Valoctocogene roxaparvovec gene transfer in participants with HIV

Abstract

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Gene Therapy in Hemophilia A: Achievements, Challenges, and Perspectives

“…Results of participants with HIV infection on highly active antiretroviral therapy (HAART) who received valoctocogene roxaparvovec and those who were previously excluded from the efficacy analysis are available. 68 Two participants received a higher viral genome dose of 6 × 10 13 vector genomes per kg and one participant received a dose of 4 × 10 13 vector genomes per kg (enrolled in GENEr8-1 [301; NCT03370913] and GENEr8-2 [302; NCT03392974], respectively). Both participants who received the higher dose responded with endogenous production of FVIII and reduced annualized FVIII infusion rate though one of them experienced bleeding events requiring FVIII infusion as FVIII activity levels declined after week 105.…”

“…The same participant had multiple hepatotoxic side effects which were thought to be related to interaction with efavirenz containing HAART. 68…”

“…The same participant had multiple hepatotoxic side effects which were thought to be related to interaction with efavirenz containing HAART. 68 In regard to other patients who are immunocompromised, the decision regarding gene therapy eligibility is at the discretion of the treating clinician.…”