[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Precision BioSciences, Inc.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":81},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,51],{"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":26,"conditions":27,"keywords":29,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":40,"lastUpdatePostDateStruct":41,"startDateStruct":44,"completionDateStruct":46,"leadSponsor":48,"locationsCount":5},"100625946","phase-1-pbgene-dmd-phase-12a-safety-and-preliminary-efficacy-study-in-duchenne-muscular-dystrophy-function-dmd-100625946",false,"NCT07429240","PBGENE-DMD Phase 1\u002F2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)","A Phase 1\u002F2a, Multi-center, Open-label Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of PBGENE-DMD in Participants With Duchenne Muscular Dystrophy (FUNCTION-DMD)","Inclusion Criteria:\n\n1. Males, 2 to 7 years of age, inclusive, at the time of informed consent\u002Fassent\n2. Molecular confirmed DMD diagnosis (DMD mutation fully contained between exons 45 to 55 \\[inclusive\\])\n3. Clinical phenotype consistent with DMD in the opinion of the Investigator\n4. Ability to complete age-appropriate motor testing assessments requirements.\n\n   Participants aged 2 to \\\u003C 4 years at the time of screening must:\n   1. Be able to walk at least 10 meters independently (without assistive devices).\n   2. Be able to rise from the floor without physical assistance (use of a Gowers' maneuver is acceptable).\n\n      Participants aged 4 to 7 years at the time of screening must:\n   3. Be able to walk at least 100 meters independently (without assistive devices).\n   4. Have an NSAA total score between 16 and 29, inclusive.\n5. Participant has received age-appropriate routine childhood immunizations per the local country's national immunization schedule.\n6. The participant's parent(s)\u002FLAR(s) are willing and able to provide written informed consent prior to the initiation of any trial-specific procedures; where applicable, the participant must provide written or verbal assent in accordance with local regulations.\n7. The participant and their parent(s)\u002FLAR(s) are willing to participate in a LTFU study after the completion of this trial.\n\nExclusion Criteria:\n\n1. Prior treatment with any gene therapy, gene editing therapy, or cell-based therapy at any time.\n2. Receipt of any investigational medication or experimental therapy within 6 months prior to Day 1.\n3. Prior or ongoing use of any product designed to increase dystrophin expression, investigational, or otherwise, including exon-skipping therapies, within 6 months of the scheduled Day 1 dose or inability or unwillingness to refrain from initiating or resuming these therapies for at least 5 years following gene therapy administration.\n4. Prior ongoing use of any product designed to increase dystrophin expression, investigational, or otherwise, including exon-skipping therapies, within 6 months of the scheduled Day 1 dose.\n5. Concurrent enrollment in another clinical trial, unless it is observational (non-interventional).\n6. A positive test for antibodies to AAV9\n7. A participant has any condition that would contraindicate treatment with immunosuppression.\n8. Participants with pathogenic mutations in exons 1-44 and\u002For exons 56-79.\n9. Evidence of cardiomyopathy or clinically significant left ventricular dysfunction, defined as LVEF \\\u003C50% on screening echocardiogram.","MALE","2 Years","7 Years",{"count":20,"type":21},18,"ESTIMATED","INTERVENTIONAL",[24,25],"PHASE1","PHASE2","The purpose of this Phase 1\u002F2a trial is to evaluate the safety, tolerability, and preliminary efficacy of PBGENE-DMD in patients with DMD harboring mutations amenable to excision of exons 45-55. Given the limitations of existing therapeutic strategies, PBGENE-DMD represents a novel, innovative approach with the potential for a one-time, durable correction of the underlying genetic defect in the largest molecular subset of patients with DMD.",[28],"Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD",[30,31,32,33,34,35,36,37,38],"Duchenne muscular dystrophy","DMD","X-linked Muscle wasting disease","Muscular dystrophy","Progressive muscle weakness","Gene editing","AAV serotype 9","AAV-9","Exon 45-55 excision","RECRUITING","2026-06-22",{"date":42,"type":43},"2026-06-23","ACTUAL",{"date":45,"type":43},"2026-04-24",{"date":47,"type":21},"2029-12",{"name":49,"class":50},"Precision BioSciences, Inc.","INDUSTRY",{"id":52,"slug":53,"hasResults":11,"nctId":54,"briefTitle":55,"officialTitle":56,"acronym":57,"eligibilityCriteria":58,"healthyVolunteers":11,"sex":59,"minAge":60,"maxAge":61,"enrollmentInfo":62,"targetDuration":4,"studyType":22,"phases":64,"briefSummary":65,"conditions":66,"keywords":68,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":72,"lastUpdatePostDateStruct":73,"startDateStruct":75,"completionDateStruct":77,"leadSponsor":79,"locationsCount":80},"100568362","phase-1-phase-1-study-to-evaluate-safety-and-antiviral-activity-of-pbgene-hbv-in-adult-patients-with-chronic-hepatitis-b-100568362","NCT06680232","Phase 1 Study to Evaluate Safety and Antiviral Activity of PBGENE-HBV in Adult Patients With Chronic Hepatitis B","A Phase 1, Open-Label, First-in-Human, Dose Escalation (Part 1) and Expansion (Part 2) Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Antiviral Activity of PBGENE-HBV in Participants With Chronic Hepatitis B (ELIMINATE-B)","ELIMINATE-B","Key Inclusion Criteria:\n\n* Male or women of non-child bearing potential\n* BMI 18.0 to 35.0\n* Good overall health deemed by the study Investigator\n* CHB infection documented at least 12 months prior to screening\n* HBeAg-negative CHB\n* Must be virologically suppressed on current NA treatment\n\nKey Exclusion Criteria:\n\n* No history of cirrhosis of the liver\n* No current infections of Hepatitis A, D, and E, human immunodeficiency virus (type 1 and 2), and no history of or current hepatitis C. In addition, no other active infections deemed clinically relevant.\n* No signs of hepatocellular carcinoma\n* Not received an organ transplant\n* No malignancy within 5 years of screening, except for specific cancers that are cured by surgical resection (e.g., basal cell skin cancer)\n* No investigational agent received within 6 months of screening","ALL","18 Years","70 Years",{"count":63,"type":21},45,[24],"This is a Phase 1, open-label, dose escalation and dose expansion study to evaluate the safety, tolerability, PK, and antiviral activity of PBGENE-HBV in adult participants with chronic hepatitis B.",[67],"HEPATITIS B CHRONIC",[67,69,70,71],"Gene Therapy","Gene Editing","PBGENE-HBV","2025-09-22",{"date":74,"type":43},"2025-09-26",{"date":76,"type":43},"2024-11-14",{"date":78,"type":21},"2026-12",{"name":49,"class":50},4,""]