Mechanism

Phosphorodiamidate morpholino (PMO) ASO that blocks Element 1 (E1), an intronic repressor in SMN2 pre-mRNA, de-repressing exon 7 inclusion. Mechanism distinct from nusinersen (which blocks 3' splice site).

Approved indications

Related claims (0)

No claims matched “e1v1.11” in free-text search.

Off-Target Findings (0)

No Boltz-2 / Chai-1 off-target panel claims recorded for this drug.