Headquartered in Boston, MA·Fiscal year ends December·NASDAQ: PEPG·pepgen.com
About PepGen
PepGen Inc. is a clinical-stage biotechnology company developing oligonucleotide therapies for rare genetic diseases. It has no product revenue.
Its proprietary Enhanced Delivery Oligonucleotide (EDO) technology uses cell-penetrating peptides to improve the cellular uptake, endosomal escape and nuclear delivery of conjugated oligonucleotides. The company says the approach gives a 98-fold increase in nuclear delivery and reaches a broad range of tissues independent of receptors.
Its lead and sole disclosed clinical candidate is PGN-EDODM1, for myotonic dystrophy type 1 (DM1). DM1 is a progressive, multisystemic genetic disease estimated to affect more than 110,000 people in the U.S. and EU, and no approved therapy addresses its underlying cause. PGN-EDODM1 binds expanded CUG repeats in pathogenic DMPK RNA to free MBNL1 and restore normal RNA splicing, without degrading DMPK RNA.
The candidate holds FDA Orphan Drug and Fast Track designations and EMA Orphan Designation. It is being evaluated in the Phase 2 FREEDOM2-DM1 multiple ascending dose trial in Canada, the UK, New Zealand, Australia and South Korea, and in the FREEDOM-OLE open-label extension. The FREEDOM2 study is under an FDA partial clinical hold in the U.S. Updated
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