Page 1

DESIGN THERAPEUTICS Design Therapeutics Reports Pipeline Progress and Third Quarter 2021 Results November 9 , 2021 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 Gene TAC TM 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 foci and corrected mRNA splicing in our in vitro experiments . These data support the continued advancement of the DM1 program and underscore the broader potential of GeneTAC ™ molecules to treat multiple additional nucleotide repeat expansion diseases . The company remains on track to initiate its clinical program in DM1 in 2023 . Upcoming Investor Conference Presentations • 33rd Annual Virtual Piper Healthcare Conference : João Siffert , M.D. , president and chief executive officer and Sean Jeffries , Ph.D. , chief operating officer , will participate in a fireside chat during the 33rd Annual Virtual Piper Healthcare Conference , being held November 30 December 2 , 2021. The pre - recorded fireside chat will be available to registered participants beginning on November 22 , 2021 . The webcast will be available in the investor section of the company's website at www.designtx.com and will be archived for 30 days following the presentation . Third Quarter 2021 Financial Results • Net loss for the third quarter ended September 30 , 2021 , was $ 11.3 million . • Research and development expenses for the third quarter of 2021 were $ 8.5 million . • General and administrative expenses for the third quarter of 2021 were $ 2.8 million . • As of September 30 , 2021 , the company had cash , cash equivalents and investment securities of $ 393.7 million . About Design Therapeutics Design Therapeutics is a biotechnology company developing a new class of therapies based on its platform of GeneTACTM gene targeted chimera small molecules . The company's Gene TAC TM molecules are designed to either turn on or turn off a specific disease - causing gene to address the underlying cause of disease . Design's lead program is focused on the treatment of Friedreich ataxia , followed by a program in myotonic dystrophy type - 1 and discovery efforts for multiple other serious degenerative disorders caused by nucleotide repeat expansions . For more information , please