[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"CorrectSequence Therapeutics Co., Ltd\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":116},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,45,69,96],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":34,"lastUpdatePostDateStruct":35,"startDateStruct":38,"completionDateStruct":39,"leadSponsor":41,"locationsCount":44},"100641104","early-phase-1-cs-121-apoc3-base-editing-in-severe-hypertriglyceridemia-100641104",false,"NCT07657780","CS-121 APOC3 Base Editing in Severe Hypertriglyceridemia","A Clinical Study to the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of CS-121, an In Vivo Base Editing Therapy Delivered by Lipid Nanoparticles Targeting APOC3 for the Treatment of Severe Hypertriglyceridemia in Adults","Inclusion Criteria:\n\n* Male or female participants aged 18 years ≤ age \\\u003C 65 years.\n* The serum triglyceride levels of the participants failed to be effectively controlled under the standard treatment regimen (or the medication that is available and tolerable as recommended by clinical practice) and were defined as having at least 3 records of different fasting triglyceride levels ≥ 5.65 mmol\u002FL (500 mg\u002FdL) within 2 years..\n* The screening period should include at least two different days with a TG level of ≥ 5.65 mmol\u002FL (500 mg\u002FdL), with an interval of at least 7 days.\n* Able to sign informed consent and comply with the requirements and restrictions specified in the informed consent form and the protocol.\n* Female participants must meet one of the following: be not of childbearing potential (e.g., documented hysterectomy, bilateral salpingectomy\u002Fsterilization, or ≥1 year postmenopausal); or, if of childbearing potential, have a negative pregnancy test at screening and be willing to use strict and effective contraception (e.g., abstinence, pharmacologic, or barrier methods) during the study. Male participants with reproductive potential must agree to use strict and effective contraception (e.g., abstinence, pharmacologic, or barrier methods) throughout the entire post-dose observation period; males without reproductive potential must provide supporting medical history (e.g., post-vasectomy).\n\nExclusion Criteria:\n\n* Currently participating in other interventional clinical studies, or having an insufficient washout period of less than 5 half-lives or 30 days (whichever is longer) since the last administration of other investigational drugs.\n* Used antisense oligonucleotide (ASO)-based or small interfering RNA (siRNA)-based lipid-lowering drugs targeting APOC3 within 3 months prior to study drug administration.\n* Requires long-term use of systemic corticosteroids and steroid drugs and cannot discontinue the medication.\n* Patients who experienced acute pancreatitis within 4 weeks prior dosing.\n* History of acute coronary syndrome (ACS) within 6 months before dosing, such as myocardial infarction or unstable angina, or prior coronary revascularization (such as coronary artery bypass grafting), angioplasty or stent implantation.\n* In the investigator's judgment to be unsuitable for the study drug due to receipt of major surgery within 3 months before dosing.\n* Any of the following laboratory abnormalities at screening:.\n* ALT or AST ≥2 × ULN;\n* Total bilirubin ≥2 × ULN;\n* eGFR \\\u003C30 mL\u002Fmin\u002F1.73 m²\n* HbA1c ≥9%;\n* Absolute neutrophil count \\\u003C 1.0 × 109\u002FL\n* Hemoglobin (female)\\\u003C 100 g\u002FL, Hemoglobin (male) \\\u003C 110 g\u002FL\n* Platelet count \\\u003C 100 × 109\u002FL\n* Coagulation function abnormalities judged by the investigator as unsuitable for CS-121 administration.\n* Positive results for HBsAg, dual positivity for HCV antibody and RNA, positive for HIV, or positive for Treponema pallidum infection.\n* Known major organ diseases, mental disorders, Cushing's syndrome, hypothyroidism, history of lymphoproliferative disorders, or malignant tumors in any organ system, which are judged by the investigator as unsuitable for study participation due to potential intolerance to adverse events such as cytokine-Release-Storm.