Sang­amo gives an­oth­er up­date on ear­ly-stage Fab­ry pro­gram, en­ters tri­al ex­pan­sion phase

As Sang­amo and Pfiz­er restart dos­ing on their Phase III he­mo­phil­ia A pro­gram, Sang­amo is al­so tick­ing off much ear­li­er-stage bench­marks on its Fab­ry gene ther­a­py.

At the Eu­ro­pean So­ci­ety of Gene and Cell Ther­a­py con­fer­ence, the Rich­mond, CA-based biotech post­ed the lat­est up­date on its gene ther­a­py for Fab­ry dis­ease, a rare X-linked con­di­tion in which pa­tients have a mu­ta­tion in the gene that codes for the al­pha-gal en­zyme, which is re­spon­si­ble for break­ing down fats and sug­ar. At the last up­date, which came in late Au­gust with a da­ta cut­off of Feb­ru­ary, Sang­amo had da­ta on six pa­tients — and up to that point, it looked like pa­tients were re­spond­ing to the gene ther­a­py.

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