--- title: "Gene Editing Experiment Halted as Patient Gets Weird Side Effects" description: "Verve Therapeutics has paused the first phase of a human gene-editing drug trial aimed at reducing heart disease due to serious side effects." date: "2024-04-03" modified: "2024-04-03" authors: - name: "Maggie Harrison Dupré" job_title: "Senior Staff Writer" link: "https://futurism.com/authors/mharrison" url: "https://futurism.com/neoscope/gene-editing-experiment-side-effects" categories: - "Gene Editing" - "Genetics" - "Health & Medicine" tags: - "cholesterol" - "gene-editing" - "health" - "heart disease" --- # Gene Editing Experiment Halted as Patient Gets Weird Side Effects ![Verve Therapeutics has paused the first phase of a human gene-editing drug trial aimed at reducing heart disease due to serious side effects.]() *\Image: Getty / Futurism\* The experimental biotech startup Verve Therapeutics has paused the first phase of a buzzy human gene-editing trial due to strange side effects in a patient, [according to a report from ]()*[Bloomberg]().* The [trial]() in question — dubbed the "Heart-1" trial — is the company's attempt to use gene editing to reduce heart-attack-causing cholesterol in patients with familial hypercholesterolemia, a passed-down genetic disorder that causes buildups of LDL cholesterol in individuals. LDL is the [bad kind]() of cholesterol, and familial hypercholesterolemia drastically [heightens]() patients' risk of early heart attack and can lower life expectancy overall. Verve's proposed solution: inject "VERVE-101," a serum designed to lower fatty LDL molecules by genetically altering the cholesterol-managing PCSK9 gene, into **trial** participants' livers. While [promising tests]() on monkeys paved the way for Verve's much-anticipated human trial, however, Heart-1 has now hit another speed bump. Late last year, it was revealed that a patient enrolled in Heart-1 had passed away from a heart attack. A panel of experts concurred that VERVE-101 wasn't responsible — [according to *Nature*](), experts concluded that the patient's death was the result of their already-advanced heart disease — but coupled with some gnarly reported side effects, the tragic incident did raise some alarm bells. And now, some particularly bad side effects experienced by another Heart-1 participant have raised enough safety concerns to pause the trial altogether. Of the first six patients enrolled in the Heart-1 trial, according to *Bloomberg,* five saw their cholesterol levels decrease. Meanwhile, though, a sixth patient developed "abnormal liver enzymes" as well as [thrombocytopenia](), which is the medical term for a low blood platelet count in a patient. Thrombocytopenia is a serious condition that, in the worst cases, can lead to deadly internal bleeding. Thankfully, the patient's troubling symptoms reportedly disappeared after a few days off the drug. And per *Bloomberg*, Verve says it believes the abnormalities were caused by the fatty lipids found in the serum used to deliver VERVE-101 into patients' livers. So, in other words, the researchers think it's a problem with the *delivery* method, rather than the gene-editing process itself. According [to Verve's website,]() 13 patients in total were treated with VERVE-101. But now, due to the abnormal side effects, the biotech startup is turning its focus to VERVE-102, a version of the gene-editing drug suspended in a different lipid serum. Per Verve, this delivery system was "[well tolerated" by patients in a third-party clinical trial. This new trial, expectedly dubbed Heart-2, is set to start in the second quarter of this year.] Genetic heart conditions are an elusive and widespread problem, and if Verve can make a successful, one-and-done gene-editing treatment that alleviates some of these ailments, the company could change — and save — a lot of lives. While the Heart-1 trials did show some promise, though, it seems that Verve still has a way to go. **More on Heart-1:** [*Patient Dies after Being Gene-Edited to Have Lower Cholesterol*]() ## Author At Futurism, I've reported extensively on the rise of AI as a cultural and business force shaping the media industry, and more broadly how those dynamics are changing how we all consume and share information and relate to one another. I'm also fascinated by public health policy and ethics, the role of emerging tech in politics and governance — and the powerful people and forces at those intersections — climate change, and the environment. My investigation on Sports Illustrated's use of AI-generated authors with fictional biographies won a 2024 Mirror Award for "Best Story on Media Coverage of Artificial Intelligence in Journalism and the Media" from Syracuse University's SI Newhouse School, I contributed to Niemen Lab's 2025 Predictions for Journalism series, and I've discussed my work for Futurism during appearances on NPR, CNN, the BBC, the CBC, and more. I grew up in rural Pennsylvania and attended the University of Massachusetts Amherst, where I played Division I field hockey for the Minutewomen as a midfielder. Since then, I've lived in New Orleans, Louisiana and Manhattan, New York. I spend my free time running, reading, perusing archival fashion, and searching for the world’s best negroni. I also have a debonair tuxedo cat, Westley, who's named after "The Princess Bride." ### Author social links [Bluesky]()