{"id":535315,"date":"2021-09-08T06:33:21","date_gmt":"2021-09-08T10:33:21","guid":{"rendered":"https:\/\/www.marketnewsdesk.com\/index.php\/design-therapeutics-announces-positive-preclinical-data-highlighting-disease-modifying-potential-of-its-novel-dm1-genetacs-as-a-treatment-for-myotonic-dystrophy-type-1\/"},"modified":"2021-09-08T06:33:21","modified_gmt":"2021-09-08T10:33:21","slug":"design-therapeutics-announces-positive-preclinical-data-highlighting-disease-modifying-potential-of-its-novel-dm1-genetacs-as-a-treatment-for-myotonic-dystrophy-type-1","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/design-therapeutics-announces-positive-preclinical-data-highlighting-disease-modifying-potential-of-its-novel-dm1-genetacs-as-a-treatment-for-myotonic-dystrophy-type-1\/","title":{"rendered":"Design Therapeutics Announces Positive Preclinical Data Highlighting Disease-Modifying Potential of its Novel DM1 GeneTACs as a Treatment for Myotonic Dystrophy Type-1"},"content":{"rendered":"

\nData to be Presented during the 2021 Virtual Myotonic Dystrophy Foundation Annual Conference
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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\u2122\u00a0program, 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 \u201cSmall molecule GeneTACs reduce toxic nuclear foci and correct splicing defects in multiple DM1 cell types,<\/em>\u201d at the 2021 Virtual Myotonic Dystrophy Foundation Annual Conference, being held virtually from September 10-11, 2021.<\/p>\n

\u201cDM1 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,\u201d said Jo\u00e3o Siffert, M.D., president and chief executive officer of Design Therapeutics. \u201cOur DM1 GeneTACs are small molecules designed to address the underlying root causes of DM1 by specifically blocking transcription of the mutant DMPK gene.\u201d<\/p>\n

\u201cNew 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,\u201d added Abhi Bhat, Ph.D., head of R&D of Design Therapeutics. \u201cWe 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.\u201d<\/p>\n

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.<\/p>\n

Design\u2019s 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\u2019s DM1 GeneTAC showed:<\/p>\n