- Review article in high impact journal outlines the evidence
that neurodegenerative diseases –including Parkinson's,
Alzheimer's, ALS and Huntington's – can be addressed by boosting
mitophagy, the quality control system cells use to remove
dysfunctional mitochondria
- Strong evidence has emerged that deficient mitophagy is an
underlying cause of neurodegenerative disease
- Momentum is building, with the first selective mitophagy
enhancers aimed at modifying the course of neurodegenerative
diseases entering clinical trials in the last 12 months
CAMBRIDGE, England, Jan. 15,
2025 /PRNewswire/ -- Mission Therapeutics
("Mission"), a clinical-stage biotech company developing
first-in-class therapeutics targeting mitophagy, today announces
the publication of a review article titled Targeting mitophagy
in neurodegenerative diseases in the journal Nature Reviews
Drug Discovery. The peer-reviewed article can be viewed
here.
It outlines the progress scientists have made in identifying
that mitochondrial dysfunction is a hallmark of neurodegenerative
disease; how their understanding of the molecular pathways that
underpin the mitophagy process has advanced; and the subsequent
development of two molecular approaches – USP30 inhibition and
PINK1 activation – which are now being tested in early-stage
clinical trials.
Dr Paul Thompson, Chief
Scientific Officer at Mission Therapeutics,
said: "Fresh scientific approaches are needed if we as
a society are to address the growing burden of neurodegenerative
diseases, which are becoming ever more common as populations age.
This timely review outlines the significant potential that
enhancing mitophagy has in tackling a wide range of
neurodegenerative diseases including Parkinson's, Alzheimer's,
amyotrophic lateral sclerosis (ALS) and Huntington's."
Dr Laura Parton, Senior Director at Mission
Therapeutics, said: "We now have a deep understanding
of how the build-up of dysfunctional mitochondria can contribute to
neurodegenerative diseases – and how we can go about addressing
this problem with selective mitophagy enhancers, particularly those
focussing on the USP30/PINK1/Parkin mitophagy pathway. After more
than 30 years of research, the area is now coming to fruition, with
three such drugs entering clinical trials in the last 12
months."
The review was written by a team of renowned mitophagy experts
from across industry and academia, including Mission scientists Dr
Paul Thompson and Dr Laura Parton; Professor Miratul Muqit and
Odetta Antico of Dundee University; and Dr Nicholas T. Hertz, co-founder of Mitokinin
LLC.
Mission Therapeutics is a global leader in discovering and
developing innovative therapeutics that promote mitophagy, the
quality control process cells use to remove dysfunctional
mitochondria, thereby safeguarding their health and function.
Effective mitophagy is particularly important for the ongoing
health of neurons as, unlike most cells, they do not self-renew
throughout an individual's lifetime.
About Mission Therapeutics
Mission Therapeutics is a world leader in discovering and
developing novel therapeutics which promote the removal of
dysfunctional mitochondria, promoting cell health and function.
Mitochondria are energy producing organelles which require lifetime
quality control through a ubiquitin-mediated clearance mechanism
known as mitophagy. In certain situations, such as cellular stress,
cell injury, and/or defects of the mitophagy process, the
mitochondria can become dysfunctional and damaging to the cell,
leading to reduced energy production, oxidative stress,
inflammation and potentially cell death. Dysfunctional mitochondria
are significant drivers of disease pathophysiology in acute kidney
injury (AKI), Parkinson's disease (PD), heart failure, Duchenne's
Muscular Dystrophy, IPF, mitochondrial diseases and
Alzheimer's.
USP30 is a deubiquitylating enzyme that constantly removes
ubiquitin from mitochondria, providing a potential brake on
clearance of dysfunctional mitochondria. Mission is currently
developing two small molecule drugs, MTX652 (peripheral) and MTX325
(targeting the CNS) which, through inhibition of the mitochondrial
DUB enzyme USP30, will promote clearance of dysfunctional
mitochondria – consequently improving overall cellular health.
Mission's USP30 inhibitors MTX652 and MTX325 could potentially be
used to treat any disease or condition driven by mitochondrial
dysfunction.
Mission is backed by blue chip investors including Pfizer
Venture Investments, Sofinnova Partners, Roche Venture Fund, SR
One, IP Group and Rosetta Capital.
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content:https://www.prnewswire.co.uk/news-releases/mission-therapeutics-announces-publication-in-nature-reviews-drug-discovery-of-article-highlighting-how-neurodegenerative-diseases-can-be-tackled-by-enhancing-mitophagy-302350811.html