Administered to first Patient With Chylomicronemia, CorrectSequence Therapeutics’ CS-121 Demonstrates Excellent Safety Profile and Meaningful Efficacy

SHANGHAI, Chine, November 9, 2025 /PRNewswire/ — On November 6, 2025, in Shanghai, China, CorrectSequence Therapeutics Co., Ltd.(Correctseq), a clinical-stage biotechnology company pioneering transformative Base Editing (tBE) technology for the treatment of serious diseases, announced that the first patient in its investigator-initiated trial (EII) CS-121 base editing therapy targetingAPOC3 for the treatment of chylomicronemia/hypertriglyceridemia had responded well to the dosage and was released from the hospital.

The patient diagnosed with chylomicronemia had long-standing fasting triglyceride (TG) levels above 12.5 mmol/L and recurrent acute pancreatitis. in the CS-121 dose escalation EII, his fasting TG level dropped substantially within three days of administering a single low dose, without any adverse effects.

It is indeed about world-wide first successful clinical treatment of hyperlipidemia based on gene editing therapy targetingAPOC3.

Picture: The world’s first patient of the gene-editing therapy targeting APOC3 (Correctseq’s CS-121) for hyperlipidemia (the 6th from the right) has successfully completed dosing and been discharged.

Photo : The world’s first patient to receive gene editing therapy targetingAPOC3 (Correctseq’s CS-121) for the treatment of hyperlipidemia (6th from right) responded well to the dosage and was discharged from the hospital.

Chylomicronemia is a metabolic disorder characterized by an abnormally high

Groundbreaking Gene Editing Treatment for Triglyceride Levels Shows Promise

A new gene editing therapy, utilizing CRISPR-Cas9 technology, is demonstrating significant potential in treating familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia. This marks a major step forward in the submission of gene editing for chronic metabolic diseases. The treatment,developed by Verve Therapeutics,aims to permanently lower triglyceride levels by disabling the APOC3 gene,which plays a key role in regulating triglyceride metabolism.

Understanding the Problem: High Triglycerides and APOC3

high triglyceride levels in the blood are a major risk factor for cardiovascular disease,including heart attacks and strokes. familial chylomicronemia syndrome (FCS) is a rare, inherited disorder characterized by extremely high triglyceride levels, frequently enough exceeding 1,000 mg/dL. This can lead to acute pancreatitis, a painful and potentially life-threatening inflammation of the pancreas.

The APOC3 gene provides instructions for making apolipoprotein C-III, a protein that inhibits the breakdown of triglycerides. By reducing the activity of APOC3, the body can more efficiently clear triglycerides from the bloodstream. Current treatments for FCS, such as fibrates and omega-3 fatty acids, often have limited efficacy and can cause side effects. In some cases, apheresis (a blood-filtering procedure) is required, which is burdensome for patients.

How CRISPR-Cas9 Gene Editing Works

CRISPR-Cas9 is a revolutionary gene editing technology that allows scientists to precisely target and modify DNA sequences. Think of it like a molecular pair of scissors. Here’s a simplified breakdown:

• CRISPR: Stands for Clustered Regularly Interspaced Short Palindromic Repeats. These are DNA sequences originally found in bacteria as a defense mechanism against viruses.
• Cas9: An enzyme that acts like the “scissors,” cutting DNA at a specific location guided by the CRISPR sequence.
• Guide RNA: A short RNA sequence designed to match the target DNA sequence (in this case, a portion of the APOC3 gene). It directs the Cas9 enzyme to the correct location in the genome.

Once the DNA is cut, the cell’s natural repair mechanisms kick in. in the Verve Therapeutics treatment,the goal is to disrupt the APOC3 gene,effectively turning it off. This is achieved through a process called non-homologous end joining (NHEJ), which often introduces small insertions or deletions that render the gene non-functional.

Clinical Trial Results and Safety Profile

Initial results from a Phase 1 clinical trial, presented in November 2025, are highly encouraging. The trial is evaluating the safety and efficacy of a single intravenous infusion of the CRISPR-Cas9 therapy. The first patient, a 63-year-old man, received a low dose on October 18, 2025. Within three days, his fasting triglyceride levels dropped significantly, and he was discharged from the hospital three days after treatment.

Preclinical studies in animals demonstrated a strong safety profile and long-term efficacy. Importantly,no off-target modifications (unintended edits to othre parts of the genome) were detected in vital organs,including the liver,lungs,muscles,spleen,ovaries,heart,and kidneys. This is a critical aspect of gene editing safety, as off-target effects could potentially lead to harmful consequences.

Future outlook and Potential Impact

While these early results are promising, further research is needed to confirm the long-term safety and efficacy of this gene editing therapy. Ongoing clinical trials will enroll more patients and assess the durability of the triglyceride-lowering effect. If successful, this treatment could offer a potentially curative option for individuals with FCS and severe hypertriglyceridemia, significantly reducing their risk of cardiovascular events and pancreatitis.

This breakthrough also paves the way for the development of gene editing therapies for other genetic diseases, offering hope for millions of people worldwide.

Key Takeaways

• A new CRISPR-Cas9 gene editing therapy is showing promise in treating high triglyceride levels.
• The treatment targets the APOC3 gene, which regulates triglyceride metabolism.
• Initial clinical trial results demonstrate significant triglyceride reduction and a favorable safety profile.
• This therapy has the potential to be a curative option for individuals with familial chylomicronemia syndrome and severe hypertriglyceridemia.

Publication Date: 2025/11/09 09:17:12