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43 results about "Trypsin deficiency" patented technology

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.

Method for treating pulmonary diseases using rho kinase inhibitor compounds

InactiveUS20100204210A1Not effectiveBiocidePharmaceutical delivery mechanismDiseaseOccupational pulmonary disease
This invention relates to methods of treating pulmonary diseases in patients that beta adrenergic receptor agonist therapy is not effective. The method comprises the steps of: identifying a patient who suffers from a pulmonary disease and has reduced responsiveness to treatment with one or more beta adrenergic receptor agonists, and administering to the patient an effective amount of a Rho kinase inhibitor compound, wherein said pulmonary disease is selected from the group consisting of: asthma, chronic obstructive pulmonary disease, respiratory tract illness caused by respiratory syncytial virus infection such as RSV-induced wheezing, airway hyperreactivity, or bronchiolitis, bronchiectasis, alpha-1-antitrypsin deficiency, lymphangioleiomyomatosis, cystic fibrosis, bronchiolitis or wheezing caused by agents other than respiratory syncytial virus, chronic bronchitis, and occupational lung diseases.
Owner:INSPIRE PHARMA

Novel genes, compositions, and methods for modulating the unfolded protein response

InactiveUS20050250182A1Increased transactivation potentialIncrease volumeSugar derivativesHydrolasesAutoimmune conditionAutoimmune disease
The present invention relates to methods and compositions for modulating the unfolded protein response. The method further relates to methods and compositions for the treatment and diagnosis of protein conformational diseases or disorders, including, but not limited to, α1-antitrypsin deficiency, cystic fibrosis, and autoimmune diseases and disorders. The invention further provides methods for modulating the unfolded protein response by modulating XBP1 mRNA splicing.
Owner:UNIV OF MICHIGAN THE

Chimeric dystrophin proteins to treat dystrophinopathies

A chimeric protein that is a fusion construct of a series of functional domains is used to deliver a therapeutic agent to a human subject suffering from disease. In some embodiments, the chimeric protein includes a therapeutic region and a transportation region. The transportation region allows the chimeric protein to be moved across a cellular membrane of an affected cell within the subject. The therapeutic region can be effective in the treatment of, for example, muscular dystrophy, diastrophic dysplasia, malignant melanoma, porphyria, alpha-1 antitrypsin deficiency, Aicardi-Goutieres syndrome, cystic fibrosis, progeria, Marfan syndrome, tuberous sclerosis, adrenoleukodystrophy, and the like.
Owner:SERENDIPITY BIOTECH INC

Kit used for screening genetic liver diseases

ActiveCN108913761AInterpretation is clear and objectiveHigh detection throughputMicrobiological testing/measurementNPC1Autosomal Recessive Polycystic Kidney Disease
The invention discloses a kit used for screening genetic liver diseases. The kit is high in detection flux, sensitivity, and specificity. The genetic liver diseases comprise progressive familial intrahepatic cholestasis, benign recurrent intrahepatic cholestasis, congenital bile acid synthesis defect, idiopathic bile acid malabsorption, Dubin-Johnson syndrome, hereditary hemochromatosis, alpha1 antitrypsin deficiency, glycogen storage disease, autosomal recessive polycystic kidney disease, Budd-Chiari syndrome, and the like. 39 genes, including ATP8B1, ABCB11, ABCB4, TJP2, NR1H4, HSD3B7, AKR1D1, CYP7B1, AMACR, ABCD3, SLC10A2, ABCC2, HFE, TFR2, SERPINA1, G6PC, SLC37A4, AGL, PYGL, SLC40A1, PKHD1, F5, GYS2, VIPAS39, SLC25A13, JAG1, NOTCH2, PHKA2, PHKB, PHKG2, PHKA1, ALAD, HAMP, HFE2, SMPD1, ATP7B, ABCA1, NPC2, and NPC1, are detected in screening of genetic liver diseases. The kit comprises targeting capture probes SEQ NO:1-SEQ NO:722 targeting at all the exons of the 39 genes, and the kitis used for gene detection.
Owner:施军平 +1

Classification and diagnosis of the molecular basis of cholestasis

The methods and compositions of the invention find use in the clinical diagnosis of cholestasis related syndromes, particularly PFIC types 1, 2, and 3; BRIC types 1 and 2; Alagille syndrome, and alpha 1-antitrypsin deficiency. The compositions of the invention include isolated nucleic acid molecules and oligonucleotide pairs suitable for use in amplifying regions of cholestasis related genes. Compositions of the invention include a cholestasis related gene resequencing microarray suitable for determining the nucleotide sequence of a region of a cholestasis related gene. Knowledge of the nucleotide sequence of one or more regions of a patient's cholestasis related gene allows diagnosis of the patient's syndrome.
Owner:CHILDRENS HOSPITAL MEDICAL CENT CINCINNATI

