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Eli Lilly's gene editing therapy cuts LDL cholesterol by 62% & Biogen/Denali fail Parkinson’s trial

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Joachim Eeckhout & Eduardo Longoria
May 26, 2026

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SNAPSHOT

Eli Lilly prepares phase 2 launch after gene editor cuts LDL cholesterol by 62%

Lilly’s one-time gene-editing therapy, VERVE-102, reduced LDL (“bad”) cholesterol by up to 62% in a phase 1b trial, matching or exceeding the efficacy of leading PCSK9 inhibitor drugs while potentially offering a permanent treatment.

Why it matters: Cardiovascular disease remains the world’s leading cause of death, and many patients stop taking chronic cholesterol medications within a year. A durable, one-and-done therapy could transform treatment for inherited high cholesterol disorders like heterozygous familial hypercholesterolemia (HeFH).

Backstory: VERVE-102 uses base editing to permanently disable the PCSK9 gene in liver cells. Earlier Verve programs targeting the same gene encountered liver toxicity issues tied to lipid nanoparticle delivery systems. The newer version uses a redesigned delivery approach incorporating GalNAc sugar molecules to improve liver targeting and safety.

Big picture: Gene editing is expanding beyond rare diseases into massive cardiovascular markets. Companies including CRISPR Therapeutics, Mammoth Biosciences and Scribe Therapeutics are racing to develop in vivo editing therapies for heart disease and metabolic disorders.

Zoom in: The phase 1b Heart-2 study has enrolled 35 patients with inherited high cholesterol or premature coronary artery disease. Patients receiving the highest tested dose (1 mg/kg) saw LDL reductions sustained for up to 18 months. Existing PCSK9 drugs such as Repatha typically lower LDL by 50%–60%. Analysts say maintaining reductions above 50% with a favorable safety profile is considered the benchmark for clinical success.

What’s next: Lilly plans to begin enrolling a phase 2 trial by the end of 2026. Regulators and clinicians will closely watch long-term safety, durability and which patient populations are best suited for permanent gene editing. The strongest commercial opportunity may be younger HeFH patients facing lifelong cardiovascular risk. However, questions remain about whether older patients or those with moderate cholesterol elevations would accept the risks and cost of irreversible genome editing.

SNIPPETS

What’s happening in biotech today?

🧠 Parkinson’s pause: Biogen and Denali Therapeutics announced that their experimental Parkinson’s disease drug, BIIB122, failed a mid-stage clinical trial involving nearly 650 patients, showing no significant benefit over placebo in slowing disease progression or improving secondary outcomes. The drug targets the LRRK2 enzyme, linked to one of the most common genetic causes of Parkinson’s disease. While exploratory analyses suggested the therapy engaged its intended target, the companies decided to discontinue development for idiopathic Parkinson’s disease. Denali will continue a separate study focused on patients with LRRK2-linked Parkinson’s.

💊 Pill power: Novo Nordisk has received a positive recommendation from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) for approval of an oral version of Wegovy (semaglutide 25 mg) for weight management in adults with obesity or overweight and at least one related condition. If approved by the European Commission, it would become the first oral GLP-1 receptor agonist authorized for weight management in the EU. The recommendation is based on results from the OASIS clinical program, including a Phase IIIb trial showing significant weight reduction and cardiovascular benefits.

🔑 Keytruda combo: OSE Immunotherapeutics announced positive topline Phase II results for its cancer vaccine Tedopi in recurrent ovarian cancer, particularly when combined with Merck & Co.’s Keytruda (pembrolizumab). In the TEDOVA trial, the Tedopi-Keytruda combination improved median progression-free survival to 4.1 months versus 2.8 months with best supportive care in patients with platinum-sensitive recurrent ovarian cancer who had progressed after or were ineligible for PARP inhibitors and bevacizumab.

🚀 SPAC attack: RA Capital Management has raised $75 million through the IPO of its new special-purpose acquisition company (SPAC), Research Alliance III, which aims to merge with a biotechnology or healthcare company, potentially based in China. Led by RA Capital partner Matthew Hammond, the entity follows two previous RA-sponsored SPACs, one of which merged with Point Biopharma before its later acquisition by Eli Lilly, while the other liquidated without completing a deal. The fundraising reflects renewed interest in biotech SPACs amid a sluggish traditional IPO market and challenging financing conditions for early-stage biotech companies.

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