DSGN Design Therapeutics Inc.

14.76
+0.15  (+1%)
Previous Close 14.61
Open 14.33
52 Week Low 12.52
52 Week High 50.5
Market Cap $813,493,400
Shares 55,114,729
Float 26,945,855
Enterprise Value $800,332,717
Volume 16,285
Av. Daily Volume 82,891
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Drug Pipeline

Drug Stage Notes
GeneTAC program
Friedreich ataxia (FA)
Phase 1
Phase 1
Phase 1 trial to commence 1H 2022.

Latest News

  1. HIGH POINT, N.C., Oct. 20, 2021 /PRNewswire/ -- vTv Therapeutics Inc. (NASDAQ:VTVT) a clinical-stage biopharmaceutical company focused on the development of orally administered treatments for type 1 diabetes and psoriasis, today announced that Deepa Prasad will lead the company as President and Chief Executive Officer, effective immediately. Stephen L. Holcombe who previously served as vTv's President and Chief Executive Officer will be retiring.

    Deepa joins vTv as it sets to launch phase 3 pivotal studies for its most advanced product, TTP399, which was granted Breakthrough Therapy Designation by the FDA in April as an oral adjunctive therapy for the treatment of type 1 diabetes.

    Recent positive results from the phase 2 study showed treatment…

    HIGH POINT, N.C., Oct. 20, 2021 /PRNewswire/ -- vTv Therapeutics Inc. (NASDAQ:VTVT) a clinical-stage biopharmaceutical company focused on the development of orally administered treatments for type 1 diabetes and psoriasis, today announced that Deepa Prasad will lead the company as President and Chief Executive Officer, effective immediately. Stephen L. Holcombe who previously served as vTv's President and Chief Executive Officer will be retiring.

    Deepa joins vTv as it sets to launch phase 3 pivotal studies for its most advanced product, TTP399, which was granted Breakthrough Therapy Designation by the FDA in April as an oral adjunctive therapy for the treatment of type 1 diabetes.

    Recent positive results from the phase 2 study showed treatment with TTP399 resulted in a statistically significant improvement in HbA1c relative to placebo and a clinically meaningful decrease (40%) in the frequency of severe and symptomatic hypoglycemia.

    Earlier this month, vTv announced positive results from a mechanistic study indicating no increased risk of ketoacidosis with TTP399 during acute insulin withdrawal in patients with type 1 diabetes. Patients taking TTP399 also reported no events of hypoglycemia, while four events of hypoglycemia were reported in the placebo arm.

    "I am thrilled to have Deepa to step in to this role at an exciting time," said Robin E. Abrams, vTv Chairwoman. "Deepa is the right choice to steer the company through this final stage of TTP399 development, given her significant experience in leadership roles at several prominent healthcare companies during pivotal moments of change and growth."

    Ms. Prasad brings over 20 years of healthcare experience spanning venture capital, biotech investment banking, general management, startups and legislation. She most recently served as Managing Director at WestRiver Group, where she led the firm's investments in Design Therapeutics (NASDAQ:DSGN), Ginger (now $3B Headspace Health), and Curai. She currently sits on the Board of Design Therapeutics and is an Independent Advisor to Equilibre Biopharmaceuticals. In June 2021, Deepa was awarded the Falk Marques General Partners Rising Star Award sponsored by Deloitte.

    "I am pleased to join vTv Therapeutics and lead us through our next phase of growth," said Prasad. "Hypoglycemia is a significant cause of morbidity and potential mortality, and vTv is well-positioned to address this serious issue for the worldwide and growing Type 1 diabetes patient population."

    About vTv Therapeutics

    vTv Therapeutics Inc. is a clinical-stage biopharmaceutical company focused on developing oral, small molecule drug candidates. vTv has a pipeline of clinical drug candidates led by programs for the treatment of type 1 diabetes and psoriasis. vTv's development partners are pursuing additional indications in type 2 diabetes, chronic obstructive pulmonary disease, renal disease, primary mitochondrial myopathy, and pancreatic cancer. For more information, please visit www.vtvtherapeutics.com or follow us on Twitter: @vTvTherapeutics.  

