MJFF $4.9M Grant Advances Work Into Mitochondria as Therapy Target

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by Patricia Inácio, PhD |

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Lucy Therapeutics (LucyTx) has received a $4.9 million, two-year grant from the Michael J. Fox Foundation for Parkinson’s Research (MJFF) to advance work in the company’s mitochondrial platform that could lead to new Parkinson’s disease treatments.

The award includes access to a network of MJFF staff and relevant foundation external partners, serving to advise and help guide company scientists.

Mitochondria are cellular compartments best known for providing cells with energy. Problems in mitochondrial function are believed to contribute to the progression of diseases of the central nervous system (CNS, brain and spinal cord), including Parkinson’s and Rett syndrome.

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LucyTx’s mitochondria platform aims to identify targets for therapies that enhance the workings of mitochondria to possibly halt the progression of these disorders.

“LucyTx’s work to better understand mitochondria and to target this underlying pathology with novel drugs is an important effort toward that shared goal,” Marco Baptista, PhD, MJFF’s vice president of research programs, said in a press release.

According to the company, this platform has already identified certain targets that, with the MJFF grant, could be advanced into potential therapeutics.

“Our platform is developing small-molecule drugs for central nervous system diseases. Building on the scientific advances in the field and combined with those that we have made in the past three years, both our Parkinson’s and Rett disease programs are nearing development candidate nominations,” said Amy Ripka, PhD, founder and CEO of LucyTx.

Therapy development has often focused on genetic approaches, targeting genes with a direct link to a particular disease. However, neurological diseases typically lack a clear and direct genetic connection, indicating that other factors are at play.

LucyTx reports that it follows a “disease-bottleneck” approach focused on mitochondrial metabolism, since these cell components not only control energy output, but are also a central hub for gene regulation that holds a key role in a cell’s fate.

“We believe our approach offers a solution since mitochondria represent a nexus point for the intersection of genetic and environmental factors critical to stopping disease progression,” LucyTx states on a company webpage.

The MJFF grant will be used in a two-year project supporting preclinical tests into the efficacy and safety of potential therapies, and to identify biomarkers for Parkinson’s. It will also help to identify other targets with likely roles in disease progression.

“MJFF funds promising research with a goal to significantly improve the lives of people with Parkinson’s and, one day, end this disease,” Baptista said.