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Design Therapeutics Announces Positive Preclinical Data Highlighting Disease-Modifying Potential of its Novel DM1 GeneTACs as a Treatment for Myotonic Dystrophy Type-1
Data to be Presented during the 2021 Virtual Myotonic Dystrophy Foundation Annual Conference CARLSBAD, Calif., Sept. 08, 2021 (GLOBE NEWSWIRE) -- Design
About this update from Design Therapeutics, Inc.
[{"type":"text","content":"Data to be Presented during the 2021 Virtual Myotonic Dystrophy Foundation Annual Conference\nCARLSBAD, 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...