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.

Source: Spinal Muscular Atrophy Foundation (SMAF)

Source: Spinal Muscular Atrophy Foundation (SMAF)

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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. It makes sense that expression at the genomic level provides a long-term solution for SMA, and is therefore the key approach being taken in the biotech race to produce the first SMA treatment ever.

Roche has shown particular enthusiasm to be a part of this groundbreaking research. In November 2011, Roche entered into a €437M partnership with New Jersey (US) PTC Therapeutics to gain worldwide exclusivity to the SMA RG7800 program alongside the Spinal Muscular Atrophy Foundation (SMAF). The PTC program is looking into improved methods of splicing the SMN2 gene to also increase functional SMA protein production. RG7800 is about to enroll on a Ib/IIa study in adult and pediatric patients, which will be overseen by a joint committee of Roche, PTC and SMAF.

In January of this year, Roche also bought Marseille (Fr) based Trophos for €486M. Research at Trophos is also SMA-centric with development of their olesoxime (TRO19622) screening platform for SMA treatment, which has the orphan drug designation for both the EU and US markets.

This is particularly significant considering this status will help Roche position itself as a market leader for potential SMA treatments, whichever biotech produces them first.

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