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Alpha-1 antitrypsin deficiency (A1AD or AATD) is a genetic disorder that may result in lung disease or liver disease. [1] Onset of lung problems is typically between 20 and 50 years of age. [ 1 ] This may result in shortness of breath , wheezing , or an increased risk of lung infections .
Alpha-1 antirypsin deficiency: In clinic, phase 3: Orphan Drug designation, partnered with Takeda: ARO-APOC3 [2] Hypertriglyceridemia: Phase 2, 3: Orphan Drug designation, Fast Track Designation, Phase 3 for FCS, Phase 2 for expanded populations ARO-HIF2 [2] Renal cell carcinoma: Preclinical Second generation being worked on presumably ARO-ENaC ...
About Alpha-1 Antitrypsin (AAT) and AAT Deficiency Alpha-1 antitrypsin deficiency is an autosomal disorder that results in disease of the lungs and liver, and afflicts roughly 10,000 patients ...
Alpha-1 antitrypsin or α 1-antitrypsin (A1AT, α 1 AT, A1A, or AAT) is a protein belonging to the serpin superfamily. It is encoded in humans by the SERPINA1 gene . A protease inhibitor , it is also known as alpha 1 –proteinase inhibitor ( A1PI ) or alpha 1 -antiproteinase ( A1AP ) because it inhibits various proteases (not just trypsin ). [ 5 ]
Vertex Pharmaceuticals Inc (NASDAQ: VRTX) has advanced its investigational program targeting alpha-1 antitrypsin deficiency (AATD), a rare genetic disease characterized by a protein folding defect ...
Alpha-1 antitrypsin deficiency is a genetic risk factor that may lead to the condition presenting earlier. [9] When associated with significant airflow limitation, emphysema is a major subtype of chronic obstructive pulmonary disease (COPD), a progressive lung disease characterized by long-term breathing problems and poor airflow.
Intellia also has a number of research programs for in vivo and ex vivo therapeutic candidates with potential applications in diseases including cancer, alpha-1 antitrypsin deficiency, and hemophilia. The company is also working on a variety of additional gene editing technologies including base editing and DNA writing. [5] [6]
Management of the disorder has been based on general recommendations for patients with liver disease, particularly Alpha 1 antitrypsin deficiency-associated liver disease. In the latter disease, autophagy , the pathway that cells use to dispose of dysfunctional or excessively stored components including proteins, has been targeted using ...