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The World's First Gene-Editing Therapy Targeting APOC3 for Hyperlipidemia

TECH

The World's First Gene-Editing Therapy Targeting APOC3 for Hyperlipidemia
TECH

TECH

The World's First Gene-Editing Therapy Targeting APOC3 for Hyperlipidemia

2025-11-06 08:00 Last Updated At:11-07 17:31

CorrectSequence Therapeutics' CS-121 Completed Dosing of First Chylomicronemia Patient, Demonstrating Excellent Safety and Significant Efficacy

SHANGHAI, Nov. 6, 2025 /PRNewswire/ -- On November 6, 2025, Shanghai, China, CorrectSequence Therapeutics Co., Ltd. (Correctseq), a clinical-stage biotechnology company pioneering transformer Base Editing (tBE) technology for the treatment of severe diseases, announced that the first patient in its Investigator-Initiated Trial (IIT) of the base-editing therapy CS-121 targeting APOC3 for chylomicronemia / hypertriglyceridemia has successfully completed dosing and been discharged from the hospital. 

The patient, diagnosed with chylomicronemia, had a long history of fasting triglyceride (TG) levels exceeding 12.5 mmol/L and recurrent acute pancreatitis. In the dose-escalation IIT for CS-121, his fasting TG level dropped significantly within three days after a single low-dose administration, with no adverse events.

This is the world's first successful clinical treatment of hyperlipidemia with the gene-editing therapy targeting APOC3.

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.

Chylomicronemia is a metabolic disorder characterized by abnormally elevated chylomicrons in the blood, associated with lipid metabolism dysfunction, leading to extremely high fasting TG levels and potentially life-threatening complications such as acute pancreatitis. It is the most severe subtype of severe hypertriglyceridemia (sHTG), including Familial Chylomicronemia Syndrome (FCS) and Multifactorial Chylomicronemia Syndrome (MCS). FCS is a rare autosomal recessive disorder caused by biallelic mutations in the Lipoprotein Lipase (LPL) gene or other key regulatory genes, with fasting TG ≥10 mmol/L (885 mg/dL) and a global prevalence of 1 in 100,000–1,000,000. MCS results from a complex interaction of genetic, lifestyle, or metabolic disorders with a prevalence as high as 1 in 600 worldwide.

Current treatments for chylomicronemia primarily aim to control fasting TG levels below the acute pancreatitis risk threshold (<5.7 mmol/L or 500 mg/dL), but available triglyceride-lowering medications are often insufficient, and very-low-fat diets are hard to maintain in a long-term manner.

Scientific studies have shown that the APOC3 protein, produced in the liver, plays a central role in TG regulation. Large-scale population analyses have shown that individuals carrying natural APOC3 loss-of-function mutations have significantly lower TG levels without adverse effects. With advances in gene-editing technologies, it is now possible to therapeutically modulate APOC3 expression at the genetic level to lower TG levels — offering a potential curative strategy for chylomicronemia and hypertriglyceridemia.

CS-121, Correctseq's first in vivo gene-editing therapy for chylomicronemia and hypertriglyceridemia, is based on the transformer Base Editor (tBE) — a highly precise base-editing system independently developed by Correctseq's scientific co-founders. Administered via intravenous injection, tBE is delivered via lipid nanoparticles (LNPs) to the liver and precisely edits the target APOC3 gene. It mimics beneficial natural APOC3 loss-of-function variants to downregulate APOC3 expression and effectively lower plasma TG levels. By addressing the disease at the genetic level, this therapy aims to achieve "one-time treatment, lifelong efficacy."

CS-121 utilizing the next-generation tBE technology, which enables precise single-base correction without DNA double-strand breaks, offering superior safety over the gene-editing therapies based on CRISPR. tBE avoids potential safety risks such as p53 activation, chromosomal damage, off-target effects, and liver toxicity caused by DNA double-strand breaks. Preclinical animal studies showed excellent safety and long-term efficacy, with no off-target editing detected in various organs including liver, lungs, muscle, spleen, ovaries, heart, and kidneys.

The first patient, a 63-year-old male, received a single low-dose intravenous administration on October 18, 2025. His fasting TG levels dropped significantly within three days after the treatment, and he was discharged three days post-treatment with no treatment-related adverse events to date.

The principal investigators of the CS-121 IIT are Professor Huan Zhou and Doctor Zhili Wu from the First Affiliated Hospital of Anhui Medical University.

Correctseq, an innovative biotechnology company at the IND clinical stage, previously developed CS-101, an ex vivo gene-editing therapy that has successfully treated dozens of patients with β-thalassemia and sickle cell disease. The company is advancing the first in vivo gene-editing therapy CS-121 toward IND clinical trials and commercialization, aiming to offer "one-time treatment, lifelong efficacy" treatment for patients with chylomicronemia, hypertriglyceridemia, and other metabolic disorders.