    On October 19, 2021, Ms. Prasad was granted stock options (the "Options") to purchase 2,498,635 shares of the Class A common stock of vTv at an exercise price of $1.47 per share pursuant to an inducement award agreement (the "Inducement Award Agreement"). Subject to potential acceleration upon the achievement of certain performance metrics as set forth in the Inducement Award Agreement, the Options will vest on the third anniversary of the grant date. Upon certain terminations of employment, a portion of the Options will vest on a pro rata basis based on the number of days employed during the three year term. The grant of Options was made as an inducement grant under NASDAQ Listing Rule 5635(c)(4).

    Forward-Looking Statements

    This release contains forward-looking statements, which involve risks and uncertainties. These forward-looking statements can be identified by the use of forward-looking terminology, including the terms "anticipate," "believe," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and, in each case, their negative or other various or comparable terminology. All statements other than statements of historical facts contained in this release, including statements regarding the timing of our clinical trials, our strategy, future operations, future financial position, future revenue, projected costs, prospects, plans, objectives of management and expected market growth are forward-looking statements. These statements involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Important factors that could cause our results to vary from expectations include those described under the heading "Risk Factors" in our Annual Report on Form 10-K and our other filings with the SEC. These forward-looking statements reflect our views with respect to future events as of the date of this release and are based on assumptions and subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. These forward-looking statements represent our estimates and assumptions only as of the date of this release and, except as required by law, we undertake no obligation to update or review publicly any forward-looking statements, whether as a result of new information, future events or otherwise after the date of this release. We anticipate that subsequent events and developments will cause our views to change. Our forward-looking statements do not reflect the potential impact of any future acquisitions, merger, dispositions, joint ventures or investments we may undertake. We qualify all of our forward-looking statements by these cautionary statements.

    Contacts

    Investors:

    Corey Davis

    LifeSci Advisors

    CDavis@LifeSciAdvisors.com

    or

    Media:

    PR@vtvtherapeutics.com 



     

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  2. CARLSBAD, Calif., Sept. 08, 2021 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (NASDAQ:DSGN), a biotechnology company developing small molecule treatments for degenerative genetic disorders, today announced new preclinical data from its novel DM1 GeneTAC™ program, which demonstrated a near-complete resolution of disease-causing foci and correction of splicing defects in myotonic dystrophy type-1 (DM1) patient cells. These data will be presented in a poster titled "Small molecule GeneTACs reduce toxic nuclear foci and correct splicing defects in multiple DM1 cell types," at the 2021 Virtual Myotonic Dystrophy Foundation Annual Conference, being held virtually from September 10-11, 2021.

    "DM1 is a devastating multi-system genetic disorder caused…

    CARLSBAD, Calif., Sept. 08, 2021 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (NASDAQ:DSGN), a biotechnology company developing small molecule treatments for degenerative genetic disorders, today announced new preclinical data from its novel DM1 GeneTAC™ program, which demonstrated a near-complete resolution of disease-causing foci and correction of splicing defects in myotonic dystrophy type-1 (DM1) patient cells. These data will be presented in a poster titled "Small molecule GeneTACs reduce toxic nuclear foci and correct splicing defects in multiple DM1 cell types," at the 2021 Virtual Myotonic Dystrophy Foundation Annual Conference, being held virtually from September 10-11, 2021.

    "DM1 is a devastating multi-system genetic disorder caused by a nucleotide repeat expansion in the DMPK gene that leads to progressive muscle weakness, and also affects the heart, the gastrointestinal and endocrine systems, and ultimately impairs respiration. There are currently no approved treatment options," said João Siffert, M.D., president and chief executive officer of Design Therapeutics. "Our DM1 GeneTACs are small molecules designed to address the underlying root causes of DM1 by specifically blocking transcription of the mutant DMPK gene."

