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.

原文： http://t.cn/A6abpX47。

体内基因编辑疗法NTLA-2001初步临床数据披露，结果积极[good]