NKG-001, a novel gene therapy product featuring a next-generation AAV vector (cceAAV for covalently closed-end double-stranded AAV), was tested intravenously in two children with proximal spinal muscular atrophy (SMA) aged one year (with two copies of SMN2) and two years (with three copies of SMN2) who were already being treated with nusinersen or risdiplam.
- NKG-001 shares the same genetic sequence as that used for Zolgensma, except at the terminal level (mITR is replaced by TeIN) in order to increase the efficiency of transduction and also the expression of the transgene.
- It was well tolerated, with no serious side effects.
- Both children showed rapid improvements in motor function.
