At PepGen, we are committed to advancing our next-generation oligonucleotide therapies to change the lives of those living with neuromuscular and neurological diseases. Our lead programs are in Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1). We are committed to working with the community—individuals, families and global advocacy groups—to discover and develop medicines that make a difference.
While our current focus is on developing transformative treatments for DMD and DM1, we are expanding to a broad range of other disease areas. Visit our pipeline page to learn more and feel free to reach out to us if you have questions.
Duchenne muscular dystrophy (DMD) is a rare genetic disease characterized by progressive muscle weakness, which leads to difficulties walking, loss of upper body function and difficulties breathing, ultimately impeding daily function and long-term survival. Eventually, heart and breathing problems lead to serious life-threatening complications.
DMD predominantly affects males and is caused by a genetic mutation on the X chromosome that leads to reduced levels of dystrophin, a protein critical to healthy muscle function. Despite significant advances in treatments for DMD, current therapies are limited by poor delivery to muscle tissue and have limited effectiveness in delaying disease progression.
Our first product candidate is PGN-EDO51, an EDO peptide conjugated to an oligonucleotide therapeutic for the treatment of individuals living with DMD who are amenable to an exon 51-skipping approach. Learn more about our approach.
We are initiating clinical trials for PGN-EDO51 in 2022 and intend to expand our pipeline of oligonucleotide therapeutics for the treatment of other DMD exon skipping populations. See additional resources and information on current ongoing research below.
Myotonic dystrophy type 1 (DM1) is a progressive multisystemic disease that causes myotonia, or difficulty relaxing muscles, progressive muscle weakness and often other symptoms such as cataracts, heart conduction defects, endocrine changes including diabetes, cognitive issues and daytime sleepiness. Symptoms and onset present uniquely to each person affected and vary from minor muscle pain to serious respiratory and cardiac issues.
DM1 is caused by toxic repeat sequences in the RNA that bind to a protein that is needed to correctly process a wide range of other RNAs. Misprocessing of RNAs leads to the wide range of symptoms. Current treatments, such as heart medications and medications to treat cramping, can help people with DM1 manage symptoms, but there are currently no approved disease-modifying treatments.
We are developing PGN-EDODM1, an EDO peptide conjugated to a therapeutic oligonucleotide, for the treatment of DM1. Learn more about our approach.
We are initiating clinical trials for PGN-EDODM1 in 2023 and intend to expand our pipeline of oligonucleotide therapeutics for the treatment of other DM1 patient populations. See additional resources and information on current ongoing research below.
PepGen has no current events scheduled at this time. This section will be updated with upcoming events soon.
Parent Project Muscular Dystrophy Annual Conference
Head and SVP of Clinical Development, Michelle Mellion MD, to present on PepGen’s DMD program
MDA Clinical & Scientific Conference
CEO Dr. James McArthur and VP of Clinical Science Dr. Jane Larkindale presented abstract, “Unlocking the potential of oligonucleotide therapeutics for Duchenne muscular dystrophy through enhanced delivery”
Thriving with Duchenne: A Rare Disease Day Panel Discussion with Jett Foundation
PepGen attended and sponsored event
Learn More About the PepGen Community
Meet Jane Larkindale, VP of Clinical Science
Do you have questions about PepGen’s approach, our technology or our current advocacy partners? Contact our Vice President of Clinical Science, Jane Larkindale, at email@example.com.
We are continually looking to build new relationships with advocacy groups in the neuromuscular disease community. Contact firstname.lastname@example.org if you’d like to partner with us.