\n* Concomitant medications\u002Ftreatments judged by the investigator to affect lipid metabolism, liver and kidney function, coagulation function, or interfere with the efficacy evaluation of the study drug.\n* Patients of childbearing potential who are planning pregnancy, breastfeeding, or have fertility plans.","ALL","18 Years","65 Years",{"count":20,"type":21},15,"ESTIMATED","INTERVENTIONAL",[24],"EARLY_PHASE1","This is an open-label, single-arm, dose-escalation IIT clinical trial to evaluate the safety, tolerability, pharmacodynamics (PD), and pharmacokinetics (PK) of CS-121, an in vivo base editing therapy delivered by lipid nanoparticles targeting APOC3, in adult participants (18-65 years) with Severe Hypertriglyceridemia(sHTG).",[27],"Severe Hypertriglyceridemia (sHTG)",[29,30,31,32],"Apolipoprotein C3 (APOC3)","In Vivo Base Editing","Lipid Nanoparticles (LNP)","Gene Editing Therapy","NOT_YET_RECRUITING","2026-06-15",{"date":36,"type":37},"2026-06-18","ACTUAL",{"date":34,"type":21},{"date":40,"type":21},"2028-07-31",{"name":42,"class":43},"CorrectSequence Therapeutics Co., Ltd","INDUSTRY",1,{"id":46,"slug":47,"hasResults":11,"nctId":48,"briefTitle":49,"officialTitle":50,"acronym":4,"eligibilityCriteria":51,"healthyVolunteers":11,"sex":16,"minAge":52,"maxAge":53,"enrollmentInfo":54,"targetDuration":4,"studyType":22,"phases":56,"briefSummary":58,"conditions":59,"keywords":4,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":61,"lastUpdatePostDateStruct":62,"startDateStruct":64,"completionDateStruct":66,"leadSponsor":67,"locationsCount":68},"100630555","phase-2-a-phase-2-safety-and-efficacy-study-evaluating-cs-101-in-participants-with--thalassemia-major-100630555","NCT07489196","A Phase 2 Safety and Efficacy Study Evaluating CS-101 in Participants With β-Thalassemia Major","A Single-arm, Open-label Phase II Clinical Trial: Evaluating the Safety and Efficacy of a Single Dose of CS-101 Injection in Participants With β-thalassemia Major","Inclusion Criteria:\n\n* Voluntarily signed informed consent. Male or female participants aged 12 to 35 years (inclusive). The participant or their legally authorized representative must sign the informed consent. If the participant is under 18 years of age, their legally authorized representative must also sign the informed consent.\n* Diagnosed with β-thalassemia major (transfusion-dependent). Received at least 8 units of red blood cell transfusions within 12 months prior to screening, and documented hemoglobin level ≤ 70 g\u002FL pre-transfusion.\n* Good general condition: Karnofsky score (≥16 years of age) ≥ 60, or Lansky Play-Performance score (\\\u003C16 years of age) ≥ 60.\n* For females of childbearing potential: From the start of the screening, highly effective contraception or complete abstinence (if this is their usual lifestyle), and agree to maintain such contraception throughout the study.\n* For males of childbearing potential: Use condoms or other methods to ensure effective contraception for sexual partners continuously from mobilization through the study period.\n\nExclusion Criteria:\n\n* Received other investigational products or other experimental interventions within 30 days prior to signing informed consent or within 6 elimination half-lives of the drug (whichever is longer).\n* Received or is receiving thalidomide, hydroxyurea, and\u002For luspatercept within 3 months prior to screening.\n* Previous received allogeneic hematopoietic stem cell transplantation, gene therapy, or gene-editing therapy; or participants who can be maintained with standard therapy.\n* Participants with a matched sibling donor, or with a matched unrelated \u002F haploidentical related donor and judged by the investigator to have no high-risk factors for allogeneic hematopoietic stem cell transplantation.\n* Participants with coexisting α-thalassemia with more than 2 α-globin chain gene deletions or non-deletional mutations.