LY3884963

LY3884963 (formerly known as PR006) is an adeno-associated virus serotype 9 (AAV9)-based gene therapy designed to deliver a functional copy of the progranulin gene (GRN) to the brain. It is being developed for the treatment of frontotemporal dementia with progranulin mutations (FTD-GRN). The therapy aims to restore normal levels of progranulin protein in patients with GRN mutations by enabling neuronal cells to produce functional progranulin. Progranulin is a secreted protein involved in lysosomal function and neurotrophic signaling; its deficiency leads to neurodegeneration characteristic of FTD-GRN. The drug is administered as a single intra-cisternal injection and has received orphan drug designation in both the US and EU, as well as fast-track status from the FDA for FTD-GRN. Development was initiated by Prevail Therapeutics, which was acquired by Eli Lilly & Co., now leading further development[1][6][7].

Other names

GRN Gene Therapy