Stargardt eye disease trial begins

SpliceBio, a clinical-stage genetic medicines company, announced that it has dosed the first patient in the phase ½ Astra study of SB-007, a dual adeno-associated viral vector, for the treatment of Stargardt disease - a rare, inherited genetic eye condition that causes progressive vision loss.

‘Stargardt disease has remained elusive to gene therapies due to the large size of the ABCA4 gene. SB-007 is the first gene therapy in clinical development designed to restore expression of the full-length ABCA4 protein across all Stargardt disease patients, regardless of their mutations,” said Miquel Vila-Perelló, chief executive officer and co-founder of SpliceBio.