pp-151 Solution for injection
Dossier type
CTD
Dossier status
Under development
Country of origin
United States
GMP approvals
FDA
Comments
Indication: Alexander disease (AxD) — rare leukodystrophy caused by gain-of-function GFAP mutations
Clinical stage: Phase 3
Modality: Oligonucleotide (antisense oligonucleotide, intrathecal)
Mechanism / Target: Targets and silences GFAP mRNA in CNS astrocytes — reduces mutant GFAP protein accumulation (Rosenthal fibres) that causes astrocyte dysfunction and neurodegeneration
Route / form: Intrathecal injection (periodic dosing)
Differentiation: Only clinical-stage disease-modifying therapy for Alexander disease; directly addresses pathological GFAP accumulation; Ionis CNS ASO platform with established intrathecal delivery
Manufacturer #39180
Leader in RNA-targeting therapeutics with multiple approved antisense drugs. Develops GalNAc-conjugated ASOs for precise liver-targeted gene silencing in cardiometabolic and rare diseases. Wholly owned commercial and late-stage pipeline with independent global development.