Z-rostudirsen (DYNE-251)Dyne Therapeutics—FDA accepts z-rostudirsen BLA with Priority Review; PDUFA January 21, 2027Z-rostudirsen (DYNE-251)Dyne Therapeutics—Dyne submits z-rostudirsen BLA for Duchenne exon 51

Dyne Therapeutics

Exon-skipping, Duchenne, neuromuscular

USpublicest. 2017dyne-tx.com (opens dyne-tx.com)Checked 11 Oct

The brief

Analyst view
  • Dyne raised $267.7M in September 2020 IPO at $19/share.
  • Z-rostudirsen BLA accepted with Priority Review; PDUFA date January 21, 2027.
  • Pipeline includes four additional exon-skipping candidates for other Duchenne mutations.

Technical approach

As reported
Fab-guided delivery

Antibody-coupled oligonucleotide

Z-rostudirsen couples an exon-skipping oligo to a muscle-targeting antibody Fab, enabling efficient delivery.

Exon skipping

Restore reading frame

Skipping the mutated exon restores the reading frame so muscle cells produce partial dystrophin.

FORCE platform

Modular targeted delivery

The proprietary platform couples oligonucleotides with antibody fragments for broader applications.

Primer