BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B

Inclusion Criteria:

• Adult Males (≥18) with moderately severe to severe Hemophilia B (FIX deficiency)

• Received ≥50 exposure days to Factor IX products preceding enrollment.

• Currently receiving prophylaxis treatment

• Adequate organ function and clinical labs

• Able to tolerate study procedures including leukapheresis.

Exclusion Criteria:

• Pre-existing or history of specific diseases

• B-Cell malignancy, EBV lymphoproliferative disease

• Primary immunodeficiency disease or disorder (PIDD) or systemicimmuno-suppression

• Arterial and/or venous thromboembolic events within 2 years prior to dosing

• History of anaphylaxis or nephrotic syndrome

• Active infection (HIV, Hep B or C)

• History of inhibitor to FIX or inhibitor

• History of an allergic reaction or anaphylaxis to FIX products

• Planned surgical procedure within 6 months from BE-101 administration

• Previously dosed with gene therapy

• Participated in an interventional study and/or received an interventional study drugwithin 30 days or five-half-lives (whichever is longer) of consent into BeCoMe-9 andfor the duration of the study

• Planned participation in clinical trial within one year after BE-101

• Administration of an investigational agent or vaccine within 28 days ofleukapheresis and dosing of BE-101

Other protocol-defined inclusion/exclusion criteria may apply.