Exosome loaded therapeutics for the treatment of non-alcoholic steatohepatitis, diabetes mellitus type 1 and type 2, atherosclerotic cardiovascular disease, and alpha 1 antitrypsin deficiency

PendingUS20200157541A1Superior in potency and efficacyHigh potencySpecial deliveryMicroencapsulation basedCytoplasmTrypsin
A composition for delivering a cargo to the cytoplasm of a cell, wherein the cargo treats Non-Alcoholic Steatohepatitis, Non-Alcoholic Fatty Liver Disease, Diabetes Mellitus Type 1 and Type 2, Atherosclerotic Cardiovascular Disease, and Alpha 1 Antitrypsin Deficiency. In another embodiment, the composition comprises: an exosome; cargo located inside the exosome, wherein the cargo comprises short interference RNA (siRNA) that depletes sodium-glucose linked transporter active sites. In another embodiment, the composition comprises: an exosome; cargo location inside the exosome, wherein the cargo corrects the missense SERPINA1 mutation from ‘Z’ to ‘M’.
Owner:EXOSOME THERAPEUTICS INC

Bone Marrow-Derived Cells Ameliorates The Pathological Consequences Of The Liver In Case Of Alpha1-Antitrypsin Deficiency

The present invention is based on the findings that bone marrow (BM)-derived progenitor cells more specifically mesenchymal stem cells (MSCs), hematopoietic stem cells (HSCs) and uncommitted hematopoietic cells (lin−) are capable of regenerating liver in case of injury. The invention provides a method for treating genetic disorder like Alpha1-antitrypsin deficiency (A1-ATD) by administering BM derived Lin− cells in human mutant A1-AT expressing transgenic mouse model. The invention also provides the state of art for replacement of mutant host hepatocytes by transplanting wild-type uncommitted donor (lin−) cells.
Owner:NATIONAL INSTUTUTE OF IMMUNOLOGY

Novel alpha-1 antitrypsin variant, preparation method thereof and use thereof

ActiveUS20140371160A1Improve stabilityMaintains inhibitory effect on elastase activityFactor VIIDipeptide ingredientsHalf-lifeBlood drug concentration
A novel alpha-1 antitrypsin variant, a method of preparing the same, and use thereof are provided. The alpha-1 antitrypsin variant has excellent stability in the body and maintains an inhibitory effect on elastase activities because the blood half-life (t1 / 2) and the area under blood drug concentration vs. time curve (AUC) are remarkably increased by adding an N-glycosylation site in animal cells through amino acid mutation between 1st and 25th positions of the N-terminus of alpha-1 antitrypsin. Therefore, the alpha-1 antitrypsin variant can be useful in preventing or treating alpha-1 antitrypsin deficiency.
Owner:ALTEOGEN

Methods Of Treatment For Alpha-1 Antitrypsin Deficiency

InactiveUS20190071670A1Reduce the burden onEnhance intracellular autophagy processOrganic active ingredientsDNA/RNA fragmentationDiseaseReticulum cell
The application relates to methods of treatment and treatment regimens for Alpha-1 Antitrypsin deficiency (AATD) and the conditions, manifestations, and diseases caused by AATD, by the administration of one or more expression-inhibiting oligomeric compounds having a nucleobase sequence complementary to a coding sequence in the Alpha-1 Antitrypsin (A1AT or AAT) gene that inhibits the expression of the AAT gene, in combination with one or more autophagy enhancing agents that enhance and / or induce the endogenous autophagy mechanism to facilitate and encourage clearance of polymerized mutant AAT protein accumulated in the endoplasmic reticulum of hepatocytes. When used in combination, delivery of the expression-inhibiting oligomeric compounds to liver cells in vivo provides for inhibition of AAT gene expression and the use of autophagy enhancing agents increases the rate of the intracellular autophagy mechanism to clear polymerized mutant Z-AAT protein accumulated in cells, leading to an improved treatment of AATD and prevention and treatment of conditions and diseases associated with AATD.
Owner:ARROWHEAD PHARMA INC

System for insurance underwriting and post policy issuance action

A system for insurance underwriting and post policy issuance action comprising obtaining personal, medical, and genetic information for an applicant and determining the applicant's eligibility for an insurance policy is provided. The applicant may additionally be provided with information regarding genetic testing for alpha-1 antitrypsin deficiency or with information regarding testing of circulating AAT levels. Furthermore, alpha-1 antitrypsin testing may be ordered for the applicant as part of the insurance underwriting process or for the insured following policy acceptance or issuance.
Owner:EGLY MARK

Beta-hairpin peptidomimetic with elastase inhibitory activity and aerosol dosage forms thereof