    "New preclinical data demonstrated the ability of our DM1 GeneTACs to potently and selectively block expression of the mutant DMPK gene in DM1 patient cells. Reduction of nuclear foci was associated with clear correction of splicing defects that are involved in the multi-system pathophysiology of DM1," added Abhi Bhat, Ph.D., head of R&D of Design Therapeutics. "We believe these data are highly meaningful both for the potential treatment of patients with DM1, as well as further validation of our GeneTAC approach to treating inherited degenerative diseases."

    Design is leveraging its proprietary GeneTAC (gene targeted chimera) platform to develop therapeutic candidates for inherited diseases driven by nucleotide repeat expansions, such as DM1. DM1 is caused by an increased number of CTG triplet repeats in the DMPK gene. Transcription of the mutant DMPK gene forms pre-mRNAs with large CUG hairpin loops that trap splicing proteins in the nucleus. Specifically, the mutant DMPK pre-mRNAs trap a critical CUG-binding protein called muscle blind-like protein 1 (MBNL1), which leads to the formation of toxic nuclear foci. These foci inhibit the ability of MBNL1 to process pre-mRNAs, which when mis-spliced disrupt muscle development and function that is characteristic of DM1.

    Design's DM1 GeneTAC program is designed to block transcription of the mutant DMPK gene and prevent the formation of the CUG hairpin structures that trap MBNL1, thereby addressing the underlying cause of the disease. New preclinical data being presented from studies of Design's DM1 GeneTAC showed:

    • near-complete resolution of toxic nuclear foci of greater than 90% in DM1 patient fibroblasts and myoblasts, with dose-responsive resolution at concentrations less than 100 nM;
    • near-complete correction of splicing defects in the MBNL1 gene of greater than 90% in DM1 patient myoblasts, also at concentrations less than 100 nM;
    • highly selective knockdown of the mutant DMPK allele without affecting the normal, wild-type allele following a 72-hour treatment period in patient fibroblasts;
    • resolution of CUG foci at substantially greater levels than with antisense oligonucleotide controls in both primary myotubes and primary myoblast patient cells;
    • redistribution of MBNL1 from aggregates in DM1 patient-derived myotubes; and
    • biodistribution to key tissues implicated in DM1, skeletal muscle and heart, at concentrations above those needed to reverse splicing defects in vitro at doses that were well-tolerated in rodents.

    These data, supplemented by a growing body of data from the company's GeneTAC program for Friedrich ataxia, support the continued advancement of the DM1 program and rationale to evaluate the utility of the GeneTAC approach in multiple additional nucleotide repeat expansion diseases.

    About Design Therapeutics

    Design Therapeutics, Inc. (NASDAQ:DSGN) is a biotechnology company developing a new class of therapies based on a platform of gene targeted chimera (GeneTAC™) small molecules. The company's GeneTAC 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 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: preclinical data and the relevance of such data; Design's DM1 GeneTAC program and its design and potential therapeutic benefits and advantages; and Design's GeneTAC approach. 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," "plans," "expects," "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, developing and commercializing 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, obtain approvals for and commercialize any of 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 and commercialize its product candidates; the risk that Design may not obtain approval to market 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; changes in Design's plans to develop and commercialize its product candidates; 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; our 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 June 30, 2021, as filed with the SEC on August 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



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  3. 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…

    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 GeneTAC was well tolerated and achieved higher concentrations in the CNS (cerebrum, cerebellum, brainstem and spinal cord), heart, and skeletal muscle than needed to restore frataxin (FXN) gene expression. In addition, the company observed that exposure to low nanomolar (nM) concentrations of its FA GeneTAC in neurons and cardiomyocytes derived from FA patient stem cells in in vitro experiments led to robust and durable increases in FXN mRNA, as well as an increase in endogenous protein reaching levels comparable to unaffected individuals.



