What Pepgen does

PepGen is a clinical-stage biotechnology company developing a new generation of disease-modifying oligonucleotide therapies for serious genetic neuromuscular and neurological diseases, with no approved products and no commercial revenue. The company's core technology is its proprietary Enhanced Delivery Oligonucleotide (EDO) platform — engineered cell-penetrating peptides conjugated to therapeutic oligonucleotides — designed to overcome the historic delivery challenge of efficiently reaching skeletal muscle, cardiac muscle, and nerve cells while maintaining minimal immunogenicity and robust stability. Composition-of-matter patent protection extends into the second half of 2042.

PepGen's lead clinical asset is PGN-EDODM1 for myotonic dystrophy type 1 (DM1), a rare disease affecting over 115,000 people in the U.S. and EU with no approved disease-modifying therapy. PGN-EDODM1 has demonstrated the highest single-dose splicing correction rates ever reported in DM1 and is being evaluated in the FREEDOM-DM1 Phase 1 and FREEDOM2-DM1 Phase 2 trials; it holds both FDA Orphan Drug and Fast Track Designations. The company's second program, PGN-EDO51, targets exon 51 skipping for Duchenne muscular dystrophy (DMD) and is in CONNECT1-EDO51 and CONNECT2-EDO51 Phase 2 studies. Clinical trial enrollment spans the U.S., U.K., Canada, South Korea, Australia, and New Zealand.

The company's business model is R&D-driven and pre-commercial: revenue is non-existent, capital is raised through equity financings (cumulative disclosed funding of approximately $354M+ across rounds including $112.5M in 2021, $80.1M in February 2024, and roughly $115M in September 2025), and cash burn is substantial with a $89.7M FY2025 net loss. Customers are defined as patient populations (DM1 and DMD amenable to exon 51 skipping), with go-to-market limited to clinical trial sites today and no disclosed commercial pricing, partnership royalties, or licensing revenue. Key risks include the March 2026 FDA partial clinical hold on FREEDOM2-DM1, mixed Phase 2 functional data at the 5 mg/kg dose, and an 18% share price decline tied to the hold.

Name

Pepgen

Legal name

PepGen, Inc.

Website

https://pepgen.com

Company type

Public

Founded year

2021

Operating status

Operating

Headcount range

51–100 employees

Short description

PepGen is a clinical-stage biotechnology company developing proprietary Enhanced Delivery Oligonucleotide (EDO) peptide-conjugated therapies for genetic neuromuscular diseases, with lead programs PGN-EDODM1 for myotonic dystrophy type 1 and PGN-EDO51 for Duchenne muscular dystrophy, both currently in Phase 2 trials.

Ownership category

akta.pro rank

Product category

Biopharmaceuticals

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414)

SIC

Biological Products, (No Disgnostic Substances) (2836), Pharmaceutical Preparations (2834)

akta.pro primary industry

Therapeutic Peptides & Protein/Peptide Biologics (HLAAAAAH)

akta.pro secondary industries

Neurology & CNS Specialty Pharmaceuticals (HLAIACAC), Engineered Protein Therapeutics (de novo/AI-designed proteins, novel scaffolds) (HLAAAAAJ)

Keywords

• Oligonucleotide therapeutics
• Neuromuscular disease treatments
• Peptide-conjugated drugs
• Genetic medicine delivery
• Rare disease therapies

Headquarters

HQ city

Oxford

HQ country

United Kingdom

HQ region

Europe

Offices2 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales

Distribution channels1 record

Marketing channels4 records

Core offering

PepGen is a clinical-stage biotechnology company developing a pipeline of investigational, disease-modifying oligonucleotide therapeutics for serious genetic neuromuscular and neurological diseases. Its lead product candidate, PGN-EDODM1, targets myotonic dystrophy type 1 (DM1), while PGN-EDO51 targets Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The company's differentiating foundation is its proprietary Enhanced Delivery Oligonucleotide (EDO) peptide platform, which conjugates engineered cell-penetrating peptides to oligonucleotide cargoes to enable delivery to skeletal and cardiac muscle and nerve cells.

Product overview

PepGen is a clinical-stage biotechnology company advancing a new generation of oligonucleotide therapies using its Enhanced Delivery Oligonucleotide (EDO) platform. The company's lead product is PGN-EDODM1 for myotonic dystrophy type 1 (DM1), currently in Phase 1 (FREEDOM-DM1) and Phase 2 (FREEDOM2-DM1) clinical trials. The company also has PGN-EDO51 for Duchenne muscular dystrophy in Phase 2 development. The EDO platform technology enables efficient delivery of therapeutic oligonucleotide cargoes into affected tissues and cells, overcoming key challenges of previous generations of therapies.

