[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"HuidaGene Therapeutics Co., Ltd.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":167},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,5,0,[8,49,75,109,139],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":26,"conditions":27,"keywords":29,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":38,"startDateStruct":41,"completionDateStruct":43,"leadSponsor":45,"locationsCount":48},"100597804","early-phase-1-an-clinical-study-evaluating-the-safety-tolerability-and-efficacy-of-hg005-in-stargardt-disease-100597804",false,"NCT07063251","An Clinical Study Evaluating the Safety, Tolerability, and efficAcy of HG005 in StaRgardT Disease","An Investigator-initiated, Open-label, Dose-escalation Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of HG005 in Pediatric Patients With Stargardt Disease (STGD1) Caused by Biallelic ABCA4 Mutations","START","Inclusion Criteria:\n\n* Patient ≥ 6 and ≤17 years at the time of signing informed consent, with clinical diagnosis of Stargardt disease;\n* At least one ABCA4 allele on each chromosome;\n* Both eyes must have well-defined macular atrophic lesions consistent with the diagnosis of Stargardt macular dystrophy.\n* Meet visual acuity criteria based on ETDRS letter chart\n* Subject must agree to contraception during the study.\n* Acceptable hematology, clinical chemistry, urine laboratory, and protocol required eye examination.\n\nExclusion Criteria:\n\n* Presence of active intraocular inflammation or uveitis history in either eye;\n* Presence of ocular or periocular infection history in either eye within 2 weeks prior to selection;\n* History or presence of corneal dystrophy in the study eye;\n* History of HIV or hepatitis A, B, or C infection;\n* Previous treatment with any gene therapy or cell therapy (e.g., stem cell transplantation);\n* Additional intraocular surgery in study eye 3 months prior to baseline visit;\n* Participation in an oral therapeutic STGD clinical trial within 3 months (or within 5 half-lives after last dose) prior to Screening\n* Any concomitant treatment that, in the opinion of the investigator, might interfere with the surgical procedure or healing process of the eye\n* Any other conditions that would not allow the potential subject to complete follow-up examinations during the study and would, in the opinion of the investigator, make the potential subject unsuitable for the study.","ALL","6 Years","17 Years",{"count":21,"type":22},6,"ESTIMATED","INTERVENTIONAL",[25],"EARLY_PHASE1","Stargardt disease type 1 (STGD1) is a rare genetic eye condition that causes progressive vision loss, often beginning in childhood or adolescence. It is the most common form of inherited macular degeneration and can lead to legal blindness. STGD1 is caused by mutations in the ABCA4 gene, which normally helps clear waste from the photoreceptor cells in the retina. When ABCA4 gene doesn't function properly, toxic substances like A2E accumulate and damage the retinal pigment epithelium (RPE), leading to vision loss.\n\nThere are currently no approved treatments for STGD1. HG005 is an investigational gene therapy designed to deliver a healthy copy of the ABCA4 gene to the retina. Because the gene is too large to fit into a single AAV (adeno-associated virus) vector, HG005 used two AAV vectors that work together in retinal cells to produce the full-length, functional ABCA4 protein. The goal of HG005 is to restore normal waste removal, protect retinal cells from further damage, and slow or stop vision loss.",[28],"Stargardt Disease Type 1 (STGD1)",[30,31,32,33,34,35],"Stargardt disease type 1 (STGD1)","Stargardt Macular Dystrophy","Stargardt Macular Degeneration","HG005","AAV","Gene Therapy","RECRUITING","2025-08-25",{"date":39,"type":40},"2025-08-27","ACTUAL",{"date":42,"type":40},"2025-08-20",{"date":44,"type":22},"2028-02-28",{"name":46,"class":47},"HuidaGene Therapeutics