On the cusp of PhI, An­gel­man-fo­cused biotech scores po­ten­tial buy­out deal from Ul­tragenyx

Ul­tragenyx has found a new rare dis­ease to de­vote it­self to, rop­ing in a young up­start as it be­gins a long trek to a new treat­ment for An­gel­man syn­drome.

Their new part­ners at GeneTx is work­ing to­ward an IND for GTX-102, an an­ti­sense oligonu­cleotide de­signed to in­hib­it the UBE3A-AS DNA strand and there­by turn on an al­lele in neu­rons that pro­duces a key pro­tein miss­ing in pa­tients with An­gel­man.

The col­lab­o­ra­tion — and po­ten­tial ac­qui­si­tion — would pit Ul­tragenyx against Je­re­my Levin’s team at Ovid, which has show­cased some con­tro­ver­sial Phase II da­ta last year for its ex­trasy­nap­tic GABAA re­cep­tor ag­o­nist. An­gel­man is al­so among the slate of neu­ro­log­i­cal tar­gets that Bio­gen and Io­n­is are tack­ling in an an­ti­sense deal.

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