summarise the evidence in ione sentece

Question

this is the first trial that uses lipid nanoparticles to deliver the genome-editing treatment. It is also the first trial to deliver genome-editing components systemically, that is, to the whole body rather than to one specific type of cell or tissue.

The phase 1 trial is sponsored by Intellia Therapeutics in collaboration with Regeneron Pharmaceuticals. The first patient volunteer was dosed in November 2020 in the United Kingdom. In total, data has been shared on fifteen patient volunteers, who received one of four potential dosages of the gene-editing reagents. Most adverse events were mild. All patient volunteers showed a reduction in TTR protein levels, with higher doses leading to greater reductions in the protein. The protein level was reduced by an average of 87% in individuals who received the highest dose. In amyloid disorders like TTR, the level of precursor proteins is related to clinical outcomes. In other words, if the patient volunteers continue to produce less TTR protein, it is very likely that they will have less severe disease. Intellia CEO John Leonard hopes that with dramatic reductions in toxic proteins being produced, people’s bodies will be able to clear out the toxic protein and even reverse damage done by the disease, but it’s too soon to tell. Currently, more patient volunteers are being enrolled in the phase 1 study.

Answer 1

The phase 1 trial sponsored by Intellia Therapeutics and Regeneron Pharmaceuticals showed that lipid nanoparticles can effectively deliver genome-editing treatment systemically, leading to reductions in TTR protein levels in patient volunteers, with higher dosages resulting in greater reductions.