The present invention relates to pharmaceutical aerosols comprising a β-hairpin peptidomimetic of formula cyclo(-OctG-Glu-Thr-Ala-Ser-Ile-Pro-Pro-Gln-Lys-Tyr-DPro-Pro-), or a pharmaceutically acceptable salt thereof, having inhibitory activity against human neutrophil elastase. It further relates to solid or liquid pharmaceutical compositions and kits for preparing and administering such aerosols. The invention can be used for the prevention, management or treatment of pulmonary diseases, such as alpha-1 antitrypsin deficiency (AATD), cystic fibrosis (CF), non-cystic fibrosis bronchiactasis (NCFB), or chronic obstructive pulmonary disease (COPD), or infections, or diseases, or conditions of the lungs, being mediated by or resulting from human neutrophil elastase activity. Thus, the invention further relates to a pharmaceutical composition or a pharmaceutical aerosol comprising the active compound cyclo(-OctG-Glu-Thr-Ala-Ser-Ile-Pro-Pro-Gln-Lys-Tyr-DPro-Pro-), or any pharmaceutically acceptable salt thereof, for use in a method for the prevention, management or treatment of diseases or conditions of the lungs being mediated by or resulting from human neurophil elastase activity in a subject.
Owner:POLYPHOR AG

Beta-hairpin peptidomimetic with elastase inhibitory activity and aerosol dosage forms thereof

The present invention relates to pharmaceutical aerosols comprising a β-hairpin peptidomimetic of formula cyclo(-OctG-Glu-Thr-Ala-Ser-Ile-Pro-Pro-Gln-Lys-Tyr-DPro-Pro-), or a pharmaceutically acceptable salt thereof, having inhibitory activity against human neutrophil elastase. It further relates to solid or liquid pharmaceutical compositions and kits for preparing and administering such aerosols. The invention can be used for the prevention, management or treatment of pulmonary diseases, such as alpha-1 antitrypsin deficiency (AATD), cystic fibrosis (CF), non-cystic fibrosis bronchiactasis (NCFB), or chronic obstructive pulmonary disease (COPD), or infections, or diseases, or conditions of the lungs, being mediated by or resulting from human neutrophil elastase activity. Thus, the invention further relates to a pharmaceutical composition or a pharmaceutical aerosol comprising the active compound cyclo(-OctG-Glu-Thr-Ala-Ser-Ile-Pro-Pro-Gln-Lys-Tyr-DPro-Pro-), or any pharmaceutically acceptable salt thereof, for use in a method for the prevention, management or treatment of diseases or conditions of the lungs being mediated by or resulting from human neutrophil elastase activity in a subject.
Owner:POLYPHOR AG

Use of a neutrophil elastase inhibitor in lung disease

The invention relates to methods for treating chronic lung disease, in particular, alpha-1 antitrypsin deficiency or emphysema resulting from alpha-1 antitrypsin deficiency, with a neutrophil elastase inhibitor. The invention further relates to pharmaceutical compositions comprising a neutrophil elastase inhibitor.
Owner:PH PHARMA CO LTD

Methods for treating alpha-1 antitrypsin deficiency (AATD)

PendingCN114222820ASlow or stop progressOrganic active ingredientsGenetic material ingredientsDiseaseIncreased hepatocellular carcinoma risk
Methods of treating alpha-1 antitrypsin deficiency (AATD) in a human patient in need thereof using a pharmaceutical composition comprising an AAT RNAi agent are described. The pharmaceutical compositions comprising AATRNAi agents disclosed herein treat liver diseases associated with AAT deficiency, such as chronic hepatitis, cirrhosis, increased risk of hepatocellular carcinoma, elevated transaminase, cholestasis, fibrosis, outbreak liver failure, and other liver-related diseases when administered to a human patient in need thereof.
Owner:ARROWHEAD RES CORP

Neutrophil inflammation inhibitor and uses thereof

ActiveUS20200148674A1Ameliorate mitigate prevent symptomTreatment and of subjectOrganic chemistryRespiratory disorderArthritisObstructive chronic bronchitis
Disclosed herein are compounds of formula (I), and pharmaceutical compositions comprising the same. The compounds of formula (I) are neutrophilic inflammation inhibitors, thus, they are useful for treatment and / or prophylaxis of inflammatory diseases and / or disorders associated with abnormal activation of neutrophils, such as ARDS, ALI, COPD, lung fibrosis, chronic bronchitis, pulmonary emphysema, α-1 anti-trypsin deficiency, cystic fibrosis, idiopathic pulmonary fibrosis, liver injury, steatohepatitis, liver fibrosis, damages caused by ischemia and reperfusion, myocardial infarction, shock, stroke, and organ transplantation, ulcerative cholitis, vasculitis, SLE, sepsis, SIRS, arthritis, psoriasis, atopic dermatitis, and inflammatory skin diseases.
Owner:CHANG GUNG UNIVERSITY OF SCIENCE AND TECHNOLOGY +2
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