NTLA-2002

Intellia Therapeutics' shares dropped pre-market after announcing a strategic reorganization to focus on late-stage pipeline candidates, including Nexiguran ziclumeran for ATTR amyloidosis and NTLA-2002 for hereditary angioedema. The company plans a 27% workforce reduction by 2025, incurring $8M in charges, aiming for a cash runway into 2027. Collaborations with Regeneron for Nexiguran ziclumeran's development continue, with ongoing phase III studies for ATTR amyloidosis and hereditary angioedema treatments.

Intellia Therapeutics is restructuring, discontinuing its NTLA-3001 program for alpha-1 antitrypsin deficiency, laying off over 25% of staff, and focusing on NTLA-2002 and nex-z for hereditary angioedema and transthyretin amyloidosis. Laura Sepp-Lorenzino retires as CSO, succeeded by Birgit Schultes.

Intellia Therapeutics (NASDAQ:NTLA) shifts focus to NTLA-2002 and nex-z, halts NTLA-3001 development, leading to a 27% workforce reduction.

Intellia Therapeutics prioritizes NTLA-2002 for HAE and nex-z for ATTR amyloidosis, aiming for Phase 3 completions and BLA submission by 2026. Strategic reorganization includes a 27% workforce reduction, $8M in charges, and extends cash runway into 1H 2027. Leadership changes announced, with Dr. Schultes promoted to CSO.

Intellia Therapeutics prioritizes NTLA-2002 for HAE and nex-z for ATTR amyloidosis, aiming for near-term value creation. Phase 3 studies for both are progressing, with NTLA-2002's HAELO study completing enrollment by 2025's second half and a Biologics License Application submission planned for 2026. The company plans a 27% workforce reduction by 2025 to support operations until 2027, focusing on these high-value programs. Laura Sepp-Lorenzino retires as CSO, with Birgit Schultes promoted to the role. Intellia aims for commercial readiness by 2026, with strategic milestones including clinical execution and commercial team buildout.

In 2024, Angioedema News highlighted top advancements in angioedema treatment, including deals for donidalorsen, NTLA-2002's 95% attack reduction, Takeda's partnerships, Takhzyro's efficacy in adolescents, deucrictibant's Phase 3 trials, FDA's lift on deucrictibant hold, Ionis' FDA application for donidalorsen, garadacimab's safety, Haegarda's real-world effectiveness, and Orladeyo's long-term benefits in Japan.

NTLA-2002, a CRISPR-based gene-editing therapy, significantly reduced angioedema attacks in a phase II study, with 75% and 77% reductions at 25 mg and 50 mg doses, respectively, compared to placebo. NTLA-2002 was well-tolerated, supporting the 50-mg dose for phase III trials.

A CRISPR-Cas9-based gene therapy, NTLA-2002, shows promise as a one-time treatment for hereditary angioedema (HAE), significantly reducing angioedema attacks and kallikrein levels in a phase 2 study.

NTLA-2002, an in vivo CRISPR-Cas9 gene-editing therapy targeting the KLKB1 gene in HAE patients, showed a single dose reduced angioedema attacks and kallikrein levels, supporting its potential as a functional cure. The therapy was well-tolerated with mild-to-moderate AEs, and Intellia plans to use the 50 mg dose in its phase 3 HAELO trial.

HuidaGene Therapeutics' HG202, a Cas13-based RNA-editing therapy for neovascular age-related macular degeneration (nAMD), received FDA approval for clinical trials. HG202 aims to treat nAMD patients resistant to anti-VEGF treatment, with pre-clinical studies showing significant reduction in CNV area. The BRIGHT trial will assess HG202's safety and tolerability in nAMD patients.