PepGen Inc. (NASDAQ:PEPG) is a U.S.-based clinical-stage biotechnology company at the forefront of genetic medicine, focused on developing next-generation oligonucleotide therapeutics for serious neuromuscular and genetic diseases. Headquartered in Boston, Massachusetts, PepGen was founded in 2018 as a spinout from the University of Oxford and Imperial College London, bringing together world-class expertise in RNA therapeutics, peptide chemistry, and neuromuscular biology. Since its inception, the company has aimed to overcome one of the most critical challenges in genetic drug development—delivering therapeutics effectively and safely to target tissues.
At the heart of PepGen’s innovation is its proprietary Enhanced Delivery Oligonucleotide (EDO) platform, a breakthrough delivery technology that conjugates peptide carriers to antisense oligonucleotides (ASOs) and other RNA-based drugs. This conjugation enhances tissue penetration, cellular uptake, and nuclear delivery—capabilities essential for unlocking the full therapeutic potential of gene-modifying medicines. By improving both pharmacokinetics and pharmacodynamics, the EDO platform enables PepGen’s therapeutics to reach difficult-to-target cells, such as skeletal muscle and cardiac tissue, which are commonly affected in diseases like Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1).
PepGen’s lead clinical candidate, PGN-EDO51, is an exon-skipping therapy designed to restore dystrophin expression in patients with DMD, one of the most severe and life-limiting genetic neuromuscular disorders. PGN-EDO51 is currently being evaluated in the CONNECT1 clinical study, which is generating vital data on dose response, safety, and functional dystrophin restoration. In parallel, the company is advancing PGN-EDODM1, which is being studied in the ongoing FREEDOM-DM1 trial to treat the underlying genetic cause of myotonic dystrophy by correcting toxic RNA splicing errors. Both programs are representative of PepGen’s vision to deliver transformative therapies that go beyond symptom relief and target disease at its genetic roots.
PepGen’s scientific leadership, built around decades of research in RNA biology and delivery optimization, has earned it a place among the most promising companies in the rare disease space. The company operates with a disciplined development strategy—focusing resources on high-impact programs while continuously expanding its platform to address additional indications, including neurodegenerative and systemic disorders. Backed by strong intellectual property, a robust cash position, and a growing body of clinical evidence, PepGen is building a sustainable pipeline of disease-modifying therapeutics with the potential to reshape the standard of care for underserved patient populations.
As it moves deeper into clinical development, PepGen continues to attract attention from investors, analysts, and the broader scientific community for its unique approach to genetic medicine. With multiple data readouts expected in the near term and long-term aspirations of expanding the EDO platform to new diseases, PepGen is well-positioned to become a leader in the next era of precision therapeutics targeting neuromuscular and genetic conditions with high unmet medical need.
Transformative Science Backed by Early Clinical Promise
At the core of PepGen’s pipeline is PGN-EDO51, an exon-skipping therapeutic candidate designed for patients with Duchenne muscular dystrophy. In the company’s CONNECT1 clinical trial, the therapy showed encouraging signs of dystrophin expression, suggesting that PepGen’s delivery platform is achieving its goal of functional delivery to muscle tissue. Meanwhile, its other lead program, PGN-EDODM1, targeting DM1, is progressing in the ongoing FREEDOM-DM1 trial, which has already demonstrated successful splicing correction at early dose levels. The second half of 2025 is expected to deliver additional data from higher-dose cohorts, potentially validating the therapy’s effectiveness and safety at clinically meaningful thresholds.
Despite being early in development, both programs exemplify PepGen’s commitment to data-driven execution. The company recently opted to pause the CONNECT2 study to strategically allocate resources toward higher-confidence programs—an example of thoughtful trial prioritization in an increasingly capital-conscious biotech market. With a balance sheet featuring approximately $98 million in cash and marketable securities, PepGen is well-positioned to continue funding key trials without immediate dilution.
CHECK THIS OUT: Gilead Sciences (GILD) is a Top Long-Term Biotech Investment for 2025 and CEL-SCI Corporation: Leading the Future of Cancer Immunotherapy with Multikine®.
