Phase I

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CRISPR Gene Editing Reduces Disease-Causing Protein in Hereditary Transthyretin Amyloidosis
The results of the phase 1 study open-label trial showed the therapy was safe, raising the possibility of a more effective and more appealing treatment for the…
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Positive Phase 1 Results of AKCEA-TTR-LRx Presented at the Heart Failure Society of America Annual Meeting
Akcea Therapeutics, Inc. (NASDAQ: AKCA), an affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), presented data today from the…
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