New Data from Harvard Medical School Highlights USP30 Reduction as a Potential Strategy for Treatment of Parkinson’s Disease

CAMBRIDGE, UK – 15 November 2022 – Mission Therapeutics (“Mission”), a drug discovery and development company focused on protein homeostasis by selectively inhibiting deubiquitylating enzymes (DUBs), welcomes the presentation of new data from the Beth Israel Deaconess Medical Center (BIDMC) in Boston, which suggests that manipulating USP30 is a potential strategy for treating Parkinson’s disease.

The data, which was presented yesterday at the 2022 Society for Neuroscience (SfN) conference in San Diego, showed that deletion of DUB USP30 significantly reduced pathological changes associated with Parkinson’s disease.

Mission is already investigating USP30 as a therapeutic target for the treatment of a range of diseases. These include renal diseases such as acute kidney injury (AKI) and chronic kidney disease (CKD), congestive heart failure/cardiomyopathy, as well as other indications, such as idiopathic pulmonary fibrosis (IPF), neurodegenerative diseases, including Parkinson’s disease, and rare genetic mitochondrial diseases.

Mitochondria play a critical role in cellular health. The Ubiquitin E3 Ligase Parkin protein marks damaged or dysfunctional mitochondria for degradation, known as mitophagy. USP30 counteracts Parkin and can therefore prevent the degradation of potentially aberrant mitochondria. Inhibition of USP30 therefore promotes mitophagy and increases clearance of impaired mitochondria, potentially improving cellular health and longevity.

There is accumulating evidence that mitochondrial dysfunction plays a role in the progression of dopaminergic neurodegeneration in Parkinson’s disease patients and model systems. The study presented yesterday by T. Fang, S. Eleuteri and D.K. Simon aimed to investigate USP30 as an active agent in the development of Parkinson’s disease.

The team demonstrated that USP30 knockout enhanced mitophagy and decreased alpha-synucleinopathy and dopaminergic neuronal loss, key pathological hallmarks of Parkinson’s.

Commenting on the new data, Dr Paul Thompson, CSO of Mission Therapeutics, said:

“USP30 is one of Mission’s key therapeutic targets, under investigation for the treatment of a number of diseases, including renal diseases, IPF, heart diseases and neurodegenerative diseases. This year, our lead USP30 DUB program, MTX652, entered the clinic for the treatment of kidney disease. The new, independent data presented yesterday from Harvard Medical School provides further evidence that inhibition of USP30 has potential as a new strategy to treat Parkinson’s disease.”

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Mission Therapeutics Ltd
Anker Lundemose MD PhD
Chief Executive Officer
Tel: +44 (0)1223 607 340
Instinctif Partners
Melanie Toyne-Sewell / Katie Duffell / Jonjo CordeyEmail:
Tel: +44 (0) 20 7457 2013


About Mission Therapeutics

Mission Therapeutics is an early-stage drug development company targeting the ubiquitin pathway for the treatment of kidney disease, neurodegenerative disease, rare mitochondrial diseases and fibrosis. The Company has built a leading platform for the discovery and development of first-in-class, small molecule drugs that selectively target deubiquitylating enzymes (DUBs) – an emerging drug class that is attracting significant commercial interest in the area of protein homeostasis.

Mission has strong links with key academic and research centers, including Prof. Steve Jackson’s Cancer Research UK Laboratories at the University of Cambridge Gurdon Institute, and leading UK centres in neurodegenerative diseases. The Company also has secured major industry partnerships, including its collaboration with AbbVie in November 2018, for the research and preclinical development of specified DUB inhibitors for the treatment of Alzheimer’s Disease and Parkinson’s Disease. The Company is managed by a team with broad international, commercial and clinical-science experience.

To date the Company has received £73 million / $101 million in funding and its investors comprise blue chip institutional and corporate investors including: Pfizer Venture Investments, Sofinnova Partners, Roche Venture Fund, SR One, IP Group and Rosetta Capital. Mission Therapeutics was founded in 2011 and is based at the Babraham Research Campus, Cambridge, UK.

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