Ultragenyx to spin off company to develop ‘high risk, high return’ gene therapy for Alzheimer’s disease

Ul­tragenyx plans to spin off a com­pa­ny ded­i­cat­ed to de­vel­op­ing a nov­el gene ther­a­py for Alzheimer’s dis­ease that makes use of a lyso­so­mal en­zyme, the com­pa­ny re­vealed Mon­day dur­ing its in­vestor day.

The drug­mak­er ex­pects to re­tain ma­jor­i­ty own­er­ship of the spin­off, which will be named Am­logenyx. The new com­pa­ny plans to close a Se­ries A by the end of the year.

Ul­tragenyx is known for its work in rare lyso­so­mal stor­age dis­or­ders, and the shift to a com­mon neu­rode­gen­er­a­tive dis­ease may come as a sur­prise. The com­pa­ny ex­plained in the pre­sen­ta­tion on Mon­day that a spe­cif­ic lyso­so­mal com­pound, known as pro­tec­tive pro­tein/cathep­sin A (PP­CA), was found to af­fect amy­loid be­ta in stud­ies of an­oth­er dis­ease, galac­tosiali­do­sis.

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