Acknowledgments: The First Affiliated Hospital of Anhui Medical University, ShanghaiTech University, Shanghai Clinical Research and Trial Center.

About CorrectSequence Therapeutics
CorrectSequence Therapeutics (Correctseq), incubated at ShanghaiTech University, is dedicated to leveraging innovative gene-editing technologies to transform the lives of people with severe diseases. The company has developed multiple state-of-the-art base-editing systems that offer exceptional precision, minimize off-target effects, and enhance in vivo editing efficiency. Its robust pipeline spans genetic disorders, metabolic diseases, and cardiovascular conditions, with several programs already advancing toward clinical development.

For more information, visit www.correctsequence.com.

Media Contact:
Business Cooperate: BD@correctsequence.com
Clinical Trial Recruitment: CT@correctsequence.com

** The press release content is from PR Newswire. Bastille Post is not involved in its creation. **

The World's First Gene-Editing Therapy Targeting APOC3 for Hyperlipidemia

The World's First Gene-Editing Therapy Targeting APOC3 for Hyperlipidemia

Two-Day Summit at Rosewood Amsterdam, October 7–8, 2026, to Convene the World's Leading Litigation Funders, Law Firms, General Counsels, and Institutional Investors

AMSTERDAM, April 1, 2026 /PRNewswire/ -- LITFINCON, the global litigation finance conference series created by Silstone Capital, today announced the launch of LITFINCON Europe, a two-day summit bringing the world's most influential litigation finance community together in Amsterdam on October 7–8, 2026. The event will be held at Rosewood Amsterdam, one of the city's most distinguished luxury hotels, marking LITFINCON's formal entry into the European market. The Rosewood Amsterdam provides a fitting backdrop for the conference as the historic building once served as Amsterdam's Palace of Justice – the city's main courthouse. LITFINCON Europe will reestablish this venue as a vibrant hub for legal dialogue and networking.

LITFINCON Europe follows the series' debut events in Houston and the upcoming inaugural Asia edition at Marina Bay Sands in Singapore on June 4, 2026. The European edition expands LITFINCON into the world's most dynamic litigation finance regulatory environment, as the UK, EU, and individual member states grapple with transformative questions about disclosure requirements, funder returns, and the future of third-party funding across the continent.

 "The regulatory environment is evolving rapidly across the UK and EU, capital is flowing into the space at an unprecedented rate, and the demand for high-quality, senior dialogue has never been higher," said Robert Le, Co-Founder of Siltstone Capital.

Conference Program
Across eleven panels over two days, LITFINCON Europe moves from the macro to the granular — opening with a global market overview before examining the regulatory divergence reshaping how funders, law firms, general counsels, and institutional investors operate across the UK, EU, and US. The program addresses the structural mechanics of European deal-making, including the distinct dynamics of loser-pays cost regimes, ATE insurance integration, and portfolio financing, alongside deep dives into collective redress, international arbitration, and cross-border enforcement.

The second day turns to the investment and innovation questions defining the industry's next chapter: how institutional allocators — pension funds, sovereign wealth funds, and family offices — are assessing European litigation finance funds; the convergence of ATE insurance and third-party funding; the Unified Patent Court's emergence as a major new venue for funded IP disputes; and the competitive implications of AI adoption under GDPR and EU AI Act constraints. The conference closes with an unscripted, 75-minute Candid Conversations session — no slides, no prepared remarks — where industry veterans reflect honestly on what Europe gets right, wrong, and differently from the rest of the world.

Venue: Rosewood Amsterdam
Rosewood Amsterdam occupies a series of five interconnected 19th-century palace buildings along the Herengracht, one of Amsterdam's most storied canal rings. Its location in the heart of Amsterdam's historic center positions it as one of Europe's premier settings for high-level conferences and private events.

"LITFINCON has built its reputation on bringing the right people into the right room. The Rosewood hotel in Amsterdam reflects the standard we set for every event in this series — an environment where senior professionals can have honest, substantive conversations away from the noise," said Jim Batson, CIO – Legal Finance, Siltstone Capital.

Sponsorship and Participation
Sponsorship packages, speaking opportunities, and registration for LITFINCON Europe 2026 are now available.

For sponsorship, speaking, and registration for LITFINCON Europe, contact: info@litfincon.com or visit www.litfinconeurope.com.

For sponsorship, speaking, and registration for LITFINCON Asia, contact: info@litfincon.com or visit www.litfinconasia.com

About LITFINCON
LITFINCON is the premier global conference series dedicated to the continued evolution of litigation finance as an institutional asset class.  Convening the world's leading funders, law firms, institutional investors, general counsels, and legal minds, LITFINCON serves as the platform for substantive dialogue, capital formation, and strategic connectivity across the global legal finance ecosystem. Created by Siltstone Capital, LITFINCON originated in Houston and has rapidly scaled into a multi-city global series spanning the United States, Asia, and Europe. LITFINCON Asia will take place on June 4, 2026 at Marina Bay Sands in Singapore and LITFINCON Europe will take place on October 7–8, 2026 at Rosewood Amsterdam in the Netherlands.

