Partnership formed to introduce novel pre-clinical models for Parkinson’s

Gene editing and gene modulation technologies company, Horizon Discovery, has announced a partnership with The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to introduce two new pre-clinical models.

The new gene knock-out and knock-in rat models will be used to investigate the role of alpha-synuclein (SNCA) in brain function and Parkinson’s disease (PD) pathogenesis. SNCA is a major constituent of Lewy bodies — protein clumps that are the pathological hallmark of PD — and as such is an important target for research into the onset and development of the disease.

As a part of the partnership, Horizon has used its SAGEspeed Custom Model Generation platform to develop and launch two innovative models with genetic modifications of SNCA, creating both a knock-out of endogenous alpha-synuclein and a humanized A53T knock-in, which has been linked to early-onset PD.

“Animal models, particularly rat given its more physiologically relevant neurobiology, play a key role in understanding the molecular mechanisms that govern brain function and the development of neurological disease,” said Dr Darrin M Disley, chief executive officer, Horizon Discovery. “We are deeply gratified to be able to continue to partner with the Michael J. Fox Foundation, deploying our leading gene editing platform to develop specialised in vivo disease models as an important part of their ground-breaking work.”

“Researchers rely on advanced research tools and pre-clinical models to understand underlying causes and mechanisms of Parkinson's disease and to develop effective treatments. When such tools are unavailable, investigators must often make their own, investing time and resources that would be better spent studying PD,” added Nicole Polinski, PhD, associate director of research programmes at The Michael J. Fox Foundation. “The growing collection of in vivo models from Horizon and The Michael J. Fox Foundation highlights the ongoing commitment of both organisations to push the boundaries of science, advance Parkinson’s disease understanding and speed therapeutic breakthroughs.”