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Positive Data Reported In PD Study In Alpha-bynuclein Worm Model.

NEW YORK, N.Y., July 9, 2018 -- MANF Therapeutics, Inc., announced the publication of positive results in a preclinical study in the worm model C. elegans advancing the orphan drug-designated therapeutic protein mesencephalic astrocyte-derived neurotrophic factor (MANF) as a disease-modifying treatment for orphan ophthalmological conditions, glaucoma and Parkinson's disease.

The study was performed by researchers at Tongji University School of Medicine and Tongji University's School of Life Science and Technology (Shanghai, PRC).

A gene therapy approach to express MANF was used in the C. elegans Parkinson's disease (PD) model of human alpha-synuclein.

Alpha-synuclein is a protein that is mis-folded and aggregates in PD, potentially driving disease progression.

The data demonstrated that MANF restores locomotory ability in the disease model, as well as restoring morphology and function of the dopaminergic (DA) neurons.

Taken together with previous data demonstrating MANF's ability to regenerate DA neurons by restoring morphological and chemical imbalances in neurotoxin-based animal models of Parkinson's disease, MANF represents a potential disease-modifying treatment for patients based on its unique mechanism of action.

Previously reported preclinical studies have demonstrated the ability to precisely deliver MANF protein to the brain areas associated with Parkinson's disease via convection-enhanced delivery (CED).

MANF Therapeutics is preparing to restart IND-enabling development of MANF in 2018.

MANF has therapeutic potential across multiple orphan ophthalmological conditions such as RAO and retinitis pigmentosa, where MANF has already received orphan drug designations from the FDA, as well as in larger indications such as glaucoma, Parkinson's disease, diabetes and cardiovascular disease, including stroke and myocardial infarction.

MANF Therapeutics is the intellectual property (IP) holder for MANF-based therapies including protein therapy, gene therapy, and cell therapy. The company owns rights to composition of matter patents and patent applications for MANF and owns, or has licenses to, method of use patents and patent applications covering the use of MANF in ophthalmology, neurology and diabetes.

MANF Therapeutics is a wholly-owned subsidiary of Amarantus Bioscience Holdings, Inc. (OTCPK:AMBS).

MANF (mesencephalic-astrocyte-derived neurotrophic factor) is believed to have broad potential because it is a naturally-occurring protein produced by the body to reduce/prevent apoptosis (cell death) in response to injury or disease, via the protein response.

Citation: MANF protects dopamine neurons and locomotion defects from a human a-synuclein induced Parkinson's disease model in C. elegans by regulating ER stress and autophagy pathways, Zhuoyu Zhang et al., Experimental Neurology, Volume 308, October 2018, Pages 59-71, DOI: 10.1016/j.expneurol.20l8.06.016


Contact: Lingjing Jin,


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Title Annotation:Preclinical Research
Publication:Stem Cell Business News
Date:Jul 23, 2018
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