    • On-track for FA GeneTAC Initiation of Clinical Trial in First Half of 2022: Design has received scientific advice from the European Medicines Agency consistent with the favorable feedback previously received from the U.S. Food and Drug Administration (FDA), supporting the development plan for its FA GeneTAC. In addition, the company has successfully manufactured both drug substance and product at a scale sufficient for clinical use. Design anticipates initiating a Phase 1 clinical trial in patients with Friedreich ataxia in the first half of 2022, with initial topline clinical data expected in the second half of 2022.
    • Continued Progress across GeneTAC Portfolio with Myotonic Dystrophy with Preclinical Data to be Reported in Second Half of 2021: Design has continued to advance its second GeneTAC program focused on treating myotonic dystrophy type-1 (DM1), a genetic disorder that causes progressive muscle weakness and for which there are currently no approved treatment options. The company plans to report preclinical data supporting its potential to develop a disease-modifying treatment for patients with DM1 at a medical meeting in the second half of 2021. In addition, leveraging its GeneTAC platform, Design is progressing several earlier-stage programs targeting diseases caused by inherited nucleotide repeat expansions, which represent significant pipeline opportunities to address serious unmet medical needs.

    Business Highlights

    • Leadership Team Strengthened with Appointment of Elizabeth Gordon, Ph.D. as Senior Vice President of Regulatory Affairs: Dr. Gordon is a recognized expert in regulatory affairs with over 30 years of experience in the pharmaceutical industry and at the FDA. Dr. Gordon most recently served as senior vice president of regulatory affairs at Amplyx Pharmaceuticals, and before that as vice president, regulatory affairs at Shire Pharmaceutics and vice president of regulatory affairs at Lumena Pharmaceuticals. Earlier in her career, Dr. Gordon served in the Center for Biologics Evaluation and Research and the Center for Drug Evaluation and Research at the FDA, where she was instrumental in developing policy for the regulation of biological products. Dr. Gordon earned her Ph.D. in microbiology from the University of Rhode Island.
    • Board of Directors Expanded with Key Appointments: In June, Design further enhanced its board with the appointments of industry veterans, Heather Behanna, Ph.D., principal of SR One, and Deepa Prasad, managing director of WestRiver Group, as directors of the company.

    Second Quarter 2021 Financial Results

    • Net loss for the second quarter ended June 30, 2021 was $7.6 million.      
    • Research and development expenses for the second quarter of 2021 were $5.0 million.
    • General and administrative expenses for the second quarter of 2021 were $2.7 million.
    • As of June 30, 2021, the company had cash, cash equivalents and investment securities of $402.8 million.

    About Design Therapeutics

    Design Therapeutics is a biotechnology company developing a new class of therapies based on a platform of gene targeted chimera (GeneTAC™) small molecules. The company's GeneTAC 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 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: the progress and expected timing of Design's development programs and any clinical trials; the effectiveness of Design's GeneTAC program in the treatment of Friedreich ataxia and myotonic dystrophy type-1; the potential advantages of these GeneTAC programs; Design's continued growth; and the strength of Design's balance sheet and the adequacy of cash on hand. 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," "anticipates," "plans," "expects," "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, developing and commercializing 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, obtain approvals for and commercialize any of 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; preliminary or expected results; changes in Design's plans to develop and commercialize its product candidates; the risk that Design may not obtain approval to market 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; changes in Design's plans to develop and commercialize its product candidates; 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; our 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. 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

    DESIGN THERAPEUTICS, INC.

    CONDENSED STATEMENTS OF OPERATIONS

    (in thousands, except share and per share data)

    (unaudited)

     Three Months Ended June 30,  Six Months Ended June 30, 
     2021  2020  2021  2020 
    Revenue:               
    Grant revenue$  $31  $  $173 
    Operating expenses:               
    Research and development 5,027   1,061   8,902   1,438 
    General and administrative 2,660   433   4,465   821 
    Total operating expenses 7,687   1,494   13,367   2,259 
    Loss from operations (7,687)  (1,463)  (13,367)  (2,086)
    Other income (expense), net 51   21   217   (19)
    Net loss$(7,636) $(1,442) $(13,150) $(2,105)
                    
    Net loss per share, basic and diluted$(0.14) $(0.06) $(0.36) $(0.08)
    Weighted-average shares of common stock outstanding, basic and diluted 55,081,397   25,597,154   36,459,244   25,558,779 
      

    DESIGN THERAPEUTICS, INC.

