for treatment of alpha-1 antitrypsin deficiency (AATD) in a human subject.SOLUTION: Described are methods for treating alpha-1 antitrypsin deficiency (AATD) in a human patient in need of treatment, using pharmaceutical compositions that include AAT RNAi agents. The pharmaceutical compositions disclosed herein that include AAT RNAi agents are administered to a human patient in need thereof for treating liver diseases associated with AAT deficiency such as chronic hepatitis, cirrhosis, increased risk of hepatocellular carcinoma, transaminitis, cholestasis, fibrosis, fulminant hepatic failure, and other liver-related diseases.SELECTED DRAWING: Figure 3
Owner:ARROWHEAD PHARMACEUTICALS INC