Swiss Roche is looking to dominate market in Spinal Muscular Atrophy (SMA) research, now also investing in AveXis as part of €58M series D investment in their SMA one-time treatment pipeline. Other veteran VC parties include Deerfield Management and Venrock, with new investors also looking to claim a stake in AveXis SMA candidate treatment.
SMA is an autosomal recessive motor neuron disease which and the leading genetic cause of infant mortality. The SMA protein is encoded by the SMN1 and SMN2 genes. Mutation of either of these genes leads to accumulation of dysfunctional SMA protein which weakens muscles and makes activities such as neck movement and breathing difficult. Currently there is no treatment for any 4 of the subtypes, with palliative care and assistive technology remaining the only option.
AveXis has therefore gained significant VC support in its development of a once-effective treatment for SMA. Using the ChariSMA technology, AveXis have developed a viral vectored transgene for healthy SMN1 gene to produce more functional SMA protein.