Co., Ltd.","INDUSTRY",1,{"id":50,"slug":51,"hasResults":11,"nctId":52,"briefTitle":53,"officialTitle":54,"acronym":4,"eligibilityCriteria":55,"healthyVolunteers":11,"sex":17,"minAge":56,"maxAge":57,"enrollmentInfo":58,"targetDuration":4,"studyType":23,"phases":60,"briefSummary":61,"conditions":62,"keywords":64,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":66,"lastUpdatePostDateStruct":67,"startDateStruct":69,"completionDateStruct":71,"leadSponsor":73,"locationsCount":74},"100518529","early-phase-1-crisprcas13-mediated-rna-targeting-therapy-for-the-treatment-of-neovascular-age-related-macular-degeneration-investigator-initiated-trial-sight-i-100518529","NCT06031727","CRISPR\u002Fcas13-medIated RNA TarGeting THerapy for the Treatment of Neovascular Age-related Macular Degeneration Investigator-initiated Trial (SIGHT-I)","A Trial to Evaluate the Safety, Tolerability, and Efficacy of CRISPR-Cas13 RNA-editing Therapy Targeting Knockdown of Vascular Endothelial Growth Factor a (HG202) in the Treatment of Neovascular Age-related Macular Degeneration (nAMD)","Inclusion Criteria:\n\n* Males or females ≥ 50 and ≤ 80 years at the time of signing the ICF\n* Diagnosed of choroidal neovascularization (CNV) secondary to AMD in the study eye;\n* Best-corrected visual acuity (BCVA) ranged from 73 to 23 early treatment diabetic retinopathy study (ETDRS)letter score (corresponding to 20\u002F32 to 20\u002F320 of Snellen visual acuity) in the study eye;\n* BCVA in the non-study eye had an ETDRS letter score of 19(equivalent to Snellen visual acuity20\u002F400) and above;\n* Able to perform visual acuity and retinal function tests and able and willing to comply with study procedures for this clinical trial;\n\nRESPONSIVE SUBJECTS:\n\n* History of need for and responsive to anti-VEGF therapy in the study eye\n\nNON-RESPONSIVE SUBJECTS:\n\n* History of receiving anti-VEGF therapy but is resistant to treatment, which is defined as: a. complete or near-complete remission of subretinal fluid after the initial 3 doses of anti-VEGF agents and thenno improvement (less than 50um reduction) or deterioration of CRT by OCT\n\nExclusion Criteria:\n\n* Subretinal hemorrhage, scarring, or fibrosis of greater than 50% of the total lesion in the study eye;\n* Any condition in the Investigator's opinion that could limit visual improvement in the study eye;\n* Other ocular diseases that may affect central vision in the study eye (e.g., retinal vein occlusion, retinal detachment, macular hole, optic nerve disease, etc.);\n* Presence of CNV not due to nAMD in the study eye,\n* Uncontrolled glaucoma in the study eye;\n* Active intraocular inflammation or a history of uveitis in either eye;\n* History or presence of corneal dystrophy in the study eye;\n* Subjects with immunodeficiency diseases prone to opportunistic infections;\n* History of other intraocular surgery in the study eye within 3 months prior to baseline that in the Investigator's opinion could impact healing or study outcome interpretation;\n* Prior gene therapy or oligonucleotide therapy;\n* History of acute coronary syndrome, myocardial infarction, coronary revascularization, cerebrovascular accident, or transient ischemic attack within 6 months prior to the Screening Visit;\n* Other conditions judged by the investigator as inappropriate for the study.","50 Years","80 Years",{"count":59,"type":22},12,[25],"Age-related macular degeneration (AMD) is a progressive disease leading to severe and irreversible vision loss of which the neovascular AMD (nAMD) accounted for 90% blindness in AMD. nAMD is primarily driven by the perturbation of vascular endothelial growth factor (VEGF). VEGF overexpression leads to abnormal growth of choroidal neovascularization (CNV), which is a hallmark of AMD. Although anti-VEGF agents are effective in treating nAMD, long-term efficacy decreases over time due to the need for repeated injections impacting patient compliance with treatment regimen while patients