Advancing a robust portfolio
focused on muscle diseases
 

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.

Visit Our Focus to learn more about DM1, DMD and FSHD.

PROGRAM TARGET
DISCOVERY
PRECLINICAL
PHASE 1
PHASE 2
PHASE 3
ESTIMATED PATIENTS
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
 

Myotonic Dystrophy Type 1 (DM1)

DM1 is a rare, progressive genetic disease that we estimate affects more than 40,000 people in the United States and over 74,000 people in Europe. DM1 is a monogenic, autosomal dominant disease caused by an abnormal expansion in a region of the DMPK gene. Our DM1 program candidates consist of a proprietary Fab conjugated with our linker to an antisense oligonucleotide (ASO). Our program candidates are designed to address the genetic basis of DM1 by reducing the levels of mutant DMPK RNA in the nucleus, releasing splicing proteins, allowing normal mRNA processing and translation of normal proteins and potentially stopping or reversing disease. In preclinical studies, we have observed reduction of nuclear foci and correction of splicing in DM1 patient cells, reversal of myotonia after a single dose in a DM1 disease model, durability of response up to 12 weeks and enhanced muscle distribution as evidenced by reduced levels of cytoplasmic wild type DMPK RNA.

Duchenne Muscular Dystrophy (DMD)

DMD is a rare disease caused by mutations in the gene that encodes for dystrophin, a protein critical for the normal function of muscle cells. We estimate that DMD affects approximately 12,000 to 15,000 people in the U.S. and approximately 25,000 people in Europe. Mutations in the dystrophin gene lead to certain exons being misread, thus resulting in the loss of function of the dystrophin protein, muscle cell death and progressive loss of muscle function. Our DMD program candidates consist of a proprietary Fab conjugated with our linker to a phosphorodiamidate morpholino oligomer (PMO). Our program candidates are designed to deliver a PMO to muscle tissue to promote the skipping of specific DMD exons in the nucleus, allowing muscle cells to create a more complete, functional dystrophin protein and potentially stop or reverse disease progression. In in vitro and in vivo preclinical studies, our program candidates have shown increased exon skipping, increased dystrophin expression, reduced muscle damage and increased muscle function. Our initial development efforts in DMD are focused on developing a therapy for patients with mutations amenable to skipping exon 51. We plan to expand our DMD franchise and develop therapies for patients with mutations amenable to skipping other exons, including exons 53, 45 and 44.

Facioscapulohumeral Muscular Dystrophy (FSHD)

FSHD is a rare disease characterized by progressive, skeletal muscle loss that we estimate affects approximately 16,000 to 38,000 people in the U.S. and 35,000 people in Europe. FSHD is caused by an aberrant expression of the DUX4 gene in muscle tissue, which leads to death of muscle and replacement by fat. Our FSHD program candidates consist of our proprietary Fab conjugated with our linker to an ASO that is designed to address the genetic basis of FSHD by reducing DUX4 expression in muscle tissue. We have an agreement with the University of Mons that provides us with exclusive access to intellectual property to target the genetic cause of FSHD and complements our own proprietary platform for precision delivery into muscle cells. In preclinical studies, we observed that administration of one of our program candidates reduced expression of key DUX4 biomarkers in FSHD patient myotubes.

Discovery programs

We intend to utilize our FORCE platform to expand our portfolio by pursuing the development of programs in additional indications, including additional rare skeletal, cardiac and metabolic muscle diseases. By rationally selecting therapeutic payloads to conjugate with our proprietary antibody and linker, we believe we can develop product candidates to address the genetic basis of additional muscle diseases. back to the pipeline