🎉 Exciting News from PepGen! 🎉
PepGen has announced positive data from the low-dose cohort of their PGN-EDO51 in the ongoing Phase 2 CONNECT1-EDO51 clinical trial for Duchenne Muscular Dystrophy (DMD)!
🔬 Highlights 🔬
🧬Well Tolerated: PGN-EDO51 at 5 mg/kg was well tolerated, with all patients continuing to the long-term extension phase.
🧬Exon Skipping: Achieved mean exon skipping levels of 2.15% after just 3 months.
🧬Dystrophin Production: A significant increase in muscle-adjusted dystrophin levels, with a mean level of 1.49%, a 0.70% rise from baseline.
🧬Safety: Demonstrated a favorable safety profile, supporting ongoing evaluations.
🧬Their Enhanced Delivery Oligonucleotide (EDO) technology is proving to be a game-changer, delivering higher levels of oligonucleotide to nuclei, indicating stronger potency at lower doses.
James McArthur, CEO, stated, "We are encouraged by the early data, which shows PGN-EDO51 produced higher levels of exon skipped transcript at lower doses and in a shorter time period compared to other therapies."
#Biotech #DMD #ClinicalTrials #Innovation #PepGen #Healthcare #MedicalResearch #Pharma
Biotech and cell and gene therapy industry commercial leader
1moFantastic news; I’m still watching closely Alex Bloom