BEQVEZ™ Nonclinical Toxicology

(fidanacogene elaparvovec-dzkt)

13 NONCLINICAL TOXICOLOGY

  

13.1 Carcinogenesis, Mutagenesis, Impairment of Fertility

Carcinogenicity, mutagenicity, reproductive toxicity, and impairment of fertility studies have not been conducted with BEQVEZ. In a 2-year vector integration study in NHPs administered 5 × 1012 vg/kg, vector DNA was mostly detected in the form of episomal DNA that was not integrated into the host genome. The integration sites were generally random with a low frequency, and there was no indication of significant clonal expansion.

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Nonclinical Toxicology

13 NONCLINICAL TOXICOLOGY

  

13.1 Carcinogenesis, Mutagenesis, Impairment of Fertility

Carcinogenicity, mutagenicity, reproductive toxicity, and impairment of fertility studies have not been conducted with BEQVEZ. In a 2-year vector integration study in NHPs administered 5 × 1012 vg/kg, vector DNA was mostly detected in the form of episomal DNA that was not integrated into the host genome. The integration sites were generally random with a low frequency, and there was no indication of significant clonal expansion.

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