“I am thrilled to welcome Jae to Design, particularly as we prepare to embark on the important transition to a clinical stage company and initiation of our Phase 1 clinical trial in
patients with Friedreich ataxia,” said João Siffert, M.D., president and chief executive officer of Design Therapeutics. “Jae has had an illustrious career spanning from his time in academic medicine to building broad clinical-stage pipelines, including playing an instrumental role in the approval of a first-in-class gene silencing therapy during his time at Alnylam. He will be invaluable to the future of Design and we look forward to his contributions and insights as we continue on our path of conducting breakthrough genomic medicine drug discovery and advancing novel small molecule drug candidates from the research bench into the clinic.”“Design is at the leading edge of a new generation of biotech companies that are leveraging the known genetic drivers of monogenic disorders to address inherited nucleotide repeat diseases,” said Dr. Kim. “I am highly compelled by Design’s approach to treating the root cause of inherited degenerative disorders; the validating preclinical data demonstrating the ability of its FA GeneTAC™ molecules to dial up FXN expression in an animal model and across multiple human cell systems provide compelling evidence for the potential to treat the cause of FA. Further, Design’s diverse portfolio of GeneTAC™ molecules have the potential to offer a disease-modifying treatment to patients with few therapeutic options across a number of devastating degenerative disorders. I am excited to join this remarkably talented team and to help change the lives of patients and their families.”
Dr. Kim joins Design from Avidity Biosciences, where he served as chief medical officer until August 2021 and led the advancement of the company’s pipeline, including the initiation of the company’s first-in-human study, a Phase 1/2 clinical trial in patients with myotonic dystrophy type-1. Prior to Avidity, he served as Clinical Research Head, Vice President of Clinical Development and Chair of the Clinical Trial Review Board at Alnylam Pharmaceuticals, where he oversaw the development of numerous investigational therapeutics in rare and prevalent diseases across cardiology, neurology, infectious disease, and inborn errors of metabolism. He was instrumental in the successful development and FDA and EMA approval of Givlaari (givosiran), a small interfering RNA therapy, for acute hepatic porphyria. Prior to Alnylam, he served in roles of increasing responsibility in global development at MyoKardia, Inc. and Amgen. Dr. Kim is a board certified cardiologist, was an NIH-funded Principal Investigator and served on the Faculty of Medicine at Harvard Medical School and the Brigham and Women’s Hospital before joining industry. Dr. Kim earned his B.A. degree in neurobiology from Cornell University and his M.D. from Cornell University Medical College. He completed his post-doctoral fellowship in genetics at Harvard Medical School and his clinical training in cardiovascular disease at the Brigham and Women’s Hospital and Massachusetts General Hospital. He has published over 40 peer-reviewed articles and book chapters.
About Design Therapeutics
Design Therapeutics is a biotechnology company developing a new class of therapies based on its platform of GeneTAC™ gene targeted chimera small molecules. The company’s GeneTAC™ molecules are designed to either dial up or dial down the expression of 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 visit designtx.com.
Forward Looking Statements
Statements in this press release that are not purely historical in nature are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements related to: Design’s potential transition to a clinical stage company; the potential initiation of a Phase 1 clinical trial in patients with Friedreich ataxia and the timing thereof; Design’s genomic medicine drug discovery potentially leading to a breakthrough; and the potential advantages of these GeneTAC™ programs. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "believes," “designed to,” "anticipates," “planned,” "expects," “estimate,” "intends," "will," "goal," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Design’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering and developing therapies that are safe and effective for use as human therapeutics and operating as a development stage company; Design’s ability to develop, initiate or complete preclinical studies and clinical trials for its product candidates; the risk that promising early research or clinical trials do not demonstrate safety and/or efficacy in later preclinical studies or clinical trials; changes in Design’s plans to develop its product candidates; uncertainties associated with performing clinical trials, regulatory filings and applications; risks associated with reliance on third parties to successfully conduct clinical trials and preclinical studies; Design’s ability to raise any additional funding it will need to continue to pursue its business and product development plans; regulatory developments in the United States and foreign countries; Design’s reliance on key third parties, including contract manufacturers and contract research organizations; Design’s ability to obtain and maintain intellectual property protection for its product candidates; Design’s ability to recruit and retain key scientific or management personnel; competition in the industry in which Design operates; and market conditions. For a more detailed discussion of these and other factors, please refer to Design’s filings with the Securities and Exchange Commission (“SEC”), including under the “Risk Factors” heading of Design’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2021, as filed with the SEC on November 9, 2021. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Design undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof, except as required by law.
Contact:
Chelcie Lister
THRUST Strategic Communications
(910) 777-3049
chelcie@thrustsc.com
No comments:
Post a Comment