Feasibility phase

Huntington‘s disease is a severe hereditary disease that causes involuntary movements, mental impairment and, ultimately, death. A cure is not yet available as current treatments can only alleviate symptoms. A central problem is the accumulation of defective Huntington‘ s proteins in the nerve cells. Current therapies aim to switch off the Huntington‘s gene or reduce the production of Huntington‘s mRNA. The Plantman research project is pursuing a new approach directly at the protein level. The researchers were inspired by plants that do not develop harmful protein aggregates. They identified a plant protein that is able to suppress the toxic Huntington‘s disease proteins.

Pioneering Protein-Level Therapy for Huntington’s Disease

The main aim of the project is to modify this plant protein so that it specifically breaks down the harmful protein aggregates in patients. This new therapeutic approach could make Huntington‘s disease curable for the first time offering a promising prospect for overcoming this serious disease. The project‘s unique approach sets it apart from previous research and has the potential to significantly improve the lives of those affected and their families.

Conceptual phase

Huntington’s disease is a deadly neurodegenerative disease that affects humans. Despite extensive research efforts and scientific advances, it is still considered incurable. Therefore, Plantman’s team wants to use the tools of synthetic plant biology to contribute to the research of a therapeutic option. A central feature of Huntington’s disease is a toxic accumulation of misfolded proteins caused by an abnormally high number of the so-called polyQ fragment of the huntingtin protein. Although plants produce numerous polyQ-rich proteins, there are no known symptoms resulting from toxic accumulation of these proteins. Plantman’s team was able to show that after introducing a human huntingtin fragment into plants, these plants actively prevent the formation of toxic huntingtin aggregates. The plant protein responsible for this, called stromal processing peptidase (SPP), was expressed in human cells using synthetic biology in vitro (i.e., in a test tube). This prevented the toxic accumulation of huntingtin in the cells. These results may enable the development of a new therapy for Huntington’s disease and other diseases with toxic protein aggregation, such as ALS and Alzheimer’s disease. In the upcoming exploratory phase a concept for the best possible utilisation of the novel therapy is to be developed. To this end an IP strategy will be developed, the market will be analysed, the team will be enlarged and initial preliminary investigations will be carried out to characterise SPP.

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