CRISPR Therapeutics presented Phase 1 data for its in‑vivo CRISPR therapy CTX310 at the American Heart Association Scientific Sessions and published the results in the New England Journal of Medicine on November 8, 2025. The study evaluated a single‑course intravenous infusion in patients with severe hypertriglyceridemia, heterozygous or homozygous familial hypercholesterolemia, and mixed dyslipidemia.
The trial demonstrated a mean reduction in circulating ANGPTL3 of 73 % (maximum 89 %) and dose‑dependent decreases in triglycerides of 55 % (maximum 84 %) and low‑density lipoprotein cholesterol of 49 % (maximum 87 %). No treatment‑related serious adverse events were reported, and there were no Grade 3 or higher elevations in liver transaminases, underscoring the safety of a single‑dose approach.
These findings are the first‑in‑human evidence that a CRISPR‑based therapy can produce durable lipid lowering from a single infusion, a potential paradigm shift for managing lifelong cardiovascular risk. Chief Medical Officer Naimish Patel said, “For the first time, we’ve shown that a single‑course in‑vivo CRISPR treatment can safely and durably lower ANGPTL3, leading to clinically meaningful reductions in triglycerides and LDL.” Lead investigator Stephen J. Nicholls added, “Seeing a single‑course treatment safely lower both LDL cholesterol and triglycerides is truly unprecedented.” Senior author Steven E. Nissen noted, “Adherence to cholesterol‑lowering therapy remains a major challenge; a one‑time treatment with durable effects would be a major advance in cardiovascular prevention.”
CRISPR plans to advance CTX310 into Phase 1b trials focused on severe hypertriglyceridemia and mixed dyslipidemia, reinforcing its strategy to expand the in‑vivo portfolio and create new revenue opportunities in the cardiovascular space. Chairman and CEO Samarth Kulkarni said in a June 2025 update, “The additional data from our ongoing Phase 1 clinical trial for CTX310 reinforce the potential of our platform to transform the treatment of serious cardiovascular diseases.”
While the CTX310 data are a milestone, CRISPR’s broader financial picture shows a decline in revenue and a net loss in recent quarters. In Q4 2024 the company reported revenue of $35.69 million and a net loss of $37.31 million, a significant drop from the prior year, but it maintains a strong cash position that supports continued investment in its pipeline. The CTX310 breakthrough may help secure future funding and justify the company’s long‑term strategy in cardiovascular gene editing.
The competitive landscape for ANGPTL3 inhibition includes Regeneron’s antibody Evkeeza and Arrowhead/Eli Lilly’s siRNA therapy. CRISPR’s advantage lies in its in‑vivo CRISPR/Cas9 platform, which offers a one‑time, durable gene edit rather than repeated dosing. The company also has other cardiovascular programs—CTX320 targeting lipoprotein(a) and CTX340 targeting angiotensinogen—illustrating a broad pipeline aimed at high‑impact lipid and blood‑pressure disorders.
In summary, the Phase 1 success for CTX310 marks a significant advance for CRISPR Therapeutics and the gene‑editing field, demonstrating a durable, single‑infusion lipid‑lowering effect that could reshape lifelong cardiovascular risk management. The data, coupled with the company’s strategic focus and financial resilience, position CRISPR to pursue further development and potentially transform the treatment landscape for lipid disorders.
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