A MULTI-FACETED APPROACH
Sarepta is pursuing an RNA-targeted approach toward its goal of treating 100% of eligible patients with DMD.
Sarepta is pursuing gene therapy approaches to treat DMD and LGMD.
This program is to 1) leverage the right vector with minimal immune response; 2) employ specific promoters that
drive expression in the intended tissues; and 3) use a transgene that produces a functioning version of the protein.
This program explores a potential surrogate gene therapy approach to DMD. This approach targets the
dystroglycan complex to preserve muscle function. GALGT2 has the potential to treat patients of all ages, disease
severity and to address several muscular dystrophies.
Sarepta’s gene editing approach, using CRISPR/Cas9 technology, is designed to restore dystrophin
expression by removing or “excising” exons from the dystrophin gene. This includes a strategy to excise exons
potentially enabling treatment for a majority of the DMD patient population.