Rare Genetic & MetabolicKnife fightData as of 2026-09-04
Duchenne Muscular Dystrophy is one of the more crowded fields on the map: 13 companies in the clinic, ranking it 3rd of 299 indications by competitor count. The median competitor is worth $607M, below the $1.20B median of the universe.
That gap is the crowding discount. When a field is deep the market pays less per company, because the same eventual revenue has to be split across more shots on goal.
3 of the 13 are in Phase 3 or filed, and 11 dated catalysts fall in the next 12 months, so this ranking is likely to move.
$12.8B of allocated capital sits in Duchenne Muscular Dystrophy, 7th of 299 indications, and 5 of the 13 companies are pure plays that trade on this indication alone.
| Ticker | Company | Lead asset | Phase | Trials | Market cap | Pure play | Indications | Next catalyst |
|---|---|---|---|---|---|---|---|---|
| DYN | Dyne Therapeutics | Zeleciment Rostudirsen | Phase 3 | 2 | $4.7B | Yes | 1 | 25 Sep 2026 |
| SRPT | Sarepta Therapeutics | delandistrogene moxeparvovec | Phase 3 | 5 | $2.4B | No | 3 | No dated event |
| SLDB | Solid Biosciences | SGT-003 | Phase 3 | 3 | $1.1B | No | 2 | 31 Dec 2026 |
| BMRN | BioMarin Pharmaceutical | BMN 351 | Phase 2 | 2 | $12.9B | No | 5 | No dated event |
| EWTX | Edgewise Therapeutics | Sevasemten Dose 1 | Phase 2 | 2 | $4.6B | No | 2 | 31 Dec 2026 |
| WVE | Wave Life Sciences | WVE-N531 | Phase 2 | 2 | $904M | No | 3 | 31 Dec 2026 |
| RGNX | REGENXBIO | RGX-202 | Phase 2 | 1 | $607M | No | 2 | 30 Sep 2026 |
| CAPR | Capricor Therapeutics | Deramiocel | Phase 2 | 1 | $537M | Yes | 1 | 22 Nov 2026 |
| TRDA | Entrada Therapeutics | ENTR-601-44 | Phase 2 | 4 | $279M | Yes | 1 | 31 Oct 2026 |
| RNA | Atrium Therapeutics | AOC 1044 | Phase 2 | 1 | $250M | No | 3 | 31 Dec 2026 |
| KROS | Keros Therapeutics | KER-065 | Phase 2 | 1 | $220M | Yes | 1 | 30 Jun 2027 |
| MSLE | Satellos Bioscience | SAT-3247 | Phase 2 | 2 | $189M | Yes | 1 | 31 Dec 2026 |
| DTIL | Precision BioSciences | PBGENE-DMD | Phase 1 | 1 | $203M | No | 16 | 31 Dec 2026 |
Sorted by phase, then by market capitalisation. Ticker links open the company profile. BMRN holds 45% of the $28.8B competing in this indication.
Crowded and cheaply priced. Six or more competitors and a median company below the universe median.
Rare Genetic & Metabolic holds $103B of the map's capital, 15.3% of the total, spread across 32 indications and 48 companies. 10 of those indications have more than one company competing.
See every Rare Genetic & Metabolic indication on the map13 US-listed companies in the $100M to $20B market cap band run clinical programmes in Duchenne Muscular Dystrophy. The largest and most advanced are Dyne Therapeutics (DYN, Zeleciment Rostudirsen, Phase 3); Sarepta Therapeutics (SRPT, delandistrogene moxeparvovec, Phase 3); Solid Biosciences (SLDB, SGT-003, Phase 3); BioMarin Pharmaceutical (BMRN, BMN 351, Phase 2); Edgewise Therapeutics (EWTX, Sevasemten Dose 1, Phase 2). The full list of 13 is in the table on this page.
Of the 13 clinical-stage programmes in Duchenne Muscular Dystrophy, 3 are in Phase 3 or filed, 9 are in mid-stage development and 1 is in early-stage development.
The median company developing a drug for Duchenne Muscular Dystrophy is worth $607M, against a median of $1.20B across the whole universe. $12.8B of allocated capital sits in the indication, ranking it 7th of 299.
The next dated catalyst in Duchenne Muscular Dystrophy is DYN on 25 Sep 2026. 11 dated events across the field fall in the next 12 months.
Allocated capital divides each company’s market capitalisation evenly across the indications it is running clinical programmes in, so every dollar of the universe is counted exactly once. Total market capitalisation competing counts a company in each of its indications and therefore sums to more than the universe.
Third-party market size estimates are not used anywhere on this page. Approved incumbents are excluded from competitor counts, and companies above $20B are outside the universe by construction.
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