still may lose vision during the 7th or 8th year of treatment. These frequent intravitreal injections can increase the risk of complications, including submacular hemorrhage, intraocular hypertension, inflammation, and retinal detachment. Furthermore, there are up to 46% of nAMD patients using anti-VEGF agents who have shown poor response or have developed tachyphylaxis with anti-VEGF therapies. HG202 is a CRISPR\u002FCas13 RNA-editing therapy packaging novel high-fidelity Cas13 technology using one single AAV vector to partially knock-down the expression of VEGFA and thus inhibit CNV formation in AMD patients who are either responsive or non-responsive to anti-VEGF agents. The long-term, stable delivery of HG202 following a one (1) time gene-editing therapy treatment for nAMD could potentially reduce the frequent injection treatment burden of currently available therapies AND treat nAMD patients who are non-responsive to anti-VEGF therapies and have no treatment.",[63],"Neovascular Age-related Macular Degeneration(nAMD)",[65],"Macular Degeneration\u002FnAMD\u002FwAMD\u002FGene-editing\u002FCRISPR\u002FHG202","2025-03-12",{"date":68,"type":40},"2025-03-17",{"date":70,"type":40},"2023-09-04",{"date":72,"type":22},"2026-06-30",{"name":46,"class":47},2,{"id":76,"slug":77,"hasResults":11,"nctId":78,"briefTitle":79,"officialTitle":80,"acronym":81,"eligibilityCriteria":82,"healthyVolunteers":11,"sex":83,"minAge":84,"maxAge":85,"enrollmentInfo":86,"targetDuration":4,"studyType":23,"phases":87,"briefSummary":89,"conditions":90,"keywords":92,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":101,"lastUpdatePostDateStruct":102,"startDateStruct":104,"completionDateStruct":106,"leadSponsor":108,"locationsCount":48},"100563364","a-first-in-human-clinical-trial-to-evaluate-the-safety-tolerability-and-efficacy-of-a-novel-crispr-rna-editing-therapy-in-patients-with-mecp2-duplication-syndrome-a-rare-orphan-disease-hero-100563364","NCT06615206","A First-in-Human Clinical Trial to Evaluate the Safety, Tolerability, and Efficacy of a Novel CRISPR RNA-editing Therapy in Patients with Mecp2 Duplication Syndrome, a Rare Orphan Disease (HERO)","An Open-label, Multiple-dose Clinical Study to Evaluating the Safety, Tolerability and Preliminary Efficacy of a Single Intracerebroventricular Injection of HG204 for the Treatment of MECP2 Duplication Syndrome","HERO","Inclusion Criteria:\n\n* Males ≥ 2 and ≤18 years at the time of signing informed consent;\n* Genetic test and clinical confirmed diagnosis of MDS;\n* Stable pattern of seizures, or has had no seizures while currently receiving medical treatment (including antiepileptics) and physical therapy are stable for at least 2 months before screening;\n* Willing to adhere to protocol, including biological samples collection and hospitalization for intracerebroventricular injection surgery;\n* Acceptable hematology, clinical chemistry, and urine laboratory parameters.\n\nExclusion Criteria:\n\n* MECP2 gene triplication;\n* Concurrent genetic syndromes other than MDS;\n* Significant brain or cerebellar atrophy, or other significant degenerative changes as shown in cranial MRI at screening;\n* Prior or current hypertension, cardiomyopathy, myocardial ischemia or atrial fibrillation and other cardiovascular diseases;\n* Prior central nervous system surgery within 6 months before enrolment;\n* Systemic use of immunosuppressive drugs within 3 months before enrolment;\n* Prior gene therapy or oligonucleotide therapy treatments;\n* Any other conditions that would not allow the potential subject to complete follow-up examinations during the study and would, in the opinion of the investigator, make the potential subject unsuitable for the study.","MALE","2 Years","18 Years",{"count":21,"type":22},[88],"NA","Methyl-CpG binding protein 2 (MECP2) is a dosage-sensitive, X-linked gene critical for central nervous system development and functional maintenance, which gain-of-function causes MECP2 duplication syndrome (MDS). Affecting primarily in males, this disorder is characterized by severe intellectual disability, motor dysfunction, infantile hypotonia, epilepsy, respiratory tract infections, and premature death before 25 years of age with no curative therapy.