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The researchers made the interesting finding using a trial involving patients with a rare genetic condition called Alpha-1 antitrypsin deficiency (AATD). ....
Inhibrx Inc. and Sanofi SA have agreed to a deal worth up to $2.2 billion, whereby the latter’s Aventis Inc. subsidiary will acquire INBRX-101, an optimized, recombinant alpha-1 antitrypsin (AAT) augmentation therapy undergoing a registrational trial for AAT deficiency (AATD), an inherited genetic disorder caused by single nucleotide variants in the SERPINA1 gene. ....
Takeda Canada Inc., the Canadian arm of Japan's Takeda Pharmaceutical Co. Ltd. (TAK) on Wednesday entered into a contract with Canadian Blood Services (CBS) for GLASSIA (alpha-1 proteinase inhibitor) resulting from CBS's request for proposal for hereditary deficiency of Alpha-1 Antitrypsin Deficiency (Alpha-1). ....
Boosting levels of a deficient protein has clear survival benefits for people with the genetic condition alpha-1 antitrypsin deficiency (AATD), according to new research led by RCSI University of Medicine and Health Sciences. ....