myotonic dystrophy Type 1

plan B fail reroute road path trail miss
Biotech

PepGen ends all DMD work after phase 2 trial falls flat

PepGen’s exon 51-skipping oligonucleotide PGN-EDO51 only increased dystrophin to 0.59% of normal levels in four patients in the 10-mg/kg cohort.
Darren Incorvaia May 28, 2025 5:30pm
Desert sand dunes

RNA-targeting drug relaxes muscles in myotonic dystrophy mice

Dec 4, 2024 7:00am
Novartis

Novartis signs $1.1B deal for muscular dystrophy biotech

Nov 21, 2024 8:19am
steps race jump stairs climb

PepGen gains a little pep in its step as FDA clears trial hold

Oct 12, 2023 7:00am
Letter opening FDA clinical hold

Awaiting FDA letter, PepGen oligonucleotide no closer to clinic

Jun 14, 2023 9:05am
Stop sign

FDA blocks PepGen's attempt to get 2nd oligonucleotide to clinic

May 31, 2023 8:11am