Neuromuscular · Rare
Updated August 2026 · DMD (dystrophin, Xp21)
By phase
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| Asset | Sponsor | Phase | Modality | Population | Signal |
|---|---|---|---|---|---|
| RGX-202 Micro-dystrophin (AAV8, novel construct) | REGENXBIO | Phase 3 | Gene therapy | Ambulatory DMD, age-gated pivotal | Early data did not clearly beat Elevidys expression; pivotal is the test. |
| SGT-003 Micro-dystrophin (next-gen capsid) | Solid Biosciences | Phase 2 | Gene therapy | Ambulatory, expanding | Reported ~110% micro-dystrophin of normal in early cohorts — watch safety at that expression. |
| del-zota (AOC 1044) Antibody-oligo conjugate, exon 44 | Avidity → Novartis (announced) | Filed / review | ASO / exon skip | Exon 44 skip-amenable | Delivery thesis: AOC should beat naked PMO on dystrophin. Strategic: Novartis ~$12B Avidity bid. |
| Deramiocel (CAP-1002) Cardiosphere-derived cells — cardiomyopathy | Capricor | Filed / review | Cell therapy | DMD cardiomyopathy | Only late-stage asset aimed at the heart. Binary for the cardiac white space. |
| z-rostudirsen (DYNE-251) Fab-oligo, exon 51 | Dyne Therapeutics | Phase 2 | ASO / exon skip | Exon 51 skip-amenable | Must show a step-change vs Exondys to justify conversion. |
| Sevasemten (EDG-5506) Fast skeletal myosin inhibitor — contraction stress | Edgewise | Phase 2 | Small molecule | Becker and DMD (protect muscle) | Mutation-agnostic muscle-protection thesis. Complementary, not competitive with GT. |
| GNT0004 AAV micro-dystrophin | Genethon | Phase 1 | Gene therapy | Paediatric ambulatory | 2026–27 |
| INS1201 AAV micro-dystrophin | Insmed Gene Therapy | Phase 1 | Gene therapy | Early DMD | Early |
| fordadistrogene movaparvovec Mini-dystrophin AAV9 | Pfizer | Discontinued | Gene therapy | CIFFREO (ambulatory) | The other GT casualty. Relevant as a PoS prior for the class. |