CRISPR updates early-stage trial data for heart disease therapy

[Genetic engineering]
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CRISPR Therapeutics (CRSP [https://seekingalpha.com/symbol/CRSP]) on Saturday announced new results from a Phase 1 trial for CTX310, an in vivo gene editing therapy for patients with high levels of triglycerides and low-density lipoprotein (LDL) cholesterol, also known as "bad" cholesterol.

After a single course of infusion, CTX310, which is designed to edit ANGPTL3, a key gene responsible for regulating LDL and triglycerides, caused an average ~55% drop (up to a maximum of ~84%) in TG at the highest dose, the company said.

Meanwhile, the average declines in LDL and ANGPTL3 reached ~49% and ~73%, respectively, with peak reductions being ~87% and ~89%, the Swiss biotech added, based on data from 15 trial participants who had completed at least 28 days of follow-up.

The experimental therapy showed a well-tolerated safety and tolerability profile that could support its continued advancement in clinical studies, according to CRISPR (CRSP [https://seekingalpha.com/symbol/CRSP]). The company said it is advancing CTX310 into Phase 1b trials, with a focus on severe hypertriglyceridemia and mixed dyslipidemia. [https://seekingalpha.com/pr/20299636-crispr-therapeutics-announces-positive-phase-1-clinical-data-for-ctx310-demonstrating-deep]

A June readout from the study indicated peak reductions of up to 82% and 86% in TG and LDL, respectively, following CTX310 therapy. [https://seekingalpha.com/news/4462716-crispr-delays-early-stage-readout-heart-drug]

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