A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)

Exclusion Criteria16

• Body mass index >30
• Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction surgery.
• Clinical evidence of severe bronchiectasis as per the discretion of the investigator (eg, excessive sputum production or recurrent infections requiring antibiotic use \[>4x/year\]).
• Liver disease with any of the following:
• FibroScan liver stiffness measurement ≥7.5 kilopascals (kPa). (For sites without access to FibroScan, APRI >0.5 can be used as a surrogate exclusion criterion \[Yilmaz, 2011\].
• Known history of liver cirrhosis or complications of cirrhosis (eg, varices, ascites, hepatic encephalopathy).
• Presence of ≥F2 liver fibrosis if a patient has previously had a liver biopsy.
• Have ALT or AST > upper limit of normal (ULN).
• Total bilirubin levels > ULN; if documented Gilbert's Syndrome, total bilirubin >2 × ULN.
• INR ≥1.2 at screening. If deemed appropriate by the investigator and/or prescribing physician, the patient may stop taking anticoagulants for an appropriate washout period or reversal with vitamin K and if indicated, a repeat INR within <1.2 would be acceptable.
• Seropositive for hepatitis B (positive surface Ag).
• Active hepatitis C by hepatitis C virus (HCV) antibody. If HCV antibody positive, must be HCV RNA polymerase chain reaction (PCR) negative.
• Part B:
• Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction surgery.
• Clinical evidence of severe bronchiectasis as per the discretion of the investigator (eg, excessive sputum production or recurrent infections requiring antibiotic use \[>4x/year\])
• Previous diagnosis of liver cirrhosis or complications of cirrhosis (eg, varices, ascites, hepatic encephalopathy).