New Technologies Initiative: Gateway to Treating Genetic Diseases – Sarepta Therapeutics – Skipping Makes Sense – Restoring Muscle Function in Duchenne Muscular Dystrophy
Approval of KYNAMRO (ISIS/Sanofi, HoFH), a first systemic Oligonucleotide Antisense (ASO) based therapy has raised expectations from the compounds in pipeline of other companies with similar technology platforms. Highly differentiated, 2nd Generation DNA/RNA-based drugs that work at the fundamental level of gene and protein expression have the potential to bring a paradigm change in treating diseases esp. rare genetic disorders like – Familial Hypercholesterolemia (FH), Duchenne Muscular Dystrophy (DMD), TTR (Transthyretin amyloidosis), SMN (spinal muscular atrophy) etc. Noteworthy, amongst them…