\n\nHG204 is a CRISPR RNA-editing therapy packaging novel high-fidelity Cas13Y (hfCas13Y) technology, using one single adeno-associated virus (AAV) vector to target and knock down MECP2 mRNA in the brain. Preclinical studies showed that a single intracerebroventricular injection of HG204 persistently decreased MECP2 mRNA and MECP2 protein in the cortex of the MDS mice, reversed the abnormal motor and social phenotypes, and significantly prolonged survival in MDS mouse models.",[91],"MECP2 Duplication Syndrome",[93,94,95,96,97,98,99,100],"MECP2","dupMECP2","CNS","neurodevelopmental disease","Gene-editing","HG204","CRISPR","MDS","2024-11-22",{"date":103,"type":40},"2024-11-26",{"date":105,"type":40},"2024-10-30",{"date":107,"type":22},"2026-10-31",{"name":46,"class":47},{"id":110,"slug":111,"hasResults":11,"nctId":112,"briefTitle":113,"officialTitle":114,"acronym":115,"eligibilityCriteria":116,"healthyVolunteers":11,"sex":17,"minAge":56,"maxAge":117,"enrollmentInfo":118,"targetDuration":4,"studyType":23,"phases":120,"briefSummary":122,"conditions":123,"keywords":125,"overallStatus":130,"whyStopped":4,"lastUpdateSubmitDate":131,"lastUpdatePostDateStruct":132,"startDateStruct":134,"completionDateStruct":136,"leadSponsor":138,"locationsCount":4},"100563985","phase-1-open-label-dose-escalation-study-for-crisprcas13--rna-targeting-therapy-for-the-treatment-of-neovascular-age-related-macular-degeneration-in-phase-i-trial-100563985","NCT06623279","Open-laBel Dose-escalation Study for CRISPR\u002Fcas13- Rna TargetInG THerapy for the Treatment of Neovascular Age-related Macular Degeneration in Phase I Trial","A Phase 1, Open-label, Multiple-cohort, Dose-escalation Study to Evaluate the Safety and Tolerability of HG202 High-fidelity CRISPR-Cas13 (hfCas13Y) RNA-targeting Therapy for Neovascular Age-related Macular Degeneration (nAMD)","BRIGHT","Inclusion Criteria:\n\n* Males or females ≥ 50 and ≤ 85 years at the time of signing the ICF;\n* Active macular choroidal neovascularization (MNV) secondary to nAMD in the study eye;\n* Sentinel (1st) subject for each dose cohort must have a BCVA ≤ 20\u002F63 and ≥ 20\u002F400 (≤63 and ≥ 19 ETDRS letters) in the study eye. Following the sentinel subject evaluation, the rest of the subjects in the dose cohort must have a BCVA between ≤ 20\u002F40 and ≥ 20\u002F400 (≤ 73 and ≥ 19 ETDRS letters) in the study eye.\n* Able to perform visual acuity and retinal function tests and able and willing to comply with study procedures for this clinical trial.\n\nExclusion Criteria:\n\n* Retinal or subretinal hemorrhage, scarring, or fibrosis of greater than 50% of the total lesion in the study eye;\n* Other ocular diseases that may affect central vision in the study eye;\n* Any other cause of CNV than nAMD in the study eye\n* Uncontrolled glaucoma in the study eye;\n* History or presence of corneal transplant or corneal dystrophy in the study eye;\n* History of other intraocular surgery in the study eye within 3 months prior to baseline;\n* Prior gene therapy or oligonucleotide therapy;\n* Other conditions judged by the investigator as inappropriate for the study.","85 Years",{"count":119,"type":22},15,[121],"PHASE1","Age-related macular degeneration (AMD) leads to severe and irreversible vision loss, while neovascular AMD (nAMD) accounts for 80-90% of AMD blindness. Current anti-VEGF therapies are the standard of care, but these therapies require life-long repeated intraocular injections. These frequent intravitreal injections increase the risk of complications, including submacular hemorrhage, intraocular hypertension, inflammation, and retinal detachment. Therefore, repeated treatments for nAMD place a substantial burden on healthcare systems, patients, and their caregivers. Additionally, approximately 25-35% of individuals with aggressive nAMD show suboptimal responses to the anti-VEGF therapies, experience treatment-extended failure, or require intensive, frequent intraocular injections, and do not prevent irreversible vision loss.