sepofarsen
Phase 2- Indications
- Leber congenital amaurosis 10
Company intelligence / Profile preview
Sepul Bio
Likely France or UK given parent company location and trial sites globally including USA/UK[1][5]sepulbio.com ↗
- Ownership
- Private
- Employees
- ~1
- Development stage
- Phase 2
01
Overview
Sepofarsen is an investigational RNA therapy designed to correct aberrant mRNA splicing caused by CEP290 mutation in LCA10. Ultevursen is an antisense oligonucleotide targeting exon13 mutations in USH2A gene to treat vision loss from Usher syndrome type II and related retinitis pigmentosa. Ultevursen has entered Phase 2b randomized sham-controlled trial named LUNA enrolling adults and children globally. Early-stage trials showed encouraging efficacy signals[1][5][6].
- Therapeutic areas
- Rare DiseasesOphthalmology
- Modalities
- RNA-based therapiesAntisense oligonucleotides (AONs)
- Industry
- Biotech
02
Drug pipeline
2 assets1 more asset in the full pipeline.
Request access →03
Partnerships
Originally developed assets acquired from ProQR; collaboration implied through asset acquisitionRetina UK supporting awareness[7]
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