Rare dis­ease drug­mak­er Ul­tragenyx builds case for its gene ther­a­py fran­chise with fresh tri­al up­dates

Fo­cused on com­bat­ing a smat­ter­ing of rare dis­eases with an ar­se­nal of bi­o­log­ics, small mol­e­cules, and gene ther­a­pies, Ul­tragenyx Phar­ma­ceu­ti­cal this week is­sued some up­dates on two of its ex­per­i­men­tal gene ther­a­pies as it works on build­ing a fran­chise for one-time cures.

On Fri­day, the com­pa­ny un­veiled ear­ly da­ta on the ef­fect of DTX401, its gene ther­a­py un­der in­ves­ti­ga­tion, in pa­tients with glyco­gen stor­age dis­ease type Ia (GS­DIa), a dis­ease that af­fects rough­ly 6,000 peo­ple glob­al­ly and is char­ac­ter­ized by the in­abil­i­ty to reg­u­late blood sug­ar.

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