Complement-Mediated Thrombotic Microangiopathy Associated with Systemic AAV Gene Therapy

Time: 4:00 pm
day: Day One


• 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