Doug Ingram, Sarepta CEO (Sarepta Therapeutics)

Sarep­ta re­veals pos­i­tive re­sults in limb-gir­dle gene ther­a­py tri­al. What does that mean for DMD?

Sarep­ta on Mon­day un­veiled a small batch of pos­i­tive da­ta for one of the gene ther­a­pies it bought out last year, sketch­ing a path to­ward a piv­otal study in 2021 and help­ing bol­ster the com­pa­ny’s broad­er gene ther­a­py am­bi­tions.

The gene ther­a­py, known as SRP-9003, is de­signed to treat a rare de­gen­er­a­tive dis­or­der called limb-gir­dle mus­cu­lar dy­s­tro­phy by boost­ing pro­duc­tion of a pro­tein pa­tients are miss­ing, called be­ta-sarco­gly­can. It was part of a bas­ket of treat­ments Sarep­ta ac­quired in its $165 mil­lion-buy­out of My­onexus last year. The biotech al­ready showed a low dose of the ther­a­py could lead to some in­creased ex­pres­sion of the miss­ing pro­tein and some im­proved mus­cle func­tion in 3 kids, but it was un­clear how long those re­sults would last or if a high­er dose would lead to bet­ter re­sults.

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