The FORCETM platform drives Dyne’s efforts to develop targeted therapeutics for muscle and the central nervous system (CNS) delivery, to stop or reverse disease progression. It leverages an antigen-binding fragment (Fab) that targets the transferrin receptor 1 (TfR1) to enable therapeutic distribution. Its modular design allows Dyne to select payloads that address the genetic basis of disease. We have a broad pipeline with clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD), and preclinical programs for facioscapulohumeral muscular dystrophy (FSHD) and Pompe disease.
DYNE-101
PAYLOAD
Gapmer ASO for DMPK knockdown: DM1
DYNE-251
PAYLOAD
PMO for exon 51 skipping: DMD
DYNE-302
PAYLOAD
siRNA for DUX4 knockdown: FSHD
DYNE-401
PAYLOAD
GAA enzyme replacement therapy (ERT): Pompe disease
Our proprietary Fab binds to TfR1 to enable delivery to muscle and the CNS. We believe a Fab offers significant advantages over a full antibody, including smaller protein load, enhanced tissue penetration, increased tolerability and reduced risk of immune system activation.
The Fab is linked to a therapeutic payload, such as gapmer antisense oligonucleotides (ASO), phosphorodiamidate morpholino oligomers (PMO), small interfering RNA (siRNA), small molecules, or even enzymes such as acid alpha-glucosidase (GAA). Each payload is carefully selected to directly address the cause of the disease to stop or reverse progression.
The linker is chosen based on payload chemistry to enable optimal production of the intended therapeutic.
We believe our FORCE platform provides several advantages, including targeted delivery to muscle and CNS, extended time between doses, and ability to re-dose.
