PepGen, a company founded in 2018 by scientists at the University of Oxford and Medical Research Laboratory in Cambridge, announced the closing of a $112.5 million crossover financing, which the company intends to use to advance its pipeline of Enhanced Delivery Oligonucleotide (EDO) therapies for neuromuscular and neurologic diseases into clinical trials.
The funds will also enable further buildout of PepGen’s rapidly growing team at their headquarters in Boston, MA and support research efforts at their laboratory in Oxford, UK.
PepGen’s lead program targets the underlying cause of Duchenne muscular dystrophy (DMD), a rare genetic disease that presently has no cure and affects approximately 250,000 people in the U.S. and 1 in every 3,500 males globally. People with DMD experience increasing muscle weakness starting at birth that leads to difficulties standing, walking and breathing, ultimately shortening their lives with the average patient passing away during their teen years.
PepGen’s academic founders spent over a decade perfecting their EDO platform at institutions in the UK, leveraging their scientific expertise to enhance delivery of oligonucleotides into muscle and ultimately increase levels of dystrophin, a protein responsible for strengthening and protecting muscles. Previous treatments have struggled to penetrate muscle tissue, and as a result, have failed to dramatically improve the condition.
Preclinical studies show that PepGen’s therapy can lead to higher levels of dystrophin production at lower doses than existing DMD therapies.
James McArthur, Ph.D., President and Chief Executive Officer of PepGen, said: “We have shown in large animal studies that we can safely achieve
industry-leading efficacy in exon 51 skipping, and we hope this will be transformative for DMD patients. We will build on this work to expand our DMD program and into other neuromuscular diseases, leveraging the enormous potential of our EDO platform technology to create a better future for people living with genetic diseases.”