Dyne Therapeutics, Inc. DYN
Dyne Therapeutics, Inc. (Nasdaq: DYN) is a rare genetic drug developer. Its lead program, zeleciment rostudirsen (z-rostudirsen / DYNE-251), is in Phase 3 for Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. Its next catalyst on record is z-rostudirsen: PDUFA target action date (January 2027).
Upcoming catalysts
- January 2027z-rostudirsen — PDUFA target action date
The FDA has granted the BLA priority review and assigned a Prescription Drug User Fee Act, or PDUFA, target action date of January 21, 2027.
Next further out: z-rostudirsen — Potential U.S. launch (Q1 2027). 4 catalysts on record beyond the next 30 days; subscribers see each with its filing sentence.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| zeleciment rostudirsen (z-rostudirsen / DYNE-251) | DMD amenable to exon 51 skipping | oligonucleotide-other | Phase 3 |
| zeleciment basivarsen (z-basivarsen / DYNE-101) | Myotonic dystrophy type 1 (DM1) | RNA (siRNA/ASO/mRNA) | Phase 3 |
| DYNE-302 | Facioscapulohumeral dystrophy (FSHD) | RNA (siRNA/ASO/mRNA) | Phase 1 |
| DYNE-244 | DMD amenable to exon 44 skipping | oligonucleotide-other | Pre-clinical |
| DYNE-245 | DMD amenable to exon 45 skipping | oligonucleotide-other | Pre-clinical |
| DYNE-253 | DMD amenable to exon 53 skipping | oligonucleotide-other | Pre-clinical |
| DYNE-255 | DMD amenable to exon 55 skipping | oligonucleotide-other | Pre-clinical |
| DYNE-401 | Pompe disease | protein/peptide | Pre-clinical |
| Conjugate 1 | MAPT-related neurological indication | RNA (siRNA/ASO/mRNA) | Pre-clinical |
| Conjugate 2 | MAPT-related neurological indication | RNA (siRNA/ASO/mRNA) | Pre-clinical |
Recent results and decisions
- Nov 5, 2026submitted BLA for z-rostudirsen in exon 51 DMD is under priority review by FDA with PDUFA target action date of January 21, 2027.
Biologics License Application (BLA) for z-rostudirsen in exon 51 Duchenne muscular dystrophy (DMD) under priority review by FDA; PDUFA target action date of January 21, 2027
Filing - Nov 5, 2026positive Additional one-year data from Phase 1/2 ACHIEVE trial of z-basivarsen in DM1 continued to support improvement in video hand opening time at 6 months as an early indicator of clinical benefit.
Additional one-year clinical data from Phase 1/2 ACHIEVE trial of z-basivarsen for myotonic dystrophy type 1 (DM1) presented at WMS and AANEM continued to support improvement at 6 months in video hand opening time (vHOT) as an early indicator of clinical benefit
Filing - Sep 29, 2026positive Pooled-dose (N=25-26) ACHIEVE MAD data at 12 months: functional improvement vs baseline, placebo and matched natural history; vHOT fell 3.2 sec from 8.2 sec baseline at 6 months vs +0.4 sec on placebo (3.6 sec relative).
Data from a pooled dose group of participants initially enrolled in the multiple ascending dose (MAD) portion of ACHIEVE showed functional improvement versus baseline, versus placebo and versus a matched natural history cohort across multiple measures at 12 months
Filing
7 more on record.
For subscribers
- Cash runway against each catalyst, with the company's own runway statement
- Track record: every Phase 3 readout, FDA decision and guidance slip since 2014
- Expected move for each dated catalyst, from comparable past events
- Phase 2 evidence behind each Phase 3 readout
- Companies on the same target or class
- Full catalyst list with filing sentences, deals, registered trials, Excel export
Other phase 3 rare genetic companies
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.