Antitrypsin

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WVE-006 is the most advanced candidate for AATD designed to restore functional wild-type AAT protein and reduce Z-AAT protein aggregation with potential for disease modification in both lung and liver phenotypes IND-enabling activities…

Statistically significant inhibition of blood neutrophil elastase activity of up to 90% in patients in both high and low dose alvelestat groups throughout the 12-week dosing period Statistically significant reductions in the…

Presentation at OPT 2022 to highlight AATD program, including reduction of liver aggregates with repeat AIMer dosing out to 19 weeks Multiple presentations and panel discussions at 3rd Annual RNA Editing Summit to highlight Wave’s…

PITTSBURGH, Oct. 14, 2021 (GLOBE NEWSWIRE) -- Krystal Biotech Inc., (“Krystal”) (NASDAQ: KRYS), the leader in redosable gene therapies for rare diseases, today announced that it will present pre-clinical pharmacology data on its second…

Los Angeles, USA, Aug. 12, 2021 (GLOBE NEWSWIRE) -- Alpha-1 Antitrypsin Deficiency Liver Disease Pipeline: Insights into New Pipeline Therapies and Landscape; Future Key Pharma Companies and Novel Therapeutic Assessment Alpha-1…

First proof-of-concept in vivo data for RNA editing using endogenous ADAR enzymes in alpha-1 antitrypsin deficiency ADAR editing resulted in therapeutically meaningful restoration of circulating functional AAT protein Wave’s program in…