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EXC 001 is a second-generation antisense oligonucleotide drug designed to inhibit the expression of connective tissue growth factor (CTGF), a protein activated during skin scarring and fibrosis. By binding to CTGF mRNA, EXC 001 suppresses CTGF protein production, thereby reducing fibrotic activity and scar formation. The drug was initially developed by Excaliard Pharmaceuticals in collaboration with Isis Pharmaceuticals (now Ionis Pharmaceuticals) and later acquired by Pfizer. Its primary indication was for the reduction of hypertrophic skin scars following surgery or injury. Clinical studies demonstrated that intradermal injection of EXC 001 significantly reduced scar severity compared to placebo, supporting its antifibrotic mechanism targeting CTGF[1][3][5][6].
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