Intellia To Test Second CRISPR Therapeutic In Hereditary Angioedema Patients
- The New Zealand Medicines and Medical Devices Safety Authority (MEDSAFE) has signed off Intellia Therapeutics Inc's (NASDAQ: NTLA) Phase 1/2 study of NTLA-2002 in hereditary angioedema (HAE).
- NTLA-2002 is a systemically administered single-dose CRISPR / Cas9-based therapeutic candidate designed to inactivate the target gene Kallikrein B1 (KLKB1) to reduce plasma kallikrein activity and thus prevent HAE attacks.
- The NTLA-2002 program represents the second systemic in vivo CRISPR genome editing therapy candidate to enter human clinical trials.
- Related: Intellia Shares Promising Biomarker Data From CRISPR/Cas9 Genome Editing Candidate.
- The Phase 1/2 study will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of NTLA-2002 in adult patients.
- The Phase 1 portion of the study is an open-label, single-ascending dose design used to identify up to two dose levels of NTLA-2002 that will be further evaluated in the randomized, placebo-controlled Phase 2 portion of the study.
- Beyond New Zealand, Intellia is submitting additional regulatory applications in other countries as part of its ongoing, multi-national development approach for NTLA-2002.
- Related: Intellia To Test CRISPR-Engineered TCR-T Cell Candidate In Acute Myeloid Leukemia Patients.
- Price Action: NTLA stock is up 0.57% at $130.5 during the premarket session on the last check Wednesday.
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