• ClinicalTrials.gov Identifier: NCT02695160¹
• Phase 1 trial to evaluate the safety, tolerability and effect on FIX antigen and activity levels of ascending doses of SB-FIX in severe hemophilia 
• Males > 18 years of age

• Terminated, 1 participant enrolled¹
• Last Update Posted: 2022-03-23¹

Male >18 years of age with severe hemophilia B (native circulating FIX activity <1%)¹

• Presence of neutralizing antibodies
• Polymorphisms in the ZFN target region
• Previously received gene therapy product

AAV2/6; vector manufacturing platform not reported

FIX donor cDNA and Zinc Finger Nucleases ZFN1 and ZFN2 (ZFN)

Expression of the FIX transgene under the control of endogenous albumin locus following AAV gene transfer and ZFN-mediated genome editing¹

Not reported

3 experimental cohorts: low, medium and high dose.

Up to 36 months

Not reported

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Not reported

1. Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B. Available at: Study Details | Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B | ClinicalTrials.gov

AAV, Adeno-associated virus; ABR, Annualized bleeding rate; AEs: adverse events; AIR, Annualized FVIII/FIX infusion rate; ALT, alanine aminotransferase; AST, aspartate aminotransferase; CH, Chromogenic Assay; Co., cohort; DOACs, Direct oral anticoagulants; D, days; EDs, exposure days; FIX, factor IX; FIX-Padua, gain of function FIX variant; FVIII, factor VIII; gc, genome copies; HEK cells, human embryonic kidney cells; IQR, interquartile range; IRR, Infusion-related reaction; NAbs, neutralizing antibodies; OS, One-stage clotting assay; Pop., population; pt., patient/participant; pts., patients/participants; P1, Participant 1; PI, phase I; PBGD, porphobilinogen deaminase; PBMC, peripheral blood mononuclear cells; SAEs, serious adverse events; SFU, spot-forming units; TAC, tacrolimus; ULN, upper limit of normal; VCN, vector copy number; vg, vector genomes; W, weeks; WT, wild type; Y, year