Slide 1: Valoctocogene Roxaparvovec Gene Transfer in Participants with HIV
VALOCTOCOGENE ROXAPARVOVEC GENE TRANSFER IN PARTICIPANTS WITH HIV Margaret V Ragni,1 Elaine Majerus,2 Sylvia Fong,3 Bridget Yates,3 Stephen Scheeler,3 Lisa Razon,3 Su Liu,3 Hua Yu,3 Divya B Reddy,3 Tara M Robinson3 1Medicine and Clinical and Translational Science, Department of Medicine, Division He
Slide 2: Disclosure for Margaret V Ragni
DISCLOSURE FOR MARGARET V RAGNI In compliance with COI policy, EAHAD requires the following disclosures to the session audience: Shareholder No relevant conflicts of interest to declare Alnylam/Sanofi, BioMarin Pharmaceutical Inc., Grant / Research Support Bioverativ/Sanofi, Spark Therapeutics, Take
Slide 3: Introduction, Objectives, Methods and Results
INTRODUCTION AND OBJECTIVES RESULTS • Valoctocogene roxaparvovec transfers a B-domain deleted human FVIII coding sequence Participant demographics and baseline characteristics controlled by a hepatocyte-selective promoter using an AAV5 vector, enabling endogenous • Overall, 3 participants with HIV e
Slide 4: Efficacy and Safety
EFFICACY SAFETY • At week 52 post-gene therapy, Participants 1 and 2 had FVIII per • Participant 1 experienced 6 Figure 2. Liver function test results for Participant 3 CSA of 24.3 and 6.1 IU/dL, respectively AEs of Grade ≤2; none – Both had reduced bleeding and FVIII utilization compared to were li
Slide 5: In Vitro Drug-Drug Interaction Analysis and Conclusions
IN VITRO DRUG-DRUG INTERACTION ANALYSIS CONCLUSIONS • ALT elevations for Participant 3 likely resulted from drug interactions between the efavirenz Figure 3. Efavirenz cytotoxicity • After valoctocogene component of his HAART regimen and valoctocogene roxaparvovec 100 roxaparvovec gene transfer, 2 •