Early Data Favorable for NTLA-2001 Gene-editing Therapy

Early Data Favorable for NTLA-2001 Gene-editing Therapy

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Early Data Favorable for NTLA-2001 Gene-editing Therapy

A single dose of Intellia Therapeutics’ investigational CRISPR/Cas9-based gene-editing therapy NTLA-2001 safely results in a rapid and strong reduction in the levels of the damaging transthyretin (TTR) protein, according to interim data from the first six familial amyloid polyneuropathy (FAP) patients given the therapy in a Phase 1 clinical trial. The clinical data provide the first evidence that CRISPR-Cas9 — a gene-editing system used by bacteria as a defense mechanism — can safely and effectively…

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