Our Science: Pipeline

For patients suffering from debilitating genetic diseases, Code Bio’s novel non-viral gene delivery platform offers new hope for meaningful gene therapy solutions, across numerous indications, that are titratable, have the potential to be re-dosable, and able to address diseases with mutations in large genes.
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.



Type 1 Diabetes Program

Code Bio’s second discovery program is Type 1 Diabetes (T1D), a disease in which patients produce little or no insulin, a critical hormone for glucose regulation. While the cause of T1D is unknown, it is typically the result of an autoimmune reaction that destroys the insulin-producing beta cells of the pancreas.  Although T1D usually first appears in childhood and adolescence, it can also develop in adults. Approximately 1.6 million Americans suffer from the disease.


In collaboration with the Juvenile Diabetes Research Foundation (JDRF), Code Bio is exploring therapeutic approaches designed to increase the density and replenish critical beta cells. Two strategic alternatives, RNA silencing and tissue remodeling, are being investigated.