This agreement follows positive, preliminary results from the Myonexus Limb-girdle muscular dystrophy 2E programme in which the first three patients dosed demonstrated robust and widespread expression of the protein beta-sarcoglycan
, properly localized to the muscle sarcolemma, as measured by immunohistochemistry.
The company reported 51% mean beta-sarcoglycan
fiber expression and also noted the potential to dose higher in an additional cohort after discussing it with the FDA, said Rama, who believes the LGMD2E program warrants broad inclusion in analysts models.
For example, one form may be called alpha-sarcoglycan deficiency and another is known as beta-sarcoglycan
Sarepta Therapeutics is expected to disclose 60-day biopsy results from three LGMD2E patients treated with 5E13 vg/kg MYO-101 a beta-sarcoglycan
gene therapy, Nomura Instinet analyst Christopher Marai tells investors in a research note.
There were also signs of co-localization of neuronal nitric oxide synthase (nNOS) and beta-sarcoglycan
associated with microdystrophin expression.
Further, Sarepta will be presenting 60-day biopsy data from Cohort 1 in early-2019 from the ongoing phase 1/2a MYO-101 beta-sarcoglycan
gene therapy program in limb-girdle muscular dystrophy type 2E, adds Rama.
MYO-101 is designed to transfect a gene that codes for and restores beta-sarcoglycan
protein with the goal of restoring the dystroglycan complex.