Adrestia Therapeutics Partners with Leading Patient Advocacy Groups to Form Ataxia Telangiectasia Research Consortium

Adrestia Therapeutics, a leader in synthetic rescue therapies for genetic diseases, has announced it has co-founded a research consortium to find new treatments for ataxia telangiectasia (AT), a fatal, inherited, progressive neurodegenerative disorder that is 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 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.

“The entire Adrestia team is moved by the dedication of the AT community and we are honored 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,” said Robert Johnson, Chief Executive Officer of Adrestia. “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.”

“AT has a devastating impact on families around the world and despite its severity and the clear understanding of its primary cause, we still cannot change the course of the disease. We welcome innovative approaches that can move us towards treatments or cures,” said Professor Penny Jeggo, noted DNA damage researcher and Trustee of the AT Society. Brad Margus, founder and Board chair of the A-T Children’s Project, added, “We are proud to begin this new initiative with Adrestia as we drive towards a brighter future for our community.”

“As a group deeply involved with advancing therapeutic research, we know collaboration and medical innovation are the path to a future where the effects of AT are minimised,” said Sean Kelly, Chief Executive of Action for AT. “We are proud to join this consortium and support a new line of promising research alongside other tireless advocacy groups.”

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. Olaparib 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.