Our Science: Pipeline
Our targeted non-viral 3DNA® delivery platform is poised to transform the field of genetic medicines
For people suffering from debilitating genetic diseases, Code Bio’s novel, targeted, non-viral delivery platform offers new hope for delivering highly targeted, titratable and re-dosable genetic medicines.
Duchenne Muscular Dystrophy Program
Code Bio’s first gene therapy discovery program is designed to treat Duchenne Muscular Dystrophy (DMD), a debilitating genetic disorder affecting 1 in 3,500 to 5,000 newborn males worldwide, and is caused by mutations in the dystrophin gene, the largest known human gene. DMD is caused by recessive mutations in the dystrophin gene on X chromosome. Boys with DMD show signs of muscle weakness early in childhood, typically between the ages of 2 and 7 years, and often lose ambulation around the time of puberty. Average life expectancy is 26.
Current gene therapies for DMD rely on AAV delivery of truncated dystrophins, which vary among patients depending upon their mutational status. Additionally, AAV delivery can lead to serious toxicities in these patients and is not re-dosable. Code Bio’s program is focused on overcoming the key limitations of other gene therapy approaches.
Additional Research Programs
Code Bio is also exploring a variety of other research programs targeting data focused in the lungs, pancreas and liver.