Ultragenyx Enters Duchenne Gene Therapy Space In $295m Deal With Solid Biosciences

Ultragenyx enters a competitive and complicated market, but the combination of its manufacturing platform with Solid’s transgene could provide an advantage. 

Handshake of businessmen

Ultragenyx Pharmaceutical Inc. is wading into a complex and competitive area of gene therapy in a partnering deal with Solid Biosciences Inc. centered on Duchenne muscular dystrophy (DMD), but the firms are hoping that Ultragenyx’s manufacturing platform will give it an advantage over other DMD gene therapy programs.

The two companies announced the nearly $300m partnership and licensing deal 23 October, whereby Ultragenyx will contribute its HeLa producer cell line and adeno-associated virus 8 (AAV8) variants, receiving an exclusive license for any

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