Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2002 in Adults with Hereditary Angioedema (HAE)

Hereditary Angioedema

Phase 1: Primary Endpoint: - Safety and tolerability as determined by adverse events (AEs) - Dose-limiting toxicities (DLTs) Phase 2: - Number of HAE attacks per month (by HAE Attack Assessment and Reporting Procedure – see Section 10.3), Weeks 1 to 16 - Placebo Crossover and Follow-on Dosing Substudy: Safety as determined by AEs

Phase 1: - Change from baseline in total plasma prekallikrein/kallikrein protein level, Phase 1: • Plasma and urine concentrations for DMG-PEG2k, LP000001, clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) messenger ribonucleic acid (mRNA), and single guide RNA (sgRNA), Phase 2: Safety and tolerability as determined by AEs, Phase 2: Number of HAE attacks per month (by HAE Attack Assessment and Reporting Procedure – see Section 10.3) (Weeks 5 to 16) and number of HAE attacks requiring acute therapy per month (Weeks 1 to 16, Weeks 5 to 16), Phase 2: Number of moderate or severe HAE attacks per month (Weeks 5 to 16), Phase 2: Change from baseline in total plasma prekallikrein/kallikrein protein level, Phase 2: Plasma and urine concentrations for DMG-PEG2k, LP000001, Cas9 mRNA, and sgRNA, Placebo Crossover and Follow-on Dosing Substudy: • Number of HAE attacks per month (Weeks 1 to 16 and Weeks 5 to 16 following crossover or follow-on dose) • Number of HAE attacks requiring acute therapy per month (Weeks 1 to 16 and Weeks 5 to 16 following crossover or follow-on dose) • Number of moderate or severe HAE attacks per month (Weeks 1 to 16 and Weeks 5 to 16 following crossover or follow-on dose) • Plasma and urine concentrations for DMG-PEG2k, LP000001, Cas9 mRNA, and sgRNA

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