\n\nHG202 is a CRISPR\u002FCas13 RNA-editing therapy delivered through one single AAV vector to partially knock down the expression of VEGFA and thus inhibit CNV formation in AMD. The long-term, stable delivery of HG202 following a one-time gene-editing therapy treatment for nAMD may potentially reduce the frequent injections and the potential risks of currently available anti-VEGF therapies since it does not rely on the long-term expression of anti-VEGF antibodies.",[124],"Neovascular Age-Related Macular Degeneration (nAMD)",[126,127,128,97,99,129],"Macular Degeneration","nAMD","wAMD","HG202","NOT_YET_RECRUITING","2024-09-30",{"date":133,"type":40},"2024-10-02",{"date":135,"type":22},"2025-04-01",{"date":137,"type":22},"2031-02-01",{"name":46,"class":47},{"id":140,"slug":141,"hasResults":11,"nctId":142,"briefTitle":143,"officialTitle":144,"acronym":4,"eligibilityCriteria":145,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":56,"enrollmentInfo":146,"targetDuration":4,"studyType":23,"phases":148,"briefSummary":150,"conditions":151,"keywords":154,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":158,"lastUpdatePostDateStruct":159,"startDateStruct":161,"completionDateStruct":163,"leadSponsor":165,"locationsCount":166},"100508940","phase-1-safety-and-efficacy-trial-of-hg004-for-leber-congenital-amaurosis-related-to-rpe65-gene-mutations-star-100508940","NCT05906953","Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)","A Phase 1\u002F2, Open-label, Multi-national, Multiple-cohort, Dose-escalation Study to Evaluate the Safety, Tolerability, and Efficacy of HG004 Gene Therapy in Subjects with RPE65-associated Leber Congenital Amaurosis Type 2 (LCA2)","Inclusion Criteria:\n\n* Male or females between 6 and 50 years of age at the time of signing the informed consent form.\n* Willing to adhere to protocol as evidenced by written informed consent or parental permission and subject assent.\n* Clinical confirmed diagnosis of Leber congenital amaurosis (LCA) and molecular diagnosis of LCA due to RPE65 mutations.\n* Ability to perform tests of visual and retinal function.\n* Visual acuity of ≤ 20\u002F80 or visual field less than 20 degrees in the eye to be injected.\n* Acceptable hematology, clinical chemistry, and urine laboratory parameters.\n\nExclusion Criteria:\n\n* Pre-existing eye conditions that would preclude the planned surgery or interfere with interpretation of study endpoints or complications of surgery (e.g., glaucoma requiring upcoming surgery, corneal or significant lenticular opacities).\n* Presence of epiretinal membrane by OCT.\n* Complicating systemic diseases or clinically significant abnormal baseline laboratory values.\n* Complicating systemic diseases would include those in which the disease itself, or the treatment for the disease, can alter ocular function.\n* Prior ocular surgery within six months.\n* Prior gene therapy or oligonucleotide therapy treatments.\n* Any condition which leads the investigator to believe that the participant cannot comply with the protocol requirements or that may place the participant at an unacceptable risk for participation.",{"count":147,"type":22},20,[121,149],"PHASE2","The purpose of the study is to determine whether HG004 as gene therapy is safe and effective for the treatment of Leber Congenital Amaurosis caused by mutations in RPE65 gene.",[152,153],"Leber Congenital Amaurosis","Inherited Retinal Diseases Caused by RPE65 Mutations",[155,156,157],"IRD","LCA","LCA2","2024-09-13",{"date":160,"type":40},"2024-09-19",{"date":162,"type":40},"2023-10-31",{"date":164,"type":22},"2025-12",{"name":46,"class":47},3,""]