We are building a leading muscle disease company focused on advancing innovative, life-transforming therapies for genetically driven diseases. Our initial focus is on myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). Each of these disorders has a profound impact on affected communities around the world.
We are utilizing our proprietary FORCETM platform to overcome the current limitations of muscle tissue delivery and advance modern oligonucleotide therapeutics for muscle diseases. In selecting diseases to target with our FORCE platform, we seek those with clear translational potential from preclinical disease models to well-defined clinical development and regulatory pathways.
|Myotonic Dystrophy (DM1)||DMPK||US: >40,000 Europe: >74,000|
|Duchenne Muscular Dystrophy (DMD)||Exon 51 Exon 53 Exon 45 Exon 44||US: ~12,000 – 15,000 Europe: ~25,000|
|Facioscapulohumeral Muscular Dystrophy (FSHD)||DUX4||US: ~16,000 – 38,000 Europe: ~35,000|
|Pipeline Expansion Opportunities|
|Rare Skeletal Cardiac Metabolic|