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PepGen Inc is a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapeutics to transform the treatment of severe neuromuscular and neurologic diseases. The company has developed its Enhanced Delivery Oligonucleotide platform to improve the uptake and activity of conjugated oligonucleotide therapeutics. Its EDO peptides are engineered to optimize tissue penetration, cellular uptake, and nuclear delivery, and in preclinical studies, it is observed their ability to transport oligonucleotides into a broad range of target tissues, including smooth, skeletal, and cardiac muscle and the central nervous system. Its pipeline products are; PGN-EDO51, PGN-EDODM1, and others including PGN-EDO53 and PGN-EDO45.
Larimar Therapeutics Inc is a clinical-stage biotechnology company focused on developing treatments for patients suffering from complex rare diseases using its novel cell-penetrating peptide (CPP) technology platform. Its lead product candidate, CTI-1601, is a subcutaneously administered, recombinant fusion protein intended to deliver human frataxin (FXN) an essential protein, to the mitochondria of patients with Friedreich's ataxia.