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Entrada Therapeutics Announces Clinical Candidate, ENTR-601-45, for the Potential Treatment of People Living with Duchenne Muscular Dystrophy who are Exon 45 Skipping Amenable
ENTR-601-45 Expands Entrada’s Commitment to the Duchenne Community by Advancing a Potential Treatment for People Living with Duchenne Who are Exon 45 Skipping
About this update from Entrada Therapeutics, Inc.
[{"type":"text","content":"ENTR-601-45 Expands Entrada’s Commitment to the Duchenne Community by Advancing a Potential Treatment for People Living with Duchenne Who are Exon 45 Skipping Amenable\nBOSTON, Jan. 09, 2023 (GLOBE NEWSWIRE) -- Entrada Therapeutics, Inc. (Nasdaq: TRDA), a biopharmaceutical company aiming to transform the lives of patients by establishing intracellular Endosomal Escape Vehicle (EEV™)-therapeutics as a new class of medicines, today announced the selection of a second clinical candidate within its Duchenne franchise, ENTR-601-45, for the potential treatment of people living with Duchenne who are exon 45 skipping amenable. The Company plans to submit an Investigational New Drug (IND) application in the second half of 2024. “There is a profound unmet need for people living with Duchenne and the more than 1,300 people in the US and Europe who are exon 45 skipping amenable,” said Dipal Doshi, President and Chief Executive Officer of Entrada Therapeutics. “We believe that our portfolio of EEV-therapeutic candidates has the potential to provide a transformational impact for these individuals and their families and look forward to continuing to expand our pipeline of therapeutics for those in the Duchenne community.” The selection of ENTR-601-45 is based on robust in vitro exon skipping and dystrophin restoration observed in patient derived skeletal and cardiac muscle cells (Figure 1) as well as in vivo preclinical data demonstrating exon skipping levels of over 90% in skeletal muscle in a hDMD mouse model (Figure 2). Entrada plans to present additional data in support of ENTR-601-45 at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in March 2023. ENTR-601-45, a proprietary Endosomal Escape Vehicle (EEV™)-conjugated phosphorodiamidate morpholino oligomer (PMO), is the third novel clinical candidate from Entrada’s growing pipeline of EEV-therapeutics. Within Entrada’s growing neuromuscular franchise, each EEV-PMO therapeutic candidate has an oligonucleotide sequence designed and optimized for the specific subpopulation of interest (Figure 2). ENTR-601-45 is designed to address the underlying cause of Duchenne muscular dystrophy due to mutated or missing exons in the DMD gene. ENTR-601-45 is an exon skipping investigational therapy that has the potential to restore the mRNA reading frame and allow for the translat...