EDO Platform Potential: Beyond DMD and DM1
While PGN-EDO51 and PGN-EDODM1 are PepGen’s most advanced programs, they represent just the beginning of what could become a much broader pipeline powered by the EDO delivery platform. The company is exploring applications in additional neuromuscular, neurologic, and even systemic indications where delivery remains a bottleneck for oligonucleotide-based therapies. Preclinical efforts are underway to expand into these markets, with early data showing the potential for tissue penetration and target engagement far beyond what existing approaches can achieve.
PepGen’s innovation-first approach could ultimately make it a platform company—a biotech business model that not only produces individual therapies but fuels an entire ecosystem of next-generation treatments. If successful, this would place PepGen among the top-tier innovators in genetic medicine, alongside better-known names in RNA therapeutics.
Litigation Risk Surfaces but Doesn’t Deter Long-Term Vision
While PepGen’s science is gaining momentum, the company recently faced a legal headwind in the form of a securities class action lawsuit filed by Bernstein Liebhard LLP. Announced on June 10, 2025, the suit alleges that the company made misrepresentations about the effectiveness and safety of its PGN-EDO51 program between March 7, 2024, and March 3, 2025. Investors who purchased shares during that period are now part of a class action group, with a lead plaintiff motion deadline set for August 8, 2025.
This type of legal challenge is not uncommon in the high-stakes world of biotech, particularly for early-stage companies with lead clinical assets under intense scrutiny. While the legal process may create temporary uncertainty, the company has not issued any formal admission of wrongdoing, and development of its core programs continues uninterrupted. Investors should note that class actions often focus on disclosure timing or risk communication, and do not necessarily reflect the scientific or regulatory viability of a pipeline. Indeed, similar lawsuits have emerged across the biotech sector, including against peers with ultimately successful clinical outcomes.
As the litigation plays out, PepGen remains focused on executing its clinical and operational objectives. The company’s ability to weather this challenge will depend on its continued delivery of strong clinical data, transparent investor communication, and the long-term potential of its platform—a set of factors that long-term investors often prioritize over headline-driven sentiment.
Analyst Sentiment and Valuation Opportunity
Despite the lawsuit, the investment case for PepGen remains compelling. Analyst coverage remains intact, with average price targets near $7.67, representing more than 400% upside from current levels. Stock rating agencies have acknowledged recent volatility but maintain a cautiously optimistic outlook based on the strength of the EDO platform and upcoming clinical milestones.
With the company’s stock currently trading near historical lows around $1.40, the risk-reward profile appears significantly skewed in favor of upside, particularly as readouts from the FREEDOM-DM1 trial and additional CONNECT1 data could serve as powerful re-rating events. Should either program deliver clear clinical efficacy and favorable safety data, investor sentiment could pivot quickly, especially given the high unmet need and regulatory incentives associated with DMD and DM1.
Final Thoughts: A Critical Year for PepGen’s Trajectory
PepGen Inc. stands at a pivotal juncture in its evolution as a next-generation genetic medicine company. On one hand, it faces a legal challenge that will test its transparency and governance practices. On the other, it holds a scientific platform capable of disrupting the way rare neuromuscular disorders are treated—offering a potentially safer, more effective delivery solution for oligonucleotide therapeutics.
With multiple catalysts on the horizon, a strong cash position, and a management team actively refining its clinical and strategic focus, PepGen may be one of the most undervalued clinical-stage biotech stocks in the current market. For risk-tolerant investors who understand the high-stakes, high-reward nature of early-stage biotech and are comfortable with temporary headline noise, PEPG offers an attractive asymmetric opportunity tied to tangible near-term events and long-term innovation.
As 2025 unfolds, the market will be watching closely—both for courtroom developments and clinical breakthroughs. In the world of next-generation neuromuscular medicine, PepGen may still have the final word.
READ ALSO: Incyte (INCY) is the Next Big Biotech Winner and Bluebird Bio (BLUE) Posts 108% Revenue Growth in Q1 2025 Amid Acquisition Buzz.