Differentiator

Problem solved

Functional benefit

Products and services

• PGN-EDODM1 A novel peptide-conjugated oligonucleotide candidate being studied in patients with myotonic dystrophy type 1 (DM1), a rare genetic neuromuscular disease with no approved disease-modifying therapies. It has demonstrated the highest rates of splicing correction ever reported after a single dose and is generally well tolerated in clinical trials.
• PGN-EDO51 An Enhanced Delivery Oligonucleotide candidate targeting exon 51 skipping for Duchenne muscular dystrophy (DMD), being evaluated in Phase 2 CONNECT1-EDO51 and CONNECT2-EDO51 clinical studies for patients amenable to exon 51 skipping.

Quantifiable outcome

• Highest rates of splicing correction ever reported after a single dose in DM1 patients
• +1 more outcomes

Segments2 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature1 record

Scale indicators7 records

Recent moves8 records

Expansion highlights5 records

Direct peers

• Sarepta Therapeutics: Sarepta is the leading commercial-stage developer of exon-skipping therapies for Duchenne muscular dystrophy (e.g., Exondys 51, Vyondys 53, Elevidys). PepGen directly competes with Sarepta in DMD exon 51 skipping with PGN-EDO51 and in the broader neuromuscular oligonucleotide space.
• Dyne Therapeutics: Dyne is developing oligonucleotide-based therapies (e.g., DYNE-101 for DM1, DYNE-251 for DMD exon 51) using antibody-targeted conjugates for muscle delivery. It is the most direct head-to-head competitor to PepGen's EDO platform in both DM1 and DMD.
• Avidity Biosciences: Avidity uses antibody-RNA conjugates (AOCs) to deliver oligonucleotides to muscle, including DM1 (del-zota) and DMD programs. Its muscle-targeted delivery approach competes directly with PepGen's EDO platform for the same patient populations.
• Wave Life Sciences: Wave is a clinical-stage oligonucleotide platform company developing RNA-targeted therapies for rare and neurological diseases, including DM1 and DMD programs. It is comparable to PepGen as an oligonucleotide platform competitor in the same disease areas.

Broad incumbents

• Ionis Pharmaceuticals: Ionis is the established leader in antisense oligonucleotide (ASO) technology and a key partner/licensor in the oligonucleotide field. Its broad neuromuscular and neurological pipeline (e.g., Spinraza for SMA) makes it a relevant incumbent peer to PepGen's targeted delivery platform.
• Alnylam Pharmaceuticals: Alnylam is the established RNAi therapeutics leader (Onpattro, Givlaari, Amvuttra) with growing CNS and rare-disease pipeline. Its Board Chair Laurie Keating was formerly Alnylam's EVP/GC, and Alnylam is a natural strategic acquirer in the oligonucleotide delivery space where PepGen competes.

Emerging players

• Solid Biosciences: Solid is developing gene therapies and oligonucleotide candidates for Duchenne muscular dystrophy. It is comparable to PepGen as a DMD-focused emerging biotech competing for similar exon-skipping and disease-modifying patient outcomes.
• Edgewise Therapeutics: Edgewise develops muscle-targeted therapeutics for rare neuromuscular diseases including Duchenne and Becker muscular dystrophy. Its muscle biology focus and small-molecule approach provide adjacent competition to PepGen's oligonucleotide-based muscle delivery.
• Scholar Rock: Scholar Rock develops therapeutics targeting growth factor signaling (e.g., apitegromab for SMA, SRK-015-class molecules) in neuromuscular diseases. Its regulatory and clinical leadership (e.g., Hayley Parker, Lisa Wyman) directly overlaps with PepGen's neuromuscular focus.
• Capricor Therapeutics: Capricor develops cell therapy and exosome-based therapeutics (e.g., deramiocel for DMD) for rare neuromuscular diseases. It is an emerging player targeting the same DMD patient population as PepGen's PGN-EDO51 program.

Market position

Strengths4 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• Xx.com/PepGenTx
• Facebookfacebook.com/PepGenTx
• LinkedInlinkedin.com/company/pepgenltd

Revenue estimate

Valuation estimate

Number of profiles

Profiles29 records

Funding overview

Funding rounds6 records

Investors20 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Pepgen