It is a treatment based on a Nobel-prize-winning gene-editing technology known as Crispr. But unlike the traditional Crispr approach, which acts like a pair of scissors to make a cut to the DNA,

Verve

VERV -4.07%

is employing a new technology known as base editing, which acts more like a pencil and eraser, substituting a single letter of DNA for another. The gene Verve seeks to edit, PCSK9, regulates LDL levels in the body.

While the treatment is highly experimental and the chance of failure is high, early results look promising. The company raised $259 million last month at a time when early-stage biotechs are struggling to tap the capital markets. It also recently struck a deal with the larger biotech

Vertex

to develop a treatment for liver disease, giving it yet another source of cash during turbulent times for small biotech companies.

Verve’s chief executive and co-founder is the cardiologist

Sekar Kathiresan,

a former professor at Harvard Medical School and former director of the Massachusetts General Hospital Center for Genomic Medicine. Dr. Kathiresan left academia to form a company inspired by his own discoveries around natural mutations that lower LDL.

While the gene-editing approach could in theory apply to anyone at risk of heart disease, Verve’s strategy for now is to focus on patients suffering from an inherited form of high cholesterol known as familial hypercholesterolemia, or FH, which increases the likelihood of having coronary heart disease at a younger age.

Earlier this summer a New Zealand patient suffering from the condition was the first to receive the injection. Another 39 patients are set to be tested with different doses over coming months, with results starting to read out next year. The injections previously reduced bad cholesterol in monkeys by over 60%.

It is a very long shot and will take years of testing, but the start of the process in New Zealand marked a step forward for the gene-editing space. Verve’s next step is to seek approval to begin trials in the U.S., where the Food and Drug Administration has set a high bar for conducting gene-editing trials where alternative therapies already exist. FDA-approved treatments for high cholesterol currently include not only statins but newer injections targeting PCSK9.

Dr. Kathiresan says Verve has been in talks with the FDA and is confident the company can meet the high bar for a trial in the U.S., though some analysts are skeptical. The company will submit a clinical trial application in the U.S. and the U.K. later this year.

“The bottom line right now is that despite all the treatments available, roughly 3% of patients with FH are actually at the right LDL level,” says Dr. Kathiresan. “So there’s still a huge unmet need, despite the fact that there’s all these medications out there.”

Even if Verve’s technology works, it would be up against recently approved injections. The shots from

Amgen Inc.

and

Sanofi SA

must be taken every few weeks while a new

Novartis

injection is taken twice a year. None of the drugs have so far become blockbusters, partly because insurers balked at their price and they require repeated injections to be effective. To win over insurers, doctors and patients, Verve would have to offer its therapy at an attractive price.

Kostas Biliouris, an analyst at BMO Capital Markets, says the company might be able to charge $90,000 for a one-time treatment, which is equivalent to about 15 years of PCSK9 injections at $6,000 yearly. The price could be lower and the market limited to a subset of the million or so patients with FH in the U.S. But, if the company were to expand into the broader heart-disease market, Mr. Biliouris reckons potential revenue could reach billions of dollars annually. Its market value net of cash is just below $2 billion today.

The other big challenge would be persuading patients to accept the risk of permanently altering their genes. Dr. Kathiresan says it is important to remember that this procedure doesn’t edit inheritable genomes but rather makes changes to a patient’s own body, like placing a stent.

“This kind of editing that we’re doing, it’s not that different from some surgical procedures because we’re not passing on the edit to future generations,” he says.

Verve’s technology is a scientific moonshot, at the forefront of a novel approach to medicine whose risks are still largely unclear. Verve’s stock has more than tripled from a June low of $11.14 but it is still trading at about half its peak of $74 last year when biotechnology was more in favor.

For those with appetite for some risk, though, the payoff could be large.

Write to David Wainer at david.wainer@wsj.com