Business
Design Therapeutics Reports GeneTAC™ Portfolio Progress and Second Quarter 2021 Results
Preclinical Data Support Initiation of Clinical Development of Lead GeneTAC Program for Friedreich Ataxia in the First Half of 2022 Leadership Team
About this update from Design Therapeutics, Inc.
[{"type":"text","content":"Preclinical Data Support Initiation of Clinical Development of Lead GeneTAC Program for Friedreich Ataxia in the First Half of 2022 Leadership Team Strengthened with Appointment of Elizabeth Gordon, Ph.D., as Senior Vice President of Regulatory Affairs CARLSBAD, Calif., Aug. 09, 2021 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for degenerative genetic disorders, today reported recent progress with its portfolio of novel small molecule gene targeted chimeras (GeneTACsTM), as well as business highlights and second quarter 2021 financial results. “So far in 2021, we’ve made substantial progress as a company, highlighted by the compelling new data from ongoing IND-enabling studies with our lead GeneTAC program for Friedreich ataxia. Importantly, we’ve observed well-tolerated GeneTAC doses in rodents and non-human primates that produced ample biodistribution into key tissues affected by the disease, including the brain, increasing our confidence in the potential of this program as a disease-modifying treatment for patients,” said João Siffert, M.D., president and chief executive officer of Design Therapeutics. “With plans to begin clinical development in the first half of 2022, we are pleased to welcome Dr. Elizabeth Gordon to the team, who brings decades of valuable experience in overseeing US and ex-US regulatory affairs and successful regulatory submissions.” “Design is well positioned to advance our research and development activities targeting a number of nucleotide repeat expansion diseases, enabled by a talented team that includes multiple new additions in our R&D organization and a strong balance sheet to fuel our pipeline of novel GeneTAC programs,” added Pratik Shah, Ph.D., co-founder and executive chair of Design Therapeutics. “We are now preparing for the important transition to a clinical-stage organization, bringing us another step closer to delivering a new class of genomic medicines for a range of serious disorders currently without approved treatments.” Pipeline Progress New Data from IND-enabling Studies with GeneTAC Product Candidate for Friedreich Ataxia (FA) Support Initiation of Clinical Trial in First Half of 2022: Data from IND-enabling studies in rodents and non-human primates showed that multidose systemic administration of the company’s FA Gene...