We are a clinical-stage company focused on developing and commercializing novel therapies to transform the lives of patients with rare diseases who have limited or no treatment options. We are initially focused on developing treatments for neuromuscular diseases, beginning with myotubular and centronuclear myopathies, or CNM, a group of devastating rare genetic disorders characterized by severe muscle weakness, or myopathy, with shortened life expectancy and significant morbidity.

Our lead product candidate, DYN101, is an antisense oligonucleotide, or ASO, designed to be a disease-modifying medicine for treatment of the majority of CNM patients across multiple mutations in both adult and pediatric populations. ASOs are single-stranded, chemically synthesized nucleic acid analogs designed to bind a corresponding strand of ribonucleic acid and alter protein expression. We are conducting a Phase 1/2 clinical trial in Europe of DYN101, which is called UNITE-CNM, and we intend to expand this trial to sites in the United States in the second half of 2021. We expect to report interim pharmacokinetic, or PK, and safety data from this trial in the second half of 2022 and expect to report final data in 2023. DYN101 has been granted orphan drug designation by the U.S. Food and Drug Administration, or FDA, and European Medicines Agency, or EMA, and rare pediatric disease designation by the FDA. In addition, we are currently preparing to conduct a Phase 1/2 clinical trial of DYN101 called DyNaMic in a pediatric population in Europe, which may be expanded to the United States. Following interim data from the UNITE-CNM Phase 1/2 trial in adults, and subject to regulatory authorization, we expect to commence this pediatric clinical trial in the second half of 2022 in Europe.

Our second program, DYN201, is a discovery-stage program focused on developing a treatment for a group of rare inherited neurodegenerative disorders characterized by spasticity and motor dysfunction called hereditary spastic paraplegias, or HSP.

*Note: Net loss figures is in U.S. dollars, converted from euros in the prospectus, for the 12 months that ended March 31, 2021. 

(Note: Dynacure withdrew its IPO in an SEC filing dated July 19, 2021. The Dynacure IPO was initially scheduled for pricing on July 15, 2021.)