Phase I trial of gene therapy for age-related macular degeneration completed

Results from Phase I trial NCT01024998 show that intravitreous gene therapy for age-related macular degeneration is safe and well tolerated. The study, which took place in hospitals across the US, used an rAAV2 vector to express VEGF-neutralising protein sFLT01 in the eye. Modest expression of the gene was achieved in patients receiving the highest dose, without major adverse effects. Patients with pre-existing neutralising antibodies to AAV2 did not show any expression and may not benefit from the treatment. Further studies will be needed to explore higher doses and to more fully understand the role of anti-AAV2 antibodies on expression. 

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