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CRISPR Therapeutics Reports Positive Additional Phase 1 Data for CTX310™ Targeting ANGPTL3 and Provides Update on In Vivo Cardiovascular Pipeline
Globenewswire· 2025-06-26 11:00
Core Insights - CRISPR Therapeutics announced new Phase 1 clinical data for CTX310™, showing dose-dependent reductions in triglycerides (TG) by up to 82% and low-density lipoprotein (LDL) by up to 86%, with a favorable safety profile [1][5] - Complete Phase 1 data for CTX310 is expected to be presented at a medical meeting in the second half of 2025 [1][2] - Data update for CTX320™, targeting the LPA gene, is anticipated in the first half of 2026 [1][2] - The preclinical program CTX340™, targeting refractory hypertension, is advancing towards IND/CTA filings [1][7] Company Updates - CRISPR Therapeutics is focused on executing strategic priorities and advancing its innovative therapy portfolio [2] - The ongoing Phase 1 clinical trial for CTX310 reinforces the potential of the platform to transform treatment for serious cardiovascular diseases [2] - CTX310 targets ANGPTL3, a gene associated with regulating LDL and TG levels, addressing a significant unmet medical need for over 40 million patients in the U.S. [5][8] Clinical Trials - CTX310 is in a Phase 1 trial involving patients with various conditions, including homozygous familial hypercholesterolemia and severe hypertriglyceridemia, with eligibility based on specific TG and LDL-C levels [5][8] - The trial has shown promising results with peak reductions in TG and LDL, maintaining a safety profile consistent with previous findings [5] - CTX320 is also in a Phase 1 trial, focusing on patients with elevated lipoprotein(a) [Lp(a)], a risk factor for major adverse cardiovascular events [5][8] Research and Development - CRISPR Therapeutics has established a proprietary lipid nanoparticle platform for delivering CRISPR/Cas9 to the liver, enhancing its in vivo portfolio [8] - The company is advancing additional candidates, including CTX340 for refractory hypertension and CTX450™ for acute hepatic porphyria [8]