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 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.
| DISEASE | TARGET |
DISCOVERY
PRECLINICAL
PHASE 1/2
|
ESTIMATED PATIENTS |
|---|---|---|---|
| Myotonic Dystrophy Type 1 (DM1) Myotonic Dystrophy Type 1 (DM1) Information | DMPK |
DYNE-101 |
US: >40,000 Europe: >74,000 |
| Duchenne Muscular Dystrophy (DMD) Duchenne Muscular Dystrophy (DMD) Information |
Exon 51
Exon 53
Exon 45
Exon 44
Other Exons
|
DYNE-251 |
US: ~12,000-15,000 Europe: ~25,000 |
| Facioscapulohumeral Muscular Dystrophy (FSHD) Facioscapulohumeral Muscular Dystrophy (FSHD) Information | DUX4 |
DYNE-302 |
US: ~16,000-38,000 Europe: ~35,000 |
| Pipeline Expansion Opportunities | |||
| Rare Skeletal CNS Cardiac Metabolic Rare Skeletal Cardiac Metabolic Information |
|||
Myotonic Dystrophy Type 1 (DM1)
DM1 is a rare, progressive genetic disease that is estimated to affect 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.
DYNE-101 is an investigational therapeutic being evaluated in the Phase 1/2 global ACHIEVE clinical trial for people living with myotonic dystrophy type 1 (DM1). DYNE-101, consists of an antisense oligonucleotide (ASO) conjugated to a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1) which is highly expressed on muscle. DYNE-101 is 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. DYNE-101 has been granted orphan drug designation by the European Medicines Agency and the U.S. Food and Drug Administration for the treatment of DM1.
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. These mutations, the majority of which are deletions, result in the lack of dystrophin protein and progressive loss of muscle function. DMD occurs primarily in males and affects an estimated 12,000 to 15,000 individuals in the U.S. and 25,000 in Europe.
DYNE-251 is an investigational therapeutic being evaluated in the Phase 1/2 global DELIVER clinical trial for people living with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. DYNE-251 consists of a phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1) which is highly expressed on muscle. It is designed to enable targeted muscle tissue delivery and promote exon skipping in the nucleus, allowing muscle cells to create a truncated, functional dystrophin protein, with the goal of stopping or reversing disease progression. DYNE-251 has been granted fast track, orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration for the treatment of DMD mutations amenable to exon 51 skipping.
In addition to DYNE-251, Dyne is building a global DMD franchise and has preclinical programs targeting other exons, including 53, 45 and 44.
Facioscapulohumeral Muscular Dystrophy (FSHD)
FSHD is a rare disease characterized by progressive, skeletal muscle loss that is estimated to affect 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 leading to progressive wasting and skeletal muscle loss. DYNE-302 consists of a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1) which is highly expressed on muscle, conjugated to an siRNA designed to reduce DUX4 expression.
Pipeline Expansion Opportunities
We intend to utilize our FORCE platform to expand our portfolio by pursuing the development of programs in additional indications, including additional rare skeletal, CNS, 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.
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