Onasemnogene abeparvovec
Phase 3Gene therapy
NovartisOnasemnogene abeparvovec is a gene therapy that works as a SMN1 Gene Therapy.[2] It is being investigated across 2 indications, with its most advanced program in Phase 3.[1]
Onasemnogene abeparvovec is one of 13 gene therapy programs targeting Spinal Muscular Atrophy (SMA). It is currently in Phase 3.
2
Indications
9
Total Trials
3
Active Trials
12
Competitors
Pipeline
Status by Indication[3][1]
Competition
Competitive Landscape
14 competitors in Spinal Muscular Atrophy
#DrugCompanyPhaseTrials
1Spinraza
BiogenApproved
Evrysdi
RocheApproved
Prilosec
DDexcel Pharma Technologies Ltd.
Approved
Amondys 45
Sarepta TherapeuticsApproved
Salanersen
BiogenPhase 3
Onasemnogene abeparvovec
NovartisPhase 3
Apitegromab
SScholar Rock, Inc.
Phase 3
Taldefgrobep Alfa
BBiohaven Therapeutics Ltd.
Phase 3
Vesemnogene Lantuparvovec
LLantu Biopharma
Phase 3
NMD670
NNMD Pharma A/S
Phase 2
Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.
Timeline
Expected Milestones
Clinical
Trials by Indication[1]
Spinal Muscular Atrophy7 trials
Sources
References
- [1]
- [2]
- [3]Open Targets Platform — Drug–disease associations for Onasemnogene abeparvovec. EMBL-EBI & Wellcome Sanger Institute.
- [4]
- [5]