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PepGen Inc. (Nasdaq: PEPG) is a clinical-stage biotechnology company dedicated to transforming the treatment of severe neuromuscular and neurologic diseases through innovative oligonucleotide therapeutics. The company's proprietary Enhanced Delivery Oligonucleotide (EDO) platform is designed to significantly enhance the uptake and activity of conjugated oligonucleotide therapeutics. This advanced platform leverages EDO peptides to optimize tissue penetration, cellular uptake, and nuclear delivery, enabling the transport of oligonucleotides into a variety of target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system.
PepGen’s leading product candidate is PGN-EDO51, which is currently being evaluated in the CONNECT1-EDO51 Phase 2 multiple ascending dose (MAD) clinical trial for the treatment of Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping therapy. This trial marks a significant milestone as the first patient has been dosed, and initial data readout is expected in mid-2024. PGN-EDO51 has shown promising results in Phase 1 trials, demonstrating high levels of exon 51 skipping and a good safety profile.
Another key product in PepGen’s pipeline is PGN-EDODM1, which targets myotonic dystrophy type 1 (DM1). The FREEDOM-DM1 Phase 1 clinical trial is currently underway, with initial data anticipated in 2024. PGN-EDODM1 aims to restore cellular function by delivering a peptide-conjugated antisense oligonucleotide to address the root cause of DM1.
In addition to these, PepGen is developing PGN-EDO53 and PGN-EDO45 for other DMD patients, addressing exon 53 and 45 skipping, respectively.
Financially, PepGen has secured substantial backing through an $80 million underwritten offering of common stock, which will support ongoing research and clinical development. The company’s strategic partnerships with top-tier institutional investors and prominent biotech firms further strengthen its position in the market.
PepGen’s commitment to advancing therapeutic options for debilitating diseases is underscored by its ongoing research and development efforts, robust clinical pipeline, and strategic collaborations. The company continues to drive forward with the aim of delivering impactful therapies to patients with limited treatment options.
FAQ
What is the current stock price of PepGen (PEPG)?
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What is PepGen Inc.?
What is the Enhanced Delivery Oligonucleotide (EDO) platform?
What are the key products in PepGen's pipeline?
What is the CONNECT1-EDO51 clinical trial?
What achievements has PGN-EDO51 demonstrated in trials?
What is the purpose of PGN-EDODM1?
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What is Duchenne muscular dystrophy (DMD)?
What is myotonic dystrophy type 1 (DM1)?