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.