CRISPR Therapeutics Highlights ASGCT Oral Presentation and Announces New Programs Utilizing In Vivo Gene Editing Approach

- ASGCT presentation demonstrates lipid nanoparticle (LNP) mediated delivery to the eye in the context of editing of the myocilin (MYOC) gene as a potential treatment for glaucoma -

- Expands pipeline with new pre-clinical programs utilizing LNP mediated delivery to the liver for refractory hypertension targeting angiotensinogen (AGT) and acute hepatic porphyria (AHP) targeting
5’-aminolevulinate synthase 1 (ALAS1) -

- CTX340, targeting AGT in the liver, achieved durable editing of AGT and sustained ~30 mmHg blood pressure (BP) reduction out to 3 months in the spontaneously hypertensive rat (SHR) model, demonstrating its potential as a one-time therapeutic intervention -

- CTX450, targeting ALAS1 in the liver, achieved ~70% editing of ALAS1 and normalizes disease-associated biomarkers in acute porphyria mouse model, demonstrating its potential as a one-time therapeutic intervention -

- Plans to initiate clinical trials for CTX340 and CTX450 in the second half of 2025 -

ZUG, Switzerland and BOSTON, May 08, 2024 (GLOBE NEWSWIRE) --  CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced new preclinical data presented at the 27th Annual Meeting of the American Society of Cell and Gene Therapy (ASGCT) highlighting the Company’s approach to developing lipid nanoparticle (LNP) based delivery for in vivo ocular gene editing. In addition, CRISPR Therapeutics announced the expansion of its in vivo pipeline with two new programs. CTX340 and CTX450 utilize LNP-based delivery of CRISPR/Cas9 gene editing cargo to the liver, targeting angiotensinogen (AGT) for refractory hypertension and 5’-aminolevulinate synthase 1 (ALAS1) for acute hepatic porphyria (AHP), respectively.

“Over the past two years, we have made significant progress on the development of our lipid nanoparticle platform for the delivery of CRISPR/Cas9 to the liver and are now in clinical trials with CTX310 and CTX320,” said Samarth Kulkarni, Chief Executive and Chairman of the Board of CRISPR Therapeutics. “The expansion of our in vivo pipeline speaks to the scalability of the platform and the exceptional translation capabilities of our team. We continue to add programs to treat both common and rare diseases, as we look to broaden the number of areas where CRISPR could have transformational impact.”