Genen­tech takes aim at Bio­gen and No­var­tis, high­light­ing an ear­ly look at pos­i­tive re­sults for their oral SMA drug from PTC

Af­ter get­ting hit with a set­back that forced the phar­ma gi­ant Roche to aban­don its lead drug for spinal mus­cu­lar at­ro­phy, com­pa­ny ex­ecs are back with a new drug in-li­censed from PTC $PTCT that they be­lieve has legs.

It’s an in­ter­im, up­dat­ed look at a tiny da­ta set for ris­diplam (or RG7916), but re­searchers spot­light­ed ev­i­dence that the drug could help sig­nif­i­cant per­cent­ages of in­fants sit, kick and con­trol their head move­ments — all el­e­ments that the dis­ease de­stroys as it re­lent­less­ly push­es these chil­dren to an ear­ly death.

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Senior Associate

Alexandria Real Estate Equities

Durham, NC, USA