Skip to Content

Community

Fostering impactful
connections

I’ve learned over the years—a lot of what I do to manage my health works. There’s no way I’m going to stop the progression, but I’ve proven to myself that I can mitigate some of the symptoms through diet, exercise, and attitude.

Leslie, California
DM1 support group and podcast host, living with DM1 quotes
patient-Leslie

We have therapy together every week. We call it ‘couples’ therapy. We are basically a package deal. We live very well together. ~ CHAD

He needs me. ~ MICHAEL

Chad & Michael, Utah
Brothers living with DM1 quotes
patient-Chad & Michael

The worst part of this whole thing is how it passes down to the next generation.

Jubal, North Carolina
Retired University Writing and Literature professor, advocate, and father living with DM1 quotes
patient-Jubal

Living with Duchenne puts you in front of your own mortality. You’re kind of given a list of things that become impossible. Not that they necessarily do, but that’s the way it seems.

Mallory, Texas
Pursuing her Master of Social Work degree while living with DMD quotes
patient-Mallory

The power of community is an overarching theme in my life. Even though I’m unable to walk, I’m still able to get out and enjoy life and enjoy my community.

Colin, Virginia
Web designer and community advocate living with DMD quotes
patient-Colin

If I didn’t have a disability, I would just be normal. I would walk around and be normal, like other people, but I’m not. I’m different. And it’s ok if you’re different. Everybody is the same.

David, New Hampshire
Highschooler and avid road tripper living with DMD quotes
patient-David

PepGen and the
Neuromuscular
Disorder Community

The patient advocacy team at PepGen ensures PepGen employees understand the goals, preferences, and unmet needs of the neuromuscular disorder communities we serve. We partner with the community to gain insights that help inform our clinical trial designs; the preferred methods, styles, and frequency of communications; and how our clinical development plans meet the needs of the community.

Community advisory boards, participating with advocacy groups in conferences and awareness activities, and taking part in advocacy-led consortia and working groups enable us to engage and learn from the community.

Contact the PepGen Patient
Advocacy Team

We are continually looking to build new relationships with advocacy groups and members of the neuromuscular disorder community. If you’d like to partner with us or have any questions, contact community@pepgen.com.

Community Partners

Community
Community
Community
Community
Community
Community
Community
Community
Community
Community
Community
Community
Community
Community
Community
Community
Community
Community

To learn about partnering with PepGen’s community team, please email us at community@pepgen.com.

Duchenne Muscular Dystrophy (DMD)

PepGen is committed to transforming the treatment for people living with Duchenne muscular dystrophy (DMD) with our Enhanced Delivery Oligonucleotides (EDOs).

Oligonucleotides are genetic medicines that have the potential to modify the root cause of numerous neuromuscular disorders. Oligonucleotides for the treatment of DMD include exon skipping drugs. Some countries have approved exon skipping drugs for the treatment of DMD.

However, oligonucleotides, on their own, do not enter muscles easily, which limits their potential efficacy. For this reason, PepGen has developed peptides that can be conjugated (connected) to oligonucleotides, to enhance and improve their delivery and therefore their activity.

Preclinical research of our EDOs showed high levels of dystrophin production in animals.

In our Phase 1 clinical study in healthy volunteers, we observed high levels of oligonucleotides and exon skipping in muscle.

Most recently, we reported encouraging initial results from the low dose cohort of our CONNECT1-EDO51 Phase 2 trial. The results support that PGN-EDO51 delivered oligonucleotides to the nucleus of muscle cells – the site of action.

Learn how our therapy works.

See our FAQ for additional resources and information.

DMD Community FAQs

Myotonic Dystrophy Type 1 (DM1)

PepGen is committed to transforming the treatment for people living with myotonic dystrophy type 1 (DM1) with our Enhanced Delivery Oligonucleotides (EDOs). 

Oligonucleotides are genetic medicines that have the potential to modify the root cause of numerous neuromuscular disorders. They have been studied for multiple decades, however, by themselves, they do not enter into muscles easily and effectively.

PepGen has developed peptides that can be conjugated (connected) to oligonucleotides, to enhance and improve the delivery and activity of the oligonucleotides.

Recent pre-clinical research data support clinical studies of EDOs in people living with DM1.

Watch our approach here

See our FAQ for additional resources and information.

DM1 Community FAQs

Events

September 21-22

DMD China (Virtual)

NOVEMBER 3-5, 2023

Defeat Duchenne Canada 2023 Family Forum Weekend

Associate Director of Patient Advocacy, Alayna Tress presenting on the Research & Drug Development Update Panel
APRIL 21 – APRIL 23, 2023

CureDuchenne Futures Conference

VP of Clinical Development and Patient Advocacy, Jane Larkindale D. Phil, to present on PepGen’s DMD program.
MARCH 19 – MARCH 23, 2023

Muscular Dystrophy Association Conference