Pepgen receives u.s. fda fast track designation for pgn-edodm1 for the treatment of myotonic dystrophy type 1
Boston, feb. 20, 2024 (globe newswire) -- pepgen inc. (nasdaq: pepg), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today announced that the u.s. food and drug administration (fda) has granted fast track designation to pgn-edodm1, an investigational candidate for the treatment of myotonic dystrophy type 1 (dm1). “receiving fast track designation from the fda for pgn-edodm1 is a significant milestone in our efforts to deliver a potentially transformative therapy to the dm1 community,” said james mcarthur, ph.d., president and ceo of pepgen. “patients with dm1 currently have no available treatment options that target the root cause of the disease, which leads to progressive neuromuscular symptoms and reduction in life expectancy. following robust preclinical data, we are now evaluating pgn-edodm1 in the ongoing freedom-dm1 phase 1 trial and expect to report preliminary data later this year. we believe that pgn-edodm1 has the potential to be disease-modifying and improve outcomes for patients living with dm1.”
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