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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