Asset profile
BMN 351
RNA / oligonucleotide
Evidence outlook
Early-stage evidence only
Phase 1 / first-in-human studies establish safety, tolerability, and dosing; they are not designed to demonstrate efficacy, so no evidence signal is assigned yet. The first graded signal arrives with a completed confirmatory readout.
How the evidence outlook works ↗
Featured trials
The studies most instrumental to this drug's clinical progression, individually graded on design and result. Pick an indication; Research unlocks the trials, endpoints, key results and grades.
Competitive landscape
3 competitors in this universe: 0 direct rivals (same mechanism and indication), 0 same-class, 3 same-indication; 2 further along in development.
| Drug | Stage | Modality | Mechanism | Evidence signal | Reported efficacygraded featured trials |
|---|---|---|---|---|---|
| BMN 351: this drug | Phase 1/2 | RNA / oligonucleotide | — | ||
| Deramiocel (CAP-1002) | Phase 3 | Cell therapy | Cardiac cell therapy | ||
| RGX-202 | Phase 2/3 | Gene therapy / editing | — | ||
| WVE-N531 | Phase 1/2 | RNA / oligonucleotide | — |
Unlock the full comparison
Research adds each competitor's evidence grade and sourced trial results, aligned per race, so you can see who actually has the data.
Upcoming events
(2 total)
Estimated primary-completion dates from ClinicalTrials.gov. These are registry estimates, never confirmed readout dates; they can move as trials progress.
2027 (1)
April 2027 (1)
What to watch
2031 (1)
December 2031 (1)
What to watch
All registered studies
Every ClinicalTrials.gov study of this drug's own program, grouped by indication and phase. Research unlocks the studies themselves: titles, statuses, dates and featured-trial cross-links.