Dyne Therapeutics, Inc. DYN
Dyne Therapeutics, Inc. (Nasdaq: DYN) is a rare genetic drug developer. Its lead program, z-rostudirsen (DYNE-251), is in Phase 3 for Duchenne muscular dystrophy (exon 51 skipping). Its next catalyst on record is z-rostudirsen: FDA action on BLA (PDUFA target date) (January 21, 2027).
Upcoming catalysts
- January 21, 2027z-rostudirsen — FDA action on BLA (PDUFA target 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 |
|---|---|---|---|
| z-rostudirsen (DYNE-251) | DMD exon 51 | oligonucleotide-other | BLA PDUFA date 2027-01-21 |
| z-basivarsen (DYNE-101) | Myotonic dystrophy type 1 | oligonucleotide-other | REC data 2027-Q1; BLA 2027-Q3 |
| DYNE-302 | FSHD | oligonucleotide-other | Phase 1 planned after IND clearance (Jul 2026) |
| DYNE-401 | Pompe disease | biologic-other | — |
| DYNE-253/245/244/255 | DMD exons 53/45/44/55 | oligonucleotide-other | — |
Recent results and decisions
- 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 - Jul 29, 2026started Dosing began in the Phase 3 HARMONIA trial in July 2026.
Dyne began dosing participants in the global confirmatory Phase 3 HARMONIA trial of z-basivarsen in July 2026.
Filing - Jul 29, 2026accepted FDA accepted the z-rostudirsen BLA (accelerated approval) with Priority Review; PDUFA date January 21, 2027.
Dyne’s BLA was accepted for review by the FDA in July 2026, with Priority Review granted (Prescription Drug User Fee Act (PDUFA) target action date set for January 21, 2027).
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.