Adrestia leads fight for new drugs to combat lethal children’s disease

13/02/2023

Adrestia Therapeutics, the Cambridge-based leader in synthetic rescue therapies for genetic diseases, has co-founded a research consortium to find new treatments for a potentially fatal disease.

The mission is to combat ataxia telangiectasia (AT), a lethal, inherited, progressive neurodegenerative disorder typically diagnosed in young children. 

The consortium’s other founding members include the A-T Children’s Project, the AT Society and Action for AT. 

Adrestia, based at the Babraham Research Campus, is applying its synthetic rescue technology to systematically mine the human genome for novel targets which could be used as the basis for therapies for AT and related diseases. Currently, there are no corrective therapies for AT.  

Robert Johnson, CEO of Adrestia, said: “The entire Adrestia team is moved by the dedication of the AT community and we are honoured to be working closely with our consortium partners to combine patient networks, scientific and other resources to accelerate the development of new treatment options, for which there is a desperate need. 

“Many genetic diseases are untreatable with current technologies. The power of our synthetic rescue approach lies in its ability to reveal alternative therapeutic targets which lie outside the core biological pathway. 

“Our ultimate goal is to develop effective medicines which rebalance the underlying biology, unlocking new ways of treating intractable genetic diseases. AT and several related conditions are caused by an inability to repair DNA damage normally. 

“We believe the same synthetic rescue targets may be useful for a range of common diseases which also involve DNA damage repair defects.”  

A wealth of human genetic data has revealed that a range of common chronic diseases are driven by defects in DNA damage repair, which are also the root cause of ataxia telangiectasia and other related inherited conditions. 

These common diseases span neurodegeneration, immune dysfunctions, cardiovascular disease, diabetes and metabolic diseases. 

Adrestia’s co-founding scientist, Professor Steve Jackson, is a noted expert in DNA damage repair biology whose work led to olaparib. This was the first approved cancer drug to exploit DNA damage repair mechanisms via a mechanism termed synthetic lethality, paving the way to applying complementary principles to discover new drugs for genetic diseases. 

• Historical estimates of how many people are affected by the disease vary, but recent data suggests the prevalence may be significantly underestimated, with at least 3,000 diagnosed patients in the US alone. 

Published: 09.02.23