Single-dose gene silencing cure for fatal heart disease comes one step closer

As pioneers in clinical trials, Richmond is delighted to be part of the first large-scale in-human trial of a genetic cure for ATTR Amyloidosis, a fatal disease that can lead to heart failure suffered by some 200,000 - 250,000 people worldwide.

The single-dose treatment known as NTLA- 2001, based on the Nobel Prize-winning CRISPR-Cas9 genetic technology, has been developed by US company Intellia Therapeutics. Richmond is proud to have dosed the first person in the world with this new genetic therapy in Intellia’s Phase 1 study of healthy volunteers which we carried out in 2021. Now, thanks to the promising results of that study, Intellia has commissioned trials on a much larger group of patients, a Phase 3 study they are calling MAGNITUDE.

Richmond’s CEO Dr Jorg Taubel said: “Our mission has always been to accelerate therapies to market, bridging the gap between hope and answers for patients so we are delighted that our pioneering approach is advancing the prospect of a cure for ATTR Amyloidosis.”

Intellia Therapeutics announced the MAGNITUDE study on Monday 18^th March 2024. Read their full announcement here.