    CONDENSED BALANCE SHEETS

    (in thousands)

    (unaudited)

     June 30,  December 31, 
     2021  2020 
     (unaudited)     
    Assets       
    Current assets:       
    Cash, cash equivalents and investment securities$402,836  $36,091 
    Prepaid expense and other current assets 2,039   142 
    Total current assets 404,875   36,233 
    Property and equipment, net 271   71 
    Deferred offering costs    212 
    Total assets$405,146  $36,516 
    Liabilities, Convertible Preferred Stock and Stockholders' Equity (Deficit)       
    Current liabilities:       
    Accounts payable$1,585  $1,399 
    Accrued expenses 1,820   931 
    Total current liabilities 3,405   2,330 
    Other long-term liabilities 140   145 
    Total liabilities 3,545   2,475 
    Convertible preferred stock    45,356 
    Total stockholders' equity (deficit) 401,601   (11,315)
    Total liabilities, convertible preferred stock and stockholders' equity (deficit)$405,146  $36,516 

     



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  4. CARLSBAD, Calif., June 07, 2021 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (NASDAQ:DSGN), a biotechnology company developing treatments for degenerative genetic disorders, today reported that João Siffert, M.D., president and chief executive officer, will participate in a fireside chat during the Goldman Sachs 42nd Annual Global Healthcare Conference on Wednesday, June 9, 2021 at 1:20 p.m. ET.

    The live webcast will be available in the investor section of the company's website at www.designtx.com. The webcast will be archived for 60 days following the presentation.

    About Design Therapeutics
    Design Therapeutics is a biotechnology company developing a new class of therapies based on a platform of gene targeted chimera (GeneTAC™) small molecules…

    CARLSBAD, Calif., June 07, 2021 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (NASDAQ:DSGN), a biotechnology company developing treatments for degenerative genetic disorders, today reported that João Siffert, M.D., president and chief executive officer, will participate in a fireside chat during the Goldman Sachs 42nd Annual Global Healthcare Conference on Wednesday, June 9, 2021 at 1:20 p.m. ET.

    The live webcast will be available in the investor section of the company's website at www.designtx.com. The webcast will be archived for 60 days following the presentation.

    About Design Therapeutics

    Design Therapeutics is a biotechnology company developing a new class of therapies based on a platform of gene targeted chimera (GeneTAC™) small molecules. Our GeneTAC molecules are designed to either turn on or turn off a specific disease-causing gene to address the underlying cause of disease. The company'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.

    Contact:

    Alicia Davis

    THRUST Strategic Communications

    (910) 620-3302

    alicia@thrustsc.com



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  5. CARLSBAD, Calif., June 07, 2021 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (NASDAQ:DSGN), a biotechnology company developing treatments for degenerative genetic disorders, today announced the appointments of industry veterans, Heather Behanna, Ph.D., principal of SR One, and Deepa Prasad, managing director of WestRiver Group, to its board of directors effective June 15, 2021.

    "We are delighted to further strengthen the Design team with the appointments of both Heather and Deepa to our board of directors, who bring a wealth of experience and insights in corporate and financial strategy and biotechnology company growth," said João Siffert, M.D., president and chief executive officer of Design Therapeutics. "Over the course of 2021, we…

    CARLSBAD, Calif., June 07, 2021 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (NASDAQ:DSGN), a biotechnology company developing treatments for degenerative genetic disorders, today announced the appointments of industry veterans, Heather Behanna, Ph.D., principal of SR One, and Deepa Prasad, managing director of WestRiver Group, to its board of directors effective June 15, 2021.