\n* Known hypersensitivity to drugs used during autologous hematopoietic stem cell transplantation, excipients, or devices, judged by the investigator to be ineligible for this study.\n* Infection with HIV, cytomegalovirus, Epstein-Barr virus, or Treponema pallidum during screening; active HBV or HCV infection (participants with stable hepatitis B after treatment (HBV-DNA negative) and cured hepatitis C (HCV-RNA negative) may be included). Known active bacterial, viral, fungal, or parasitic infection.\n* Echocardiographic ejection fraction \\\u003C 50%.\n* Laboratory abnormalities: AST or ALT \\> 3 × upper limit of normal (ULN); or International normalized ratio (INR) \\> 1.5 × ULN.\n* Cardiac severe iron overload detected by MRI during screening, judged by the investigator to be unsuitable for hematopoietic stem cell transplantation.\n* Current or history of malignancy.\n* Participants with known neurological consciousness disorders, psychological problems, or psychiatric diseases judged by the investigator to be unable to comply with study procedures.\n* Participants with known history of uncontrolled seizures judged by the investigator to be ineligible for this study.\n* Uncontrolled bleeding disorders.\n* Leukocyte count \\\u003C 3 × 10⁹\u002FL and\u002For platelet count \\\u003C 100 × 10⁹\u002FL not due to hypersplenism.\n* Participants with other severe cardiovascular, pulmonary, renal, gastrointestinal, hepatic diseases, and\u002For other organ disorders judged by the investigator to be ineligible for this study.\n* Pregnant or lactating females; females of childbearing potential with a positive serum pregnancy test.\n* Received live or live-attenuated vaccine within 90 days prior to myeloablation.\n* Participants with autoimmune diseases","12 Years","35 Years",{"count":55,"type":21},20,[57],"PHASE2","The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating patients with β-Thalassemia Major",[60],"β-thalassemia Major","2026-03-19",{"date":63,"type":37},"2026-03-24",{"date":65,"type":21},"2026-04-05",{"date":40,"type":21},{"name":42,"class":43},3,{"id":70,"slug":71,"hasResults":11,"nctId":72,"briefTitle":73,"officialTitle":74,"acronym":4,"eligibilityCriteria":75,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":76,"enrollmentInfo":77,"targetDuration":4,"studyType":22,"phases":78,"briefSummary":79,"conditions":80,"keywords":82,"overallStatus":87,"whyStopped":4,"lastUpdateSubmitDate":88,"lastUpdatePostDateStruct":89,"startDateStruct":91,"completionDateStruct":93,"leadSponsor":95,"locationsCount":44},"100606543","early-phase-1-cs-121-apoc3-base-editing-in-fcs-100606543","NCT07176923","CS-121 APOC3 Base Editing in FCS","A Clinical Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of CS-121, an In Vivo Base Editing Therapy Delivered by Lipid Nanoparticles Targeting APOC3, in Adults With Familial Chylomicronemia Syndrome","Inclusion Criteria:\n\n* Male or female aged 18 to 55 years (inclusive) at the time of signing informed consent.\n* On regular standard therapy with good compliance, but fasting triglyceride (TG) levels have not been consistently reduced below 10 mmol\u002FL (880 mg\u002FdL); i.e., before screening, there must be records of at least three separate fasting TG values \\>10 mmol\u002FL (880 mg\u002FdL), or the participant is intolerant to standard therapy.\n* North American Familial Chylomicronemia Syndrome (NAFCS) score ≥45\n* Able to sign informed consent and comply with the requirements and restrictions specified in the informed consent form and the protocol, such as dietary guidance and intake restrictions.\n* Female participants must meet one of the following: be not of childbearing potential (e.g., documented hysterectomy, bilateral salpingectomy\u002Fsterilization, or ≥1 year postmenopausal); or, if of childbearing potential, have a negative pregnancy test at screening and be willing to use strict and effective contraception (e.g., abstinence, pharmacologic, or barrier methods) during the study. Male participants with reproductive potential must agree to use strict and effective contraception (e.g., abstinence, pharmacologic, or barrier methods) throughout the entire post-dose observation period; males without reproductive potential must provide supporting medical history (e.g., post-vasectomy).