CRSP - CRISPR Therapeutics Announces Preclinical Data at the American Heart Association (AHA) Scientific Sessions 2023 | Benzinga
-CTX310™ led to durable reductions of angiopoietin-like 3 protein (ANGPTL3) and triglyceride levels in non-human primates (NHPs) after a single dose-
-CTX320™ led to durable reductions of lipoprotein(a) (Lp(a)) levels in NHPs after a single dose-
-Both CTX310™ and CTX320™ were well-tolerated in NHPs at clinically relevant dose levels-
-Clinical trial initiated for CTX310™, targeting ANGPTL3-
-CTX320™ targeting Lp(a) is on track to enter the clinic in the first half of 2024-
ZUG, Switzerland and BOSTON, Nov. 06, 2023 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ:CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced preclinical data from the Company's investigational programs for the treatment of cardiovascular disease, at the American Heart Association (AHA) Scientific Sessions 2023. The data will be presented on Saturday, November 11, 2023, in two oral sessions, entitled "CTX310: An Investigational in vivo CRISPR-Based Therapy Efficiently and Durably Reduces ANGPTL3 Protein and Triglyceride Levels in Non-Human Primates After a Single Dose" and "CTX320: An Investigational in vivo CRISPR-Based Therapy Efficiently and Durably Reduces Lipoprotein(a) Levels in Non-Human Primates After a Single Dose."
"We're excited to share these preclinical data at AHA highlighting the progress made across our in vivo programs targeting ANGPTL3 and Lp(a). The findings demonstrate the potential of a one-time treatment to produce clinically meaningful, long-lasting reductions in risk factors for atherosclerotic cardiovascular disease," said Phuong Khanh (P.K.) Morrow, M.D., FACP, Chief Medical Officer at CRISPR Therapeutics. "These data increase our confidence in our goal to shift the treatment paradigm for patients at risk of cardiovascular disease away from burdensome chronic care to a potentially one-time, durable therapy."
CRISPR Therapeutics is advancing a pipeline of in vivo gene editing programs using lipid nanoparticle (LNP) delivery of Cas9 mRNA and a guide RNA (gRNA) to the liver. The first two in vivo programs, CTX310 and CTX320, each aim to reduce expression of a validated target for cardiovascular disease.
CTX310 is an investigational in vivo CRISPR/Cas9 gene editing therapy designed to knock out hepatic expression of angiopoietin-like 3 protein (ANGPTL3). In humans, ...