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Intellia Therapeutics
NEWS

Intellia Reports Long-Term P-I Trial Data of Nexiguran Ziclumeran for Hereditary ATTR Amyloidosis with Polyneuropathy (ATTRv-PN)

Shots: Intellia has reported P-I trial data assessing nexiguran ziclumeran (nex-z) in ATTRv-PN; results were presented at the International ATTR Amyloidosis Meeting 2025 & published in The NEJM Single dose of nex-z achieved mean TTR reductions of 92% at 24mos. & 90% in 12 pts followed for 36mos. At 24mos., 13/18 pts met ≥4-point mNIS+7 improvement, incl.…
September 26, 2025

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AstraZeneca & Ionis
NEWS

AstraZeneca and Ionis’ Wainzua (Eplontersen) Receives the EC’s Approval to Treat Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy

Shots: The EC has approved Wainzua to treat ATTRv-PN (stage 1/2 polyneuropathy) in adults pts based on P-III (NEURO-TTRansform) trial assessing eplontersen vs external PBO over 66wks., with follow-up to Wk. 85 & an end-of-trial evaluation. Eligible pts could then enter an ongoing OLE study Trial showed sustained benefits in co-1EPs of serum transthyretin…
March 11, 2025

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Viewpoints_Mina Makar
VIEWPOINTS

Curbing Misfolded TTR with RNA Therapy: Mina Makar from AstraZeneca in a Stimulating Conversation with PharmaShots

Shots:  Misfolded TTR can lead to chronic conditions like ATTRv-PN or ATTR-CM. AstraZeneca’s Wainua (eplontersen) is a once-a-month RNA-targeted medication that suppresses TTR production  Mina Makar SVP and Commerical Lead of the Global Cardiovascular, Renal, and Metabolism (CVRM), Biopharmaceuticals Business Unit at AstraZeneca, sheds light on eplontersen and its ongoing P-III clinical trial on ATTR-CM …
February 17, 2025

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