Mission becomes possible for new medical discoveries

Cambridge scientists are spearheading an area of research that promises to trigger treatments for multiple diseases in areas of severe unmet need.

Once again, the capabilities of Mission Therapeutics and its co-founder – Cambridge don Professor Steve Jackson – are thrust into the spotlight.

Mission has just published a seminal review of its field in the prestigious journal Nature Reviews Drug Discovery on an up-and-coming area of medical discovery and innovation, called deubiquitylating enzymes (DUBs). Professor Jackson is one of the authors.

Mission’s vast expertise, combined with the great potential of DUBs as new potential therapeutic targets, led to the invite.

A medical expert on the inside track of the technology tell Business Weekly: “This area of medical research has accelerated over the past decade and is now on the cusp of preclinical and clinical testing for a range of different disease areas, including Parkinson’s.”

Mission’s drug discovery team wrote the peer-reviewed article which outlines how understanding the physiological and pathophysiological roles of human DUBs has accelerated in recent times.

It highlights how elucidating the biological complexities of this enzyme family is providing opportunities for the development of potentially transformative therapies for a range of clinical indications.

DUBs comprise a group of some 100 proteins that are known to play important roles in regulating ubiquitylation, the process whereby ubiquitin – a small protein so called because it is present in all complex organisms and virtually all cells in the body – controls protein homeostasis, protein activity, intracellular location, and sub-cellular turnover or degradation.

The clinical development of DUB inhibitors initially proved challenging, in large part due to issues linked to specificity and selectivity. However, recent progress in DUB enzymology, biology and technological developments, described in the review, has confirmed that DUBs are druggable, novel therapeutic targets. 

As a result, first-generation inhibitors are now moving into, or are on the threshold of entering, patient trials in a number of clinical settings.

Professor Jackson (pictured above) said: “This article in Nature Reviews Drug Discovery covers seminal advances that have yielded critical insights into DUB target biology and chemistry over the past decade. 

“A strong foundation in DUB research has been fundamental to Mission Therapeutics’ novel target validation and drug discovery platforms. To date, the platform has demonstrated the diverse clinical potential of novel chemistries selectively targeting different members of the DUB enzyme class.”

Dr Anker Lundemose, CEO at Mission, added: “This review showcases Mission’s leadership position and strength in the DUB field. It also re-enforces the value of our proprietary DUB platform for the discovery and development of first-in-class drugs that selectively target specific disease associated DUBs. 

“We are now progressing several programmes through preclinical development exemplifying our platform’s translational potential.”

Mission Therapeutics is an early-stage drug development company targeting the ubiquitin pathway for the treatment of neurodegenerative disease, fibrosis, inflammation, cancer and other diseases of unmet need. 

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 centres, including Prof Jackson’s Cancer Research UK Laboratories at the University of Cambridge Gurdon Institute, and leading UK centres in neurodegenerative diseases. The company is managed by a team with broad international, commercial and clinical-science experience.

In February 2016, the company completed an $86m financing that was led by Imperial Innovations and Woodford Patient Capital Trust and included participation from existing investors Sofinnova Partners, Roche Venture Fund, Pfizer Venture Investments and SR One. 

Mission Therapeutics was founded in 2011 and is based at the Babraham Research Campus.