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Intellia and Regeneron Present Updated Interim Data from Phase 1 Study of CRISPR-based NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis Demonstrating that Deep Serum TTR Reductions Remained Durable After a Single Dose

CAMBRIDGE, Mass. and TARRYTOWN, N.Y., June 24, 2022 (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc. (NASDAQ:NTLA) and Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) today announced additional positive interim data from an ongoing Phase 1 study of their lead investigational in vivo genome editing candidate, NTLA-2001, which is being developed as a single-dose treatment for transthyretin (ATTR) amyloidosis.


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AFFIRM-AL Study Protocol Amendment

Prothena has recently implemented an amendment for the AFFIRM-AL Phase 3 study of birtamimab in Mayo Stage IV AL (light chain) amyloidosis which is currently recruiting patients. Birtamimab is an investigational monoclonal antibody designed to target and clear the abnormal protein (amyloid) that can accumulate and cause organ failure in patients with AL amyloidosis. 


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Alnylam Announces FDA Approval of AMVUTTRA™ (vutrisiran), an RNAi Therapeutic for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Jun. 13, 2022– Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the U.S.