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Design Therapeutics Completes Dosing in First Patient Cohort of Phase 1 Trial of DT-216 GeneTAC™ Molecule for the Treatment of Friedreich Ataxia
Topline Data from Phase 1 Trial Expected in the Second Half of 2022 DT-216 Granted FDA Fast Track Designation for Patients with Friedreich Ataxia CARLSBAD,
About this update from Design Therapeutics, Inc.
[{"type":"text","content":"Topline Data from Phase 1 Trial Expected in the Second Half of 2022 DT-216 Granted FDA Fast Track Designation for Patients with Friedreich Ataxia CARLSBAD, Calif., March 30, 2022 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for degenerative genetic disorders, today announced that it has completed dosing in the first single ascending dose (SAD) cohort of its Phase 1 clinical trial of DT-216 in patients with Friedreich ataxia (FA). DT-216 is a novel GeneTAC™ gene targeted chimera small molecule designed to specifically target the GAA repeat expansion mutation, the underlying cause of FA, and restore frataxin (FXN) gene expression. Additionally, Design announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to DT-216 for the treatment of patients with FA. “The rapid advancement of our Phase 1 trial of DT-216 – from initiation to dosing completion in the first SAD cohort – marks both our transition to a clinical-stage company and underscores the urgent need for effective treatments for patients with degenerative genetic disorders,” said João Siffert, M.D., president and chief executive officer of Design Therapeutics. “The FDA’s decision to grant Fast Track designation to DT-216 reflects the tremendous need for an effective treatment for people with FA. Our GeneTAC™ molecules are thoughtfully designed to dial up or dial down the expression of disease-causing genes, thereby addressing the root cause of the disease without the need for irreversible genetic modification. In a disease like FA, where deficiency of FXN causes its clinical manifestations, we believe the potential FXN restoration by DT-216 could have a meaningful clinical impact. Preclinical data demonstrated that DT-216 was well tolerated at dose levels projected to achieve concentrations in the CNS, heart and skeletal muscle in excess of those required to restore FXN gene expression in FA patient derived cells in vitro. We look forward to assessing the potential of DT-216 in the clinic with hopes of offering a disease modifying treatment for people living with FA.” “FA is a devastating and life-shortening degenerative condition for which there are no effective treatments or cures available today,” said Jennifer Farmer, MS, Chief Executive Officer of the Fried...