September 4, 2024
Advancing the Use of Peptide-Conjugated Oligonucleotides to Target Neuromuscular Disorders: Enhanced Delivery Oligonucleotides for DMD and DM1
April 9, 2024
FREEDOM-DM1: Phase 1 Study Design to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PGN-EDODM1 for Myotonic Dystrophy Type 1
March 3, 2024
PGN-EDODM1: Single- and Repeat-Dose Nonclinical Data Indicated Mechanistic and Meaningful Activity for Potential Treatment of Myotonic Dystrophy Type 1 (DM1)
March 3, 2024
PGN-EDO51: Single- and Repeat-Dose Nonclinical Data Demonstrated Potential for the Treatment of Duchenne Muscular Dystrophy (DMD)
March 3, 2024
FREEDOM-DM1: Phase 1 Study Design to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PGN-EDODM1 for Myotonic Dystrophy Type 1
Summary: The Enhanced Delivery Oligonucleotide (EDO) platform is engineered to optimize the tissue penetration, cellular uptake and nuclear delivery of oligonucleotide therapeutic candidates.
March 3, 2024
CONNECT1-EDO51 and CONNECT2-EDO51: Phase 2 Study Designs to Evaluate Safety and Efficacy for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
November 6, 2023
FREEDOM-DM1: Nonclinical Data Support the Phase 1 Study Design to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PGN-EDODM1 in Adults with Myotonic Dystrophy Type 1 (DM1)
Summary: The Enhanced Delivery Oligonucleotide (EDO) platform is engineered to optimize the tissue penetration, cellular uptake and nuclear delivery of oligonucleotide therapeutic candidates.
November 6, 2023
PGN-EDODM1 Nonclinical Data Demonstrated Mechanistic and Meaningful Activity for the Potential Treatment of Myotonic Dystrophy Type 1 (DM1)
Summary: The Enhanced Delivery Oligonucleotide (EDO) platform is engineered to optimize the tissue penetration, cellular uptake and nuclear delivery of oligonucleotide therapeutic candidates.
November 6, 2023
Nonclinical Data Demonstrate the Potential of the Enhanced Delivery Oligonucleotide (EDO) PGN-EDO51 for the Treatment of Duchenne Muscular Dystrophy
Summary: PGN-EDO51 is PepGenās clinical-stage EDO candidate for the treatment of people with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
October 3, 2023
Three Novel Enhanced Delivery Oligonucleotide Candidates for Duchenne Muscular Dystrophy Mediate High Levels of Exon 53, 45, and 44 Skipping
September 6, 2023
PGN-EDODM1 Nonclinical Data Demonstrate Mechanistic and Meaningful Activity for Potential Treatment of Myotonic Dystrophy Type 1
Summary: The Enhanced Delivery Oligonucleotide (EDO) platform is engineered to optimize the tissue penetration, cellular uptake and nuclear delivery of oligonucleotide therapeutic candidates
March 22, 2023