*

Benefit seen in 4 people on highest dose

*

Trial is first test of its gene therapy in common disease,

CEO

says

*

Company aims to start mid-stage trials in 2026

By Julie Steenhuysen

CHICAGO, Nov 8 (Reuters) - A single infusion of CRISPR

Therapeutics' experimental gene therapy was safe and

reduced levels of harmful LDL cholesterol and triglycerides by

half in four people taking the highest dose, raising hope for a

one-time treatment.

"We've never had anything that could lower both LDL and

triglycerides by around 50%," said Cleveland Clinic cardiologist

Dr. Steven Nissen, lead researcher of the first-in-human study

of the therapy.

Although still very early in development, if future trials

prove the treatment, CTX310, to be safe and effective, it could

be practice-changing, said Cleveland Clinic's Dr. Luke Laffin,

the study's co-leader.

"Rather than a once-daily pill or monthly injection, this

therapy would potentially offer a one-time infusion that is safe

and durable for patients with high cholesterol," he said.

Results were presented on Saturday at the American Heart

Association meeting in New Orleans and published in the New

England Journal of Medicine.

High LDL, the so-called "bad" cholesterol, can cause plaques

to build up in artery walls, raising the risk of a heart attack

or stroke. High triglycerides, another blood fat, can also

increase those risks.

SWITCHING OFF A GENE

CTX310 works by switching off a gene called ANGPTL3 through

a single, two-hour infusion. It was inspired by studies showing

people born with an inactive version of the ANGPTL3 gene have a

lower lifetime risk of heart disease with no apparent adverse

consequences.

Regeneron's Evkeeza, which treats a rare genetic

disorder called homozygous familial hypercholesterolemia,

targets the same gene but requires monthly infusions.

CRISPR's trial of 15 patients aged 31-68, conducted in

Australia, New Zealand and the UK, tested five different doses.

All participants had high triglycerides, high LDL cholesterol or

both and had failed to respond to other treatments.

Among four patients who received the highest dose,

triglycerides on average were cut by 55% and LDL by 50% two

weeks after treatment. Levels stayed low for at least two

months.

"We're going to try to demonstrate the safety and efficacy

of these one and done therapies because we think these options

are important for patients," Nissen said.

Three participants had temporary reactions to the therapy,

including nausea and elevated liver enzymes that resolved

quickly, Laffin said.

Participants will be monitored for a year following the

trial, with the option of an additional 15 years of follow-up.

TARGETING COMMON DISEASES

"I think we're just beginning to uncover the power of gene

editing in common diseases," Switzerland-based CRISPR

Therapeutics chief Sam Kulkarni said in an interview, noting

that most gene therapies have been tried in rare diseases.

The company plans to take its data to U.S. regulators with

the aim of starting phase 2 studies in 2026. CEO Kulkarni said

he hoped to have a product on the market in the next four or

five years.

The company would first target people with genetics-related

high cholesterol, though eventually it could be an option for

tens of millions of Americans, if approved.

CRISPR and Vertex already market the gene therapy

Casgevy for sickle-cell disease. Unlike the nearly $2 million

sickle cell therapy that involves a year-long process in which

the patient's cells are collected and genetically altered, the

cholesterol treatment would be a simple infusion.

"We don't know what the cost of this therapy is going to be

ultimately, but it's very likely to be less than $100,000,"

Kulkarni said.