Dyne Therapeutics(DYN) Newsfilter·2024-06-13 20:40Core Findings - FSHD is a severe muscle disorder caused by aberrant expression of the DUX4 gene, leading to progressive muscle wasting and skeletal muscle loss [1] - DYNE-302, developed using the FORCE platform, consists of a fragment antibody (Fab) that binds to transferrin receptor 1 (TfR1) and is conjugated to an siRNA designed to reduce DUX4 expression [1] - DYNE-302 demonstrated robust and durable DUX4 suppression and functional benefit in FSHD preclinical models, with effects lasting up to three months [4][7] FORCE™ Platform - The FORCE platform is designed to develop targeted oligonucleotide therapeutics for serious muscle diseases, leveraging TfR1's role in muscle biology [5] - The platform overcomes current limitations in delivering therapeutics to muscle tissue, aiming to stop or reverse disease progression [5] - Dyne links therapeutic payloads to its TfR1-binding Fab to create targeted treatments for muscle diseases [5] DYNE-302 Preclinical Data - In hTfR1/iFLExD mouse models, a single intravenous dose of DYNE-302 resulted in dose-dependent and robust reduction of the DUX4 transcriptome (D4T) [4] - DYNE-302 also showed high in vitro potency in FSHD patient-derived myotubes [4] - The data highlight the modularity of the FORCE platform to conjugate different oligonucleotides and target underlying disease mechanisms [4] FSHD Overview - FSHD is a rare, progressive genetic disease caused by a mutation in the DUX4 gene, leading to muscle weakness, wasting, and limited mobility [11] - An estimated 16,000-38,000 individuals in the US and approximately 35,000 in Europe are affected by FSHD, with no currently approved therapies [11] Company Overview - Dyne Therapeutics is a clinical-stage muscle disease company focused on advancing innovative therapeutics for genetically driven diseases [6] - The company has a broad pipeline, including clinical programs for DM1 and DMD, and a preclinical program for FSHD [6]