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Design Therapeutics Reports Pipeline Progress and Third Quarter 2021 Results
Clinical Initiation of Lead GeneTACTM Program for Friedreich Ataxia On-track for the First Half of 2022 CARLSBAD, Calif., Nov. 09, 2021 (GLOBE NEWSWIRE) --
About this update from Design Therapeutics, Inc.
[{"type":"text","content":"Clinical Initiation of Lead GeneTACTM Program for Friedreich Ataxia On-track for the First Half of 2022 CARLSBAD, Calif., Nov. 09, 2021 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for degenerative genetic disorders, today reported recent pipeline progress and third quarter 2021 financial results. “At Design, our mission is to bring a new class of small-molecule genomic medicines to patients with inherited degenerative diseases. We’ve taken major strides toward achieving our goals, with advancements across our pipeline and GeneTACTM platform, and expansion of our leadership team to support our expected near-term transition to a clinical-stage organization,” said João Siffert, M.D., president and chief executive officer of Design Therapeutics. “We expect 2022 will be a milestone-rich and transformational year for the company, including initiation of the clinical program in Friedreich ataxia, progress with our myotonic dystrophy type-1 GeneTACTM program, and expansion of our pipeline with several earlier stage programs that represent exciting opportunities to treat additional diseases caused by nucleotide repeat expansions.” Recent Pipeline Highlights Friedreich Ataxia (FA) GeneTACTM Program On-track for Clinical Initiation in First Half of 2022: In ongoing IND-enabling studies, Design’s FA GeneTACTM clinical candidate has been shown to be well tolerated in repeat dose GLP toxicity studies in rats and non-human primates at doses that exceed what we estimate to be biologically active in the clinic. Design remains on track to initiate a Phase 1 clinical trial in patients with FA in the first half of 2022, with initial topline clinical data expected in 2022.Positive Preclinical Data Reported Highlighting Disease-Modifying Potential of DM1 GeneTACTM Molecules for Myotonic Dystrophy Type-1 (DM1): Design reported new preclinical data from its novel DM1 GeneTACTM program at the 2021 Virtual Myotonic Dystrophy Foundation Annual Conference in September 2021, including demonstration of near-complete resolution of disease-causing foci and correction of splicing defects in DM1 patient cells. Preclinical in vivo studies demonstrated distribution of DM1 GeneTACTM molecules to key target tissues including skeletal muscle and heart, achieving tissue concentrations that reduced nuclear...