Intellia to Showcase Phase 1/2 NTLA-2002 HAE Treatment Results at EAACI 2024

Intellia Therapeutics, a prominent gene-editing firm utilizing CRISPR technology, has announced that their Phase 1 NTLA-2002 study will be presented at the 2024 European Academy of Allergy and Clinical Immunology (EAACI) Congress. NTLA-2002 is an innovative CRISPR-based therapy designed as a single-dose treatment for hereditary angioedema (HAE), a rare genetic disorder that can cause severe swelling attacks. The presentation will showcase the latest safety and efficacy data from the study across three dosage groups.

The session, titled "CRISPR-based gene editing of KLKB1 resulted in long-term plasma kallikrein protein reduction and decreased attack rate in patients with hereditary angioedema: Updated results from a phase 1 study," will be presented by Dr. Hilary Longhurst, the principal investigator from New Zealand, on June 2, 2024. The study aims to demonstrate how NTLA-2002 can potentially be the first one-time treatment for HAE by inactivating the kallikrein B1 gene, which is responsible for the production of prekallikrein, a precursor protein linked to HAE attacks.

Intellia's Phase 1/2 study is an international, open-label trial that has completed enrollment and is set to identify an optimal dosage for further Phase 3 evaluation. The company is planning to initiate the pivotal Phase 3 study in the latter half of 2024, contingent upon regulatory feedback. NTLA-2002 has garnered significant regulatory attention, with designations such as Orphan Drug and RMAT from the FDA, Innovation Passport from the UK's MHRA, PRIME from the EMA, and Orphan Drug Designation from the European Commission.

HAE is a rare genetic disease that leads to painful, unpredictable, and potentially life-threatening inflammatory attacks. Current treatments for HAE are lifelong and may involve frequent intravenous or subcutaneous administration, yet breakthrough attacks can still occur. The interim Phase 1 data for NTLA-2002 has shown significant promise with substantial reductions in attack rates and kallikrein levels.

Intellia Therapeutics is a clinical-stage company dedicated to transforming medicine through CRISPR-based therapies. Their in vivo and ex vivo programs aim to edit disease-causing genes directly within the human body or engineer human cells outside the body for the treatment of cancer and autoimmune diseases. The company is expanding its CRISPR platform with novel editing and delivery technologies to unlock the full potential of gene editing.

The company will also host an investor webcast on June 3, 2024, to discuss the new data. Interested parties can join the live webcast through the provided link or the Events and Presentations page on Intellia's website. A replay will be available for at least 30 days after the call.

Intellia's mission is to set a new standard in medicine by leveraging its deep scientific, technical, and clinical development expertise, along with its talented team. The company's commitment to innovation and excellence is evident in its pursuit to develop transformative therapies for patients in need.