\n\nExclusion Criteria:\n\n* Currently participating in another interventional clinical study, or last use of another investigational product with a washout period of less than 5 half-lives or 30 days (whichever is longer).\n* Use of APOC3-targeted antisense oligonucleotides (ASO) or siRNA lipid-lowering agents within 6 months prior to dosing.\n* History of acute pancreatitis within 3 months before dosing.\n* History of acute coronary syndrome (ACS) within 6 months before dosing, such as myocardial infarction or unstable angina, or prior coronary revascularization (e.g., coronary artery bypass grafting, angioplasty, or stent implantation).\n* In the investigator's judgment, receipt of major surgery within 3 months before dosing that would make the participant unsuitable for this study drug or unable to tolerate possible cytokine-release events.\n* Any of the following laboratory abnormalities at screening:\n\nALT or AST ≥3 × ULN Total bilirubin ≥1.5 × ULN eGFR \\\u003C30 mL\u002Fmin\u002F1.73 m² Random urine albumin-to-creatinine ratio (UACR) \\>30 mg\u002Fg LDL-C \\>130 mg\u002FdL (3.4 mmol\u002FL) HbA1c ≥9%\n\n* Coagulation abnormalities deemed by the investigator to make CS-121 administration unsuitable.\n* Positive at screening for HBsAg, HCV antibody and RNA, HIV, or Treponema pallidum (syphilis).\n* Known major organ disease, psychiatric disorders, Cushing's syndrome, or malignancy that, in the investigator's judgment, would make the participant unsuitable for the study or unable to tolerate possible cytokine-release-like events.\n* Concomitant medications or treatments judged by the investigator to affect lipid metabolism, hepatic or renal function, or coagulation, or to interfere with the evaluation of study drug efficacy, including but not limited to: systemic glucocorticoids, anabolic steroids, antiretroviral agents used within 4 weeks prior to dosing, plasma exchange, blood donation, or blood loss \\>200 mL.\n* Planning pregnancy, currently pregnant, or breastfeeding.","55 Years",{"count":20,"type":21},[24],"This is an open-label, single-arm, dose-escalation Phase I clinical trial to evaluate the safety, tolerability, pharmacodynamics (PD), and pharmacokinetics (PK) of CS-121, an in vivo base editing therapy delivered by lipid nanoparticles targeting APOC3, in adult participants (18-55 years) with familial chylomicronemia syndrome (FCS).",[81],"Familial Chylomicronemia Syndrome (FCS)",[81,83,84,29,30,31,32,85,86],"Severe Hypertriglyceridemia","Acute Pancreatitis","First-in-Human Study","Rare Metabolic Disorder","RECRUITING","2026-02-08",{"date":90,"type":37},"2026-02-11",{"date":92,"type":37},"2025-10-15",{"date":94,"type":21},"2026-12",{"name":42,"class":43},{"id":97,"slug":98,"hasResults":11,"nctId":99,"briefTitle":100,"officialTitle":101,"acronym":4,"eligibilityCriteria":102,"healthyVolunteers":11,"sex":16,"minAge":52,"maxAge":53,"enrollmentInfo":103,"targetDuration":4,"studyType":22,"phases":105,"briefSummary":106,"conditions":107,"keywords":4,"overallStatus":87,"whyStopped":4,"lastUpdateSubmitDate":88,"lastUpdatePostDateStruct":109,"startDateStruct":111,"completionDateStruct":113,"leadSponsor":115,"locationsCount":44},"100559507","early-phase-1-cs-206-in-patients-with-sickle-cell-disease-100559507","NCT06565026","CS-206 in Patients With Sickle Cell Disease","An Open-Label Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CD34+ Human Hematopoietic Stem Cells Modified Using Transformer Base Editor in Participants With Severe Sickle Cell Disease","Inclusion Criteria:\n\n* Participants must be between 12 to 35 years old (inclusive). Participants or their legal guardians (for participants below 18 years old) must provide written informed consent before any study-related procedures.