At the International Liver Congress 2022 in London results were presented of a 2021 study demonstrating that CRISPR–Cas9 gene editing can be deployed directly into the body to treat ATTR Amyloidosis – presenting longer-term prospects of a cure for the condition.
Transthyretin Amyloidosis is a rare systemic condition caused when molecules of TTR protein converge together, forming fibres that interfere with organ function. The condition can be hereditary and leads to heart disease, peripheral nerve damage and fatalities.
The study was sponsored by Intellia Therapeutics and Regeneron Pharmaceuticals with patients administered treatment in the UK and New Zealand. Richmond Pharmacology are leaders in trialling CRISPR gene editing therapies in patients for the first time and were the UK based contract research organisation to deliver the first-in-human study. More information available about the research is available here.
Additionally at the Congress organised by the European Association for the Study of the Liver (EASL), Dr Dominic Pimenta Lead Specialty Research Physician at Richmond Research Institute (RRI) presented on the RRI’s work trialling methods to predict the probability of non-alcoholic fatty liver disease in clinical trial patients, using a combination of biomarkers, traditional statistics, and machine learning. This interim report from 269 patients presented our prototype nomogram, utilising several blood tests and body measurements to predict significant liver fat (steatosis) and scarring (fibrosis) with high accuracy (AUROC 0.93 and 0.82 respectively).
Dr Dominic Pimenta said: “These results are hugely encouraging. This is the interim report of an ongoing study – we have currently recruited 369 volunteers out of a planned 500, and will analyse sub-cohorts for clinical use in trial units in the next stage of the study.
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