The gene editing and gene modulation technology company partnered with The Michael J. Fox Foundation for Parkinson's Research (MJFF) to develop two new gene knock-out and knock-in rat models to help researchers investigate the role of alpha-synuclein (SNCA) in brain function.
As the company explained, SNCA is a major constituent of Lewy bodies, protein clumps that are the “pathological hallmark of Parkinson’s Disease.” As such, SNCA is an important target for research into the disease’s onset and development.
“These models will be invaluable for enabling researchers to study the biology, etiology, and potential new therapeutics for Parkinson’s disease,” explained Nicole K. Polinski, associate director, research programs, MJFF.
“This A53T alpha-synuclein knock-in model is especially exciting as this model is the first of its kind and will provide researchers with a key tool for understanding the biology behind a mutation linked to Parkinson’s disease, the importance of the alpha-synuclein protein in the brain and how dysfunction could lead to disease, and a platform for testing possible new therapeutics for the disease,” she told Outsourcing-Pharma.com.
Developing tools for industry research
The partnership between MJFF and Horizon’s in vivo business unit, formerly SAGE Labs, began several years ago when the two collaborated to develop custom knockout models for genes implicated in genetic forms of Parkinson’s disease.
Following a positive response from the research community, Andrew Brown, product manager, in vivo, Horizon Discovery, said the companies decided to expand the partnership to generate, scale up, maintain, and distribute two new models to academic and pharma investigators – “at an affordable price with no licenses required.”
“These two new models were developed as a key initiative in the MJFF Industry Research Tools Consortium, gathering input and investment from industry partners to embark on the generation of these research-enabling models at Horizon using their premium custom model generation service,” Brown told Outsourcing-Pharma.com.
The MJFF Industry Research Tools Consortium is a partnership between the foundation and various industry groups through which it works to develop new tools to address challenges in Parkinson’s Disease research
“These models will be important for academic and industry research partners alike, speeding the understanding of alpha-synuclein biology, Parkinson’s disease etiology, and treatments for Parkinson’s disease,” he added.
Through Horizon’s Sponsored Breeding Program, MJFF said it will to help ensure the models are available to the broader research community, including both for-profit and not-for-profit organizations.