Changing the Lives of Patients with

Myotubular and Centronuclear Myopathies (CNM)

Our Pipeline

Our focus is to become a global leader in transforming the lives of patients living with CNM. To do this, we are leveraging our core strengths and our relationships in the biotechnology industry to develop DYN101.

DYN101

Our lead product candidate, DYN101, is an antisense oligonucleotide or ASO, designed to reduce the expression of dynamin 2 or DNM2, for the treatment of CNM.   DNM2 is a protein, which when elevated, is toxic in muscle cells.

Our co-founder and Chief Scientific Officer, Dr. Belinda Cowling, conducted the foundational research that identified the central role that the DNM2 protein plays in disease pathogenesis in several forms of CNM, and that the down regulation of the expression of DNM2 could provide a novel therapeutic approach. DNM2 is an enzyme present in all cells of the body that is involved in membrane remodeling. We have demonstrated that increased DNM2 is toxic to muscle cells and is the principal cause of disease in all three classical forms of CNM, which make up approximately 72% of the CNM patient population in the United States, European Union, Japan and Australia.

There are three classical forms of CNM. The most severe form of CNM is X-linked myotubular myopathy, or XLCNM, caused by mutations in the MTM1 gene, which accounts for approximately 57% of CNM patients. Mutations in the DNM2 gene result in autosomal dominant CNM, or ADCNM (approximately 11% of CNM patients), and mutations in the BIN1 gene result in autosomal recessive CNM, or ARCNM (approximately 4% of CNM patients).

An estimated # of patients4,000 – 5,000In US, EU, Japan & Australia
Addressable Population~3,000 – 3,500Dynacure
¹ NeuromusculDisord.2018 Sep;28(9):766-777. doi: 10.1016/j.nmd.2018.06.012. Epub2018 Jul1. https://doi.org/10.1016/j.nmd.2018.06.012 

By targeting DNM2 with our lead product candidate, DYN101, we are potentially able to address the majority of CNM patients. In preclinical studies, we observed reversion of CNM with a dose-dependent effect. We observed in these studies that a reduction in DNM2 reversed disease features, improved muscle mass and muscle force and extended the lifespan of mice with the most severe form of CNM.

We are conducting our UNITE-CNM Phase 1/2 clinical trial in Europe of DYN101, for the treatment of patients aged 16 or older with the two most common forms of CNM, which together comprise approximately 68% of the estimated 4,000 to 5,000 CNM patient population in the United States, European Union, Japan and Australia. In addition to our ongoing UNITE-CNM clinical trial study, we plan to investigate DYN101 in pediatric patients in a Phase 1/2 clinical trial. We believe that DYN101, if approved, could provide the first disease-modifying treatment for the majority of patients living with CNM.

DYN101 clinical trials »