\n* Participants must have a Documented βS\u002FβS, βS\u002Fβ0 or βS\u002Fβ+ genotype.\n* Participants must have at least one of the following conditions\n\n  1. At least 2 occurrences of any of the following events within 2 years prior to screening.\n\n     1. Acute pain crisis: requiring a visit to a medical facility and administration of pain medications (opioids or intravenous NSAIDs) or red blood cell transfusions.\n     2. Acute chest syndrome: defined by the presence of a new pulmonary infiltrate on a chest X-ray, associated with pneumonia-like symptoms, including chest pain, fever, or respiratory distress.\n     3. Priapism lasting more than 2 hours and necessitating a visit to a medical facility for intervention.\n     4. Stroke or transient ischemic attack (TIA): confirmed by imaging studies (e.g., MRI or CT scan), including silent stroke, and overt stroke leading to neurological deficits lasting \\>24 hours.\n  2. Presence of red cell alloimmunization (\\>2 antibodies) and the need for ongoing chronic transfusions.\n  3. Participants who have failed, not tolerated, refused the standard of care for Sickle Cell Disease (SCD), or are unable to access the standard of care due to the availability\n  4. Other situations deemed appropriate for hematopoietic stem cell transplantation according to the sickle cell anemia treatment guidelines, as determined by the investigator.\n* Laboratory Parameters:\n\n  1. Documented Hemoglobin S (HbS) level ≥30% of total hemoglobin (Hb) concentration prior to transfusion.\n  2. HbF at screening \\\u003C 20%\n* Participants must have a Karnofsky Performance Status (KPS for participants above 16 years old, inclusive) or Lansky Play-Performance Scale (LPPS for participants below 16 years old) score of ≥70, indicating sufficient functional status to undergo the intervention.\n* Willing to comply with the protocol requirements, use contraception as required, attend regular follow-up visits, and cooperate with examinations.\n\nExclusion Criteria:\n\n* Female participants who are pregnant, breastfeeding, or planning pregnancy during the study period are excluded.\n* Participation in another investigational drug trial within 30 days prior to screening or within 5 half-lives (whichever is longer).\n* Subjects who have received or are receiving luspatercept treatment within 3 months prior to screening.\n* Subjects who have previously received any gene therapy for the disease.\n* Subjects with a fully matched related donor who are already scheduled for allogeneic hematopoietic stem cell transplantation.\n* More than 10 unplanned hospitalizations or emergency visits within 12 months prior to screening, which the investigator believes are related to significant chronic pain rather than acute pain crisis (VOC).\n* Severe liver dysfunction:\n\n  1. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \\>3× the upper limit of normal (ULN) or:\n  2. International Normalized Ratio (INR) \\>1.5× ULN\n* Severe renal impairment (creatinine clearance \\\u003C30 mL\u002Fmin\u002F1.73 m²) are excluded.\n* Subjects with HIV, cytomegalovirus (CMV), Epstein-Barr virus (EBV), or Treponema pallidum infection during the screening period; those with active HBV or HCV infection; or known tuberculosis or parasitic infection, etc. Excludes subjects with stable hepatitis B (HBV-DNA negative) after treatment and those cured of hepatitis C (HCV-RNA negative). Known active bacterial, viral, or fungal infections.\n* Deemed unsuitable for autologous hematopoietic stem cell transplantation procedures as determined by the investigator.\n* Other situations deemed unsuitable for this study as determined by the investigator.",{"count":104,"type":21},5,[24],"The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 injection in treating sickle cell disease.",[108],"Sickle Cell Disease",{"date":110,"type":37},"2026-02-10",{"date":112,"type":37},"2024-09-02",{"date":114,"type":21},"2027-06-30",{"name":42,"class":43},""]