MANF Therapeutics announces positive data in Parkinson’s model


  • The degeneration of DA neurons occurs before locomotion defects in C. elegans, MANF rescues DA neural degeneration and locomotion defects during all the period of PD progression.
  • MANF reduces the aggregation of -synuclein, restores dopamine levels and rescues the function of DA neurons in the -synuclein C. elegans model of PD.
  • ER stress and autophagy pathways are involved in MANF-mediated neuroprotection.

MANF Therapeutics announced the publication of a scientific article entitled “MANF protects dopamine neurons and locomotion defects from a human alpha-synuclein induced Parkinson’s disease model in C. elegans by regulating ER stress and autophagy pathways” in the peer-reviewed journal Experimental Neurobiology. The authors are researchers at Tongji University School of Medicine and Tongji University’s School of Life Science and Technology.

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 pre-clinical 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 front-runner and primary worldwide 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.

Citation: Experimental Neurology, Volume 308, October 2018, Pages 59-71


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