25 November 2024 - Intellia Therapeutics today announced that the US FDA has granted regenerative medicine advanced therapy designation to nexiguran ziclumeran (nex-z, also known as NTLA-2001) for the treatment of hereditary transthyretin amyloidosis with polyneuropathy. 

Nex-z is an in vivo CRISPR based investigational therapy designed as a single-dose treatment to inactivate the TTR gene and thereby prevent the production of TTR protein for the treatment of hereditary transthyretin amyloidosis.

Read Intellia Therapeutics press release