Media Contact
Jacob Varghese
LITFINCON
info@litfincon.com
www.litfinconeurope.com
www.litfinconasia.com

Two-Day Summit at Rosewood Amsterdam, October 7–8, 2026, to Convene the World's Leading Litigation Funders, Law Firms, General Counsels, and Institutional Investors

AMSTERDAM, April 1, 2026 /PRNewswire/ -- LITFINCON, the global litigation finance conference series created by Silstone Capital, today announced the launch of LITFINCON Europe, a two-day summit bringing the world's most influential litigation finance community together in Amsterdam on October 7–8, 2026. The event will be held at Rosewood Amsterdam, one of the city's most distinguished luxury hotels, marking LITFINCON's formal entry into the European market. The Rosewood Amsterdam provides a fitting backdrop for the conference as the historic building once served as Amsterdam's Palace of Justice – the city's main courthouse. LITFINCON Europe will reestablish this venue as a vibrant hub for legal dialogue and networking.

LITFINCON Europe follows the series' debut events in Houston and the upcoming inaugural Asia edition at Marina Bay Sands in Singapore on June 4, 2026. The European edition expands LITFINCON into the world's most dynamic litigation finance regulatory environment, as the UK, EU, and individual member states grapple with transformative questions about disclosure requirements, funder returns, and the future of third-party funding across the continent.

 "The regulatory environment is evolving rapidly across the UK and EU, capital is flowing into the space at an unprecedented rate, and the demand for high-quality, senior dialogue has never been higher," said Robert Le, Co-Founder of Siltstone Capital.

Conference Program
Across eleven panels over two days, LITFINCON Europe moves from the macro to the granular — opening with a global market overview before examining the regulatory divergence reshaping how funders, law firms, general counsels, and institutional investors operate across the UK, EU, and US. The program addresses the structural mechanics of European deal-making, including the distinct dynamics of loser-pays cost regimes, ATE insurance integration, and portfolio financing, alongside deep dives into collective redress, international arbitration, and cross-border enforcement.

The second day turns to the investment and innovation questions defining the industry's next chapter: how institutional allocators — pension funds, sovereign wealth funds, and family offices — are assessing European litigation finance funds; the convergence of ATE insurance and third-party funding; the Unified Patent Court's emergence as a major new venue for funded IP disputes; and the competitive implications of AI adoption under GDPR and EU AI Act constraints. The conference closes with an unscripted, 75-minute Candid Conversations session — no slides, no prepared remarks — where industry veterans reflect honestly on what Europe gets right, wrong, and differently from the rest of the world.

Venue: Rosewood Amsterdam
Rosewood Amsterdam occupies a series of five interconnected 19th-century palace buildings along the Herengracht, one of Amsterdam's most storied canal rings. Its location in the heart of Amsterdam's historic center positions it as one of Europe's premier settings for high-level conferences and private events.

"LITFINCON has built its reputation on bringing the right people into the right room. The Rosewood hotel in Amsterdam reflects the standard we set for every event in this series — an environment where senior professionals can have honest, substantive conversations away from the noise," said Jim Batson, CIO – Legal Finance, Siltstone Capital.

Sponsorship and Participation
Sponsorship packages, speaking opportunities, and registration for LITFINCON Europe 2026 are now available.

For sponsorship, speaking, and registration for LITFINCON Europe, contact: info@litfincon.com or visit www.litfinconeurope.com.

For sponsorship, speaking, and registration for LITFINCON Asia, contact: info@litfincon.com or visit www.litfinconasia.com

About LITFINCON
LITFINCON is the premier global conference series dedicated to the continued evolution of litigation finance as an institutional asset class.  Convening the world's leading funders, law firms, institutional investors, general counsels, and legal minds, LITFINCON serves as the platform for substantive dialogue, capital formation, and strategic connectivity across the global legal finance ecosystem. Created by Siltstone Capital, LITFINCON originated in Houston and has rapidly scaled into a multi-city global series spanning the United States, Asia, and Europe. LITFINCON Asia will take place on June 4, 2026 at Marina Bay Sands in Singapore and LITFINCON Europe will take place on October 7–8, 2026 at Rosewood Amsterdam in the Netherlands.

Media Contact
Jacob Varghese
LITFINCON
info@litfincon.com
www.litfinconeurope.com
www.litfinconasia.com

** This press release is distributed by PR Newswire through automated distribution system, for which the client assumes full responsibility. **

LITFINCON Announces European Debut: Global Litigation Finance Conference Series Comes to Amsterdam

LITFINCON Announces European Debut: Global Litigation Finance Conference Series Comes to Amsterdam

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