Table 2.

Adverse events/lack of efficacy reported with the use of the first generation of AAV vectors

Sponsor	Disease	Serotype	Vector dose (vg/kg)	Symptoms/outcome
Solid Biosciences	DMD	AAV9	3 × 1014	thrombocytopenia, renal failure, cardio-pulmonary insufficiency
Pfizer	DMD	AAV9	2 × 1014	acute kidney injury, atypical hemolytic uremic syndrome-like complement activation, thrombocytopenia
Sarepta Therapeutics	DMD	AAVrh74	2 × 1014	failed to reach primary clinical endpoint
Amicus Therapeutics	BD	AAV9	5 × 1013	failed to reach primary clinical endpoint

DMD, Duchenne muscular dystrophy; BD, Batten disease.