Protein therapy for retinitis pigmentosa shown safe

Amarantus BioScience Holdings, Inc., announced that intravitreal injection of mesencephalic-astrocyte-derived neurotrophic factor (MANF) was shown safe in an animal model, advancing its development as a potential treatment for retinitis pigmentosa.

MANF is believed to have broad potential because it is a naturally-occurring protein produced by the body for the purpose of reducing and preventing apoptosis in response to injury or disease via the unfolded protein response of the endoplasmic reticulum.

"Today's data announcement marks the beginning of our path toward an IND for MANF in retinitis pigmentosa," said Gerald E. Commissiong, president and CEO of Amarantus. "Given what we are seeing with the results to date, we believe there is a significant opportunity to further develop MANF in retinitis pigmentosa. We have already begun the process of preparing for an orphan drug designation filing with the FDA, and will be updating the market on progress in that area in the near future."

By manufacturing MANF and administering it to the body, Amarantus is seeking to use a regenerative medicine approach to assist the body with higher quantities of MANF when needed. MANF's current lead indication is Retinitis Pigmentosa, with other applications including Parkinson's disease, Alzheimer's disease and Wolfram's Syndrome. Additional applications for MANF may include Traumatic Brain Injury, myocardial infarction, antibiotic-induced ototoxicity and certain other rare orphan diseases currently under evaluation.