Our proprietary FORCETM platform drives our efforts to develop targeted, modern oligonucleotide therapeutics with the potential to be life-transforming for patients with serious muscle diseases. We designed the FORCE platform using our deep knowledge of muscle biology and oligonucleotide therapeutics to overcome the current limitations in delivery to muscle tissue with the goal of stopping or reversing disease progression. Our initial focus includes programs for myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD).
Our proprietary antigen-binding fragments (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.