Duchenne muscular dystrophy (DMD) is a devastating muscle wasting disease which presents a clear unmet treatment need. Strategies to restore the dystrophin protein that is otherwise lacking in Duchenne patients will have enormous potential to stabilize or improve disease progression. We have developed MyoDys45-55, a gene editing platform that can restore a functional dystrophin protein in DMD and significantly slow disease progression and improve lifespan. This therapy is applicable for half of all patients.

DISEASE PROGRESSION

Duchenne muscular dystrophy is a progressive disorder, so muscle weakness becomes more prominent with age, leading to premature death.

NUMBERS

  • Incidence of ~1 in 3500-5000 newborn boys worldwide
  • Estimated 50,000 DMD patients in the US and Europe
  • Average lifespan is 27 years
  • 33% of cases are sporadic, with no family history of the disease

CURRENT STANDARD OF CARE

There is currently no cure and only limited approved treatments. The standard of care for Duchenne patients has a modest effect on the disease course and some, such as steroids, have many negative side effects. The need for therapies that have the potential to drastically change the disease course by restoring the dystrophin protein, such as MyoDys45-55, is critical.

BECKER MUSCULAR DYSTROPHY

Becker muscular dystrophy (BMD) is a similar disease to Duchenne but some dystrophin protein is made. Becker patients can present a variety of clinical progressions but one of the most mild phenotypes has been observed in patients with an exon 45-55 deletion, where some patients have been asymptomatic into their 60s. Thus MyoDys45-55 aims to mimic this mild deletion in a permanent fashion by utilizing gene editing. MyoDys45-55 could also be applicable for Becker patients with mutations within exon 45-55 who do not have a mild phenotype.