Complement-Mediated Thrombotic Microangiopathy Associated with Systemic AAV Gene Therapy
Time: 4:00 pm
day: Day One
Details:
• Review of the complement system and pathophysiology of thrombotic microangiopathy (TMA)
• Learnings from the TMA experience in AAV gene therapy programs to date
• Mechanistic hypotheses regarding TMA risk
• Practical considerations around TMA surveillance, prophylaxis and management