Intellia Therapeutics’ Nexiguran Ziclumeran Secures the US FDA’s RMAT Designation to Treat Hereditary Transthyretin (ATTR) Amyloidosis with Polyneuropathy
Shots:
- The US FDA has designated nexiguran ziclumeran (nex-z/NTLA-2001) with its Regenerative Medicine Advanced Therapy (RMAT) designation for treating hereditary transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN)
- Nexiguran ziclumeran, in comparison with PBO, is being assessed under the P-III (MAGNITUDE) trial for its safety & efficacy in the above mentioned patient population
- Nex-z is a single-dose in vivo CRISPR therapy that is being developed in collaboration with Regeneron. It prevents TTR protein production by inactivating TTR gene to treat ATTR amyloidosis
Ref: Intellia Therapeutics | Image: Intellia Therapeutics
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Disha is a content writer at PharmaShots. She is passionate and curious about recent updates and developments in MedTech and Pharma industry. She covers news related to clinical trial results and updates. She can be contacted at connect@pharmashots.com.
