AUG 16, 2013
Comprehensive Ophthalmology, Ocular Pathology/Oncology, Pediatric Ophth/Strabismus, Retina/Vitreous
Amarantus BioScience Holdings this week announced data showing recombinant human MANF protein protects both rods and cones from retinal degeneration in an animal model of retinitis pigmentosa.
The company estimates that the market opportunity for retinitis pigmentosa, an orphan indication, exceeds $10 billion annually.
MANF (mesencephalic-astrocyte-derived neurotrophic factor) is a protein that corrects protein misfolding, one of the major causes of apoptosis.
MANF's current lead indication is Parkinson's disease with additional focus on traumatic brain injury. Future indications may include myocardial infarction and certain rare and ultra-rare orphan diseases for which MANF is currently being evaluated.
"The identification of an orphan indication for MANF is a key milestone towards the initial commercialization for MANF," said Dr. Joseph Rubinfeld, Amgen co-founder and a member of the Amarantus’ Board of Advisors. "The delivery of MANF directly to the eye in this indication will be local, potentially significantly reducing timelines to get this into the marketplace versus systemic applications."