    "We are delighted to further strengthen the Design team with the appointments of both Heather and Deepa to our board of directors, who bring a wealth of experience and insights in corporate and financial strategy and biotechnology company growth," said João Siffert, M.D., president and chief executive officer of Design Therapeutics. "Over the course of 2021, we have continued to progress our pipeline of novel GeneTAC™ therapeutic candidates, and their expertise will be invaluable as we look to make the important transition to a clinical-stage company with the anticipated initiation of clinical development for our Friedreich ataxia program in the first half of next year."

    Dr. Behanna currently serves as a principal at SR One Capital Management, concentrating on early-stage innovative therapeutic opportunities. Dr. Behanna is currently a board member of Second Genome and Entasis Therapeutics (ETTX) and a board observer of Dren Bio. Prior to SR One, she was with Sofinnova Investments, and prior to that, was an equity research analyst at Wedbush and JMP Securities, focused primarily on therapies for rare disease. Dr. Behanna was formerly a chemist at the Astellas Research institute and adjunct faculty at the Feinberg Northwestern School of Medicine. Dr. Behanna received her Ph.D. in chemistry at Northwestern University, MSc. in organic chemistry at the Weizmann Institute of Science and B.S. from Tufts University.

    "Friedreich ataxia is devastating, with no cure or approved disease-modifying treatment option today. I am highly encouraged by the opportunity enabled by Design's approach to address the underlying cause of this disease," said Dr. Behanna. "I look forward to working alongside the entire team to help guide the business strategy and development plans, so that we may potentially deliver the first treatment to increase endogenous frataxin for patients with Friedreich ataxia."

    Ms. Prasad currently serves as a managing director at WestRiver Group (WRG), where she leverages her more than 20 years of investing and operating experience to focus on investments in healthcare innovation across biotech and digital health and artificial intelligence. Prior to WRG, Ms. Prasad served as chief of staff at Blue Shield, regional vice president and general manager for Optum, head of managed care at the California Hospital Association, and Coherus Biosciences (CHRS) where she led financial strategy and business development. She began her career in investment banking working with biotech and pharma companies on private placements and buyside and sellside mergers and acquisitions. She currently serves on the Grant Funding Committee for UC Davis and as a charter member for TiE, a non-profit global network of entrepreneurs and venture capitalists. Ms. Prasad earned her bachelor's degree in business administration at the University of California, Berkeley and her M.B.A. from the Kellogg School of Management at Northwestern University with emphasis in finance and health industry management.

    "Design's proprietary GeneTAC platform has a unique opportunity to address the root cause of genetic diseases driven by inherited nucleotide repeat expansions and make a significant impact on the lives of those affected," said Ms. Prasad. "The team has made remarkable progress since its founding in 2017, and I am pleased to partner with the Design team and board of directors, so that we may bring forward a new class of small molecule therapeutics that provide a potentially disease-modifying therapy for these patients."

    About Design Therapeutics

    Design Therapeutics is a biotechnology company developing a new class of therapies based on a platform of gene targeted chimera (GeneTAC™) small molecules. Our GeneTAC molecules are designed to either turn on or turn off a specific disease-causing gene to address the underlying cause of disease. The company'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: the progress and expected timing of Design's development programs and any clinical trials; the effectiveness of Design's GeneTAC program in the treatment of Friedreich ataxia and myotonic dystrophy type-1; 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," "anticipates," "plans," "expects," "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, developing and commercializing 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, obtain approvals for and commercialize any of its product candidates; the risk that early research or clinical trials do not demonstrate safety and/or efficacy in later preclinical studies or clinical trials; uncertainties associated with performing clinical trials, regulatory filings and applications; changes in Design's plans to develop and commercialize its product candidates; 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; the loss of key scientific or management personnel; competition in the industry in which Design operates; and general market conditions. For a more detailed discussion of these and other factors, please refer to Design's filings with the Securities and Exchange Commission. 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:

    Alicia Davis

    THRUST Strategic Communications

    (910) 620-3302

    alicia@thrustsc.com



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