Our proprietary FORCETM platform drives our efforts to develop and deliver targeted, modern oligonucleotide therapies with the potential to be life-transforming for patients with serious muscle diseases. We are targeting muscle tissue with the goal of stopping or reversing progression in a broad range of diseases. Dyne’s initial focus is on myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD).
We designed the FORCE platform using our deep knowledge of muscle biology and oligonucleotide therapeutics with the goal of overcoming the current challenges of delivery to muscle tissue.
Our proprietary antigen-binding fragment (Fabs) are engineered to bind to a receptor which is highly expressed on muscle cells (TfR1) to enable targeted delivery to skeletal, cardiac and smooth muscle. We believe a Fab offers significant muscle-delivery advantages over a monoclonal antibody (mAb), including enhanced tissue penetration, increased tolerability and reduced risk of immune system activation. We selected our linker based on the safety and efficacy it has demonstrated in approved products, as well as its stability and ability to release therapeutics within muscle cells. Finally, we attach the Fab and linker to a therapeutic such as an antisense oligonucleotide (ASO), phosphorodiamidate morpholino oligomers (PMO), siRNA or small molecule, aimed at addressing the genetic basis of the disease. We believe our FORCE platform provides several advantages, including targeted delivery to muscle tissue, extended time between doses, the ability to re-dose, and an ability to target the genetic basis of disease to stop or reverse its progression.