Advanced therapies


Luxturna is a gene therapy used for treating inherited retinal dystrophies caused by changes in the RPE65 gene. RPE65-related retinitis pigmentosa is a rare genetic disorder of the retina, the light sensitive membrane at the back of the eye.


Zolgensma  is a gene therapy for treating Spinal Muscular Atrophy (SMA). SMA is a serious condition of the nerves caused by changes in the  SMN1  gene that results in  muscle wasting and weakness. Zolgensma is intended for patients diagnosed with SMA type 1, the most severe type.