Seeking Novel Solutions to Treat Rare Genetic Skeletal Muscle Diseases

A global leader in therapies for rare genetic diseases is seeking novel approaches to target the primary cause of rare genetic skeletal muscle diseases. They aim to transform patient outcomes in conditions such as Muscular Dystrophies, Myotonic Dystrophy, Pompe Disease, and other rare genetic skeletal muscle diseases. 

Approaches of interest:

• Oligonucleotide approaches (RNAi, siRNA, ASO, saRNA)
• Non-viral gene therapy
• Small molecule approaches
• Gene correction and editing
• Cell therapy, mRNA, and protein replacement/substitution

Out of scope:

• Neuromuscular disorders (e.g., ALS, MS)
• Viral gene therapy approaches

Developmental stage of interest: The company prioritizes novel approaches with demonstrated in vivo proof-of-concepts but will consider research at any stage from early studies through Phase III and registration.