CureDuchenne Ventures is happy to announce its latest research funding is an investment in PepGen, a therapeutics company targeting Duchenne muscular dystrophy and other neuromuscular diseases with next-generation antisense oligonucleotides. With the funding from CureDuchenne and a group of other investors, PepGen will advance to the clinic their Duchenne exon-skipping program, which will use a cell-penetrating peptide conjugated to phosphorodiamidate morpholino oligomers (PPMOs).
PepGen’s cell-penetrating peptide technology for oligonucleotide delivery was developed in the UK by Matthew Wood at the University of Oxford and Mike Gait at the MRC Laboratory of Molecular Biology in Cambridge. The technology they developed aims to get into muscle cells better, including cardiac muscle, to increase levels of dystrophin. Cardiomyopathy is common in Duchenne, and as individuals age they can develop cardiac failure. Therefore, a treatment that can increase dystrophin in both skeletal and cardiac muscle could have significant impact on the morbidity and mortality of the disease.