Human genetics pioneer at Cambridge University joining Adrestia to discover and validate new synthetic rescue targets for intractable genetic diseases
Large-scale human population analysis will help identify and validate the safety and efficacy of new targets emerging from the Company’s platform
Human genetics enriches Adrestia’s Synthetic Rescue Atlas, helping to inform indication expansion from ‘gateway’ genetic diseases into more prevalent diseases with a shared genetic component
Human genetic validation builds conviction in the Company’s portfolio of first-in-class therapies to treat neurologic, neuromuscular and cardiomyopathic diseases
CAMBRIDGE, England, May 18, 2022--(BUSINESS WIRE)--Adrestia Therapeutics, a leader in synthetic rescue therapies for genetic diseases, today announced the appointment of Professor John R.B. Perry as its Vice President, Human Genetics. Dr Perry is a world-renowned expert in human population genetics at the University of Cambridge with a track record of identifying genetic drivers of complex, multigenic diseases and health-related traits. His expertise will complement Adrestia’s technology platform, which is already progressing a series of first-in-class therapies to treat both rare and common diseases with a genetic component.
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Professor John R.B. Perry, Vice President, Human Genetics (Photo: Business Wire)
"We are on the cusp of a revolution in drug discovery" said Dr Perry. "Within the 8 billion living humans, each single base substitution in the genome that is compatible with life is likely to be present somewhere. Studying this genetic diversity in population studies gives us a hypothesis-free, genome wide screen for identifying and validating genes causing or influencing human disease. Due to advances in genome sequencing technologies and the advent of large-scale population studies, we can now phenotype human genetic knockouts at scale, leading to rapid validation of new drug discovery targets." said Dr Perry. "I’m excited to apply these advanced techniques for maximum clinical impact. Combining population genetics with Adrestia’s synthetic rescue platform provides unprecedented opportunities to efficiently unlock new ways of treating some of the world’s most intractable diseases".
Throughout his career and in recent years at the University of Cambridge, Professor Perry has been involved in discovering many of the first genetic drivers of diverse complex diseases including obesity, Type 2 diabetes, reproductive disorders and aspects of behavior. His work to better understand the genetic architecture of disease will enable more efficient drug target selection and the design of more predictive models of disease, both translating into higher drug development success rates. His career has been deeply embedded in the UK’s world-leading projects in population genomic analysis, including the UK Biobank, a ground-breaking resource containing in-depth genetic and health information from half a million participants. These projects have created deep expertise and an infrastructure that can be further leveraged for effective drug development at Adrestia.
Despite advances in genetic technologies, many genes known to directly cause disease remain undruggable. Synthetic rescue offers a fresh approach: rather than targeting the causative mutation, a synthetic rescue drug modulates the product of another, functionally connected gene, correcting the effects of the disease mutation and ‘rescuing’ cells from disease. The concept is underpinned by a deep understanding of genetic networks, and its therapeutic potential is illustrated in families where a member born with a disease-causing mutation is protected from disease due to a second ‘rescue’ mutation. Synthetic rescue presents new opportunities for drug development across all diseases that have a genetic component, including prevalent conditions.
"Traditional drug development is risky and expensive" said Robert Johnson, Chief Executive Officer of Adrestia. "We believe that our synthetic rescue platform, supplemented by John’s expertise in human genetics, offers a new paradigm, where the probability of success is markedly higher, the cost of development is dramatically lower and the time to clinical proof of concept is substantially shorter. We warmly welcome John to our team, which is united in a singular mission: to deliver revolutionary new medicines to patients suffering from devastating diseases".
Dr Perry is Professor of Molecular Endocrinology at the University of Cambridge and Medical Research Council (MRC) Programme Leader and Investigator at the University’s Institute of Metabolic Science. He also serves as an Associate Group Leader at The Gurdon Institute at the University of Cambridge, as well as being a Fellow and Director of Studies at King’s College Cambridge. He will continue in these roles while serving at Adrestia.
Prior to joining the MRC Epidemiology Unit, Dr Perry was a Sir Henry Wellcome Fellow, holding visiting positions at the University of Exeter Medical School; the Wellcome Trust Centre for Human Genetics at the University of Oxford; the Department of Twin Research at Kings College London and the Center for Statistical Genetics at the University of Michigan. He has co-authored over 150 publications in peer reviewed journals, many in leading journals such as Nature, Science, Nature Genetics and Nature Medicine. Dr Perry completed his Ph.D. in Human Genetics at the University of Exeter, UK and before then had a background in computer science.
About Adrestia’s synthetic rescue platform
Adrestia has developed a leading synthetic rescue drug development platform, which has already identified completely new approaches to treating intractable genetic diseases. The platform includes multiple components, each designed to yield new insights into how every gene in the human genome modifies a particular disease’s cellular phenotype. These insights are married with human genetic datasets to provide robust validation of new druggable targets and build an ever-growing synthetic rescue ‘Atlas’ of the human genome. As the Atlas expands, the opportunities to use synthetic rescue drugs to treat both rare and common diseases continue to grow.
This scalable platform builds on decades of research by Professor Steve Jackson’s laboratory at the University of Cambridge into synthetic rescue and the related concept of synthetic lethality for the treatment of cancer. The Jackson Laboratory’s work has yielded a wealth of concepts, techniques, and know-how, now translated into Adrestia’s platform.
Adrestia is a leader in synthetic rescue therapies for genetic diseases. As many directly causative mutations are not druggable, synthetic rescue embraces the much broader target set represented by functionally connected genes, to correct the effects of the causative mutations and ‘rescue’ health. Adrestia is creating a synthetic rescue ‘Atlas’ of the human genome and advancing a portfolio of first-in-class therapies, initially for neurologic, neuromuscular and cardiomyopathic diseases. Adrestia’s platform and in-house programs are complemented by a target discovery alliance with GSK and a Huntington’s disease collaboration with noted researchers including Dr Sarah Tabrizi at University College London.
Adrestia was co-founded by Professor Steve Jackson and the deep technology investment fund Ahren Innovation Capital, which co-led Adrestia’s Series A financing along with GSK. Jackson co-originated the first synthetic lethality drug, olaparib, which was the first drug approved to treat cancers caused by inherited mutations. For further information, please visit: http://www.adrestia.com.
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