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PepGen

PEPG Rare disease Lead: Phase 2 1 Biotech Distilled article ↓

Neurology Nucleic Acid

Most recent annual filing: 2026-03-04

Pipeline (1 active programs)

PGN-EDODM1

Nucleic Acid Target: DMPK mRNA, CUG repeat expansion, MBNL1

Myotonic Dystrophy Type 1 Phase 2 intravenous

Technology platforms

EDO

Enhanced Delivery Oligonucleotide platform leveraging cell-penetrating peptides (CPPs) conjugated to oligonucleotide therapeutics (PPMOs) to optimize tissue penetration, cellular uptake, and nuclear delivery; focus on neuromuscular and neurologic diseases including myotonic dystrophy type 1

Biotech Distilled coverage (1)

2026-05-29 PepGen (PEPG) — Scientific Deep Dive for PGN-EDODM1 and the EDO Platform 🔒 Paid

Articles open on Substack. Some deep dives are subscriber-only.