Trial reportSignal transduction and targeted therapy2024
Gene replacement therapy in Bietti crystalline corneoretinal dystrophy: an open-label, single-arm, exploratory trial.
5 registry-linked trialsOpen access · goldAbstract readClinical Trial
In one paragraph
Trial report in Signal transduction and targeted therapy, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 5 registered trials, which are not on this map. Cited by 20 papers, 1 of them a synthesis that pooled it.
0numbers the graph read from it
0cells of the map it votes in
20citing papers in PubMed, 1 pooled it
4.9field-weighted citation impact, top 4% of its field
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Safety Trial of rAAV2/8-hCYP4V2 Gene Replacement Therapy Drug Administered as a Single Subretinal Injection in Patients With Bietti's Crystalline Dystrophy (BCD)
A Phase I/II Clinical Study to Evaluate the Safety and Efficacy of ZVS101e Administered as a Single Monocular Subretinal Injection in Subjects With Bietti's Crystalline Dystrophy (BCD)
A Multicenter, Randomized，Controlled，Phase 3 Clinical Trial to Evaluate the Efficacy and Safety of ZVS101e Administered as a Single Subretinal Injection in Subjects With Bietti's Crystalline Dystrophy (BCD)
NCT07307469 naenrolling by invitationnot on this mapstarted 2026, after this paper: background citation
Compassionate Administration of ZVS101e for Extended Treatment
Clinical Translation and Application Protocol for ZVS101e in the Gene Therapy of Bietti Crystalline Corneoretinal Dystrophy
Who cites it
20 citing papers in PubMed, 1 synthesis or guideline pooled it, 21 citations in OpenAlex.
- Retinal Viral Gene Therapy: Impact of Route of Administration on Serious Adverse Events-A Systematic Review.Clinical & experimental ophthalmology · 2025Pooled it
- Retinoic acid in health and disease.Signal transduction and targeted therapy · 2026Review
- Development and Validation of a Method to Determine CYP4V2 Enzyme Activity in rAAV-hCYP4V2 Gene Therapy Products Using a Bioluminescent Substrate Assay.Molecules (Basel, Switzerland) · 2026Article
- Phase I/II Study of NGGT001 Gene Therapy for Bietti Crystalline Dystrophy.Ophthalmology science · 2026Article
- Development and Validation of a Quantitative LC-MS/MS Method for Measuring CYP4V2 Enzyme Activity via 12-Hydroxylauric Acid in rAAV-hCYP4V2 Gene Therapy Products.Molecules (Basel, Switzerland) · 2026Article
- Designs of the clinical trials aiming at evaluating cell and gene therapy products: A critical appraisal from a literature review.Molecular therapy. Advances · 2026Review
- The role of full-field stimulus threshold in evaluating Bietti crystalline dystrophy.Graefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie · 2026Observational
- Exploration and Application of Multiluminance Mobility Test in Bietti Crystalline Dystrophy.Translational vision science & technology · 2026Article
- Efficient derivation of hiPSC-derived photoreceptor precursor cells and their neuroprotective effects in retinal degeneration.iScience · 2025Article
- Bilateral retinal rescue through unilateral AAV8-CYP4V2 gene delivery in Bietti crystalline dystrophy.Molecular therapy. Methods & clinical development · 2025Article
- Base and Prime Editing for Inherited Retinal Diseases: Delivery Platforms, Safety, Efficacy, and Translational Perspectives.Pharmaceutics · 2025Review
- Visual Neurorestoration: An Expert Review of Current Strategies for Restoring Vision in Humans.Brain sciences · 2025Review
- One down but many more to go: the state of gene therapy for inherited retinal disease.Regenerative medicine · 2025Review
- A Comprehensive Review of Clinically Applied Adeno-Associated Virus-Based Gene Therapies for Ocular Disease.Human gene therapy · 2025Review
- The clinical safety landscape for ocular AAV gene therapies: A systematic review and meta-analysis.iScience · 2025Article
- Safety and Vision Outcomes Following Gene Therapy for Bietti Crystalline Dystrophy: A Nonrandomized Clinical Trial.JAMA ophthalmology · 2025Article
- Bietti crystalline corneoretinal dystrophy: Advances in understanding and gene therapeutic approaches.Molecular vision · 2025Review
- Recombinant adeno-associated virus as a delivery platform for ocular gene therapy: A comprehensive review.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Review
- Ocular Gene Therapy: An Overview of Viral Vectors, Immune Responses, and Future Directions.The Yale journal of biology and medicine · 2024Review
- Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
24 authors at 2 institutions in 1 country.
Jinyuan WangBeijing Tongren Eye Center, Beijing Key Laboratory of Intraocular Tumor Diagnosis and Treatment, Beijing Ophthalmology & Visual Sciences Key Lab, Medical Artificial Intelligence Research and Verification Key Laboratory of the Ministry of Industry and Information Technology, Beijing Tongren Hospital, Capital Medical University, 100730, Beijing, China.ORCID 0000-0002-8378-1249
Jinlu ZhangChigenovo Co., Ltd., 102206, Beijing, China.
Shicheng YuDepartment of Ophthalmology, Peking University Third Hospital, Beijing Key Laboratory of Restoration of Damaged Ocular Nerve, Peking University Third Hospital, 100191, Beijing, China.
Hongyan LiChigenovo Co., Ltd., 102206, Beijing, China.
Shaohong ChenChigenovo Co., Ltd., 102206, Beijing, China.
Jingting LuoBeijing Tongren Eye Center, Beijing Key Laboratory of Intraocular Tumor Diagnosis and Treatment, Beijing Ophthalmology & Visual Sciences Key Lab, Medical Artificial Intelligence Research and Verification Key Laboratory of the Ministry of Industry and Information Technology, Beijing Tongren Hospital, Capital Medical University, 100730, Beijing, China.
Haibo WangBeijing Tongren Eye Center, Beijing Key Laboratory of Intraocular Tumor Diagnosis and Treatment, Beijing Ophthalmology & Visual Sciences Key Lab, Medical Artificial Intelligence Research and Verification Key Laboratory of the Ministry of Industry and Information Technology, Beijing Tongren Hospital, Capital Medical University, 100730, Beijing, China.
Yuxia GuanChigenovo Co., Ltd., 102206, Beijing, China.
Haihan ZhangBeijing Tongren Eye Center, Beijing Key Laboratory of Intraocular Tumor Diagnosis and Treatment, Beijing Ophthalmology & Visual Sciences Key Lab, Medical Artificial Intelligence Research and Verification Key Laboratory of the Ministry of Industry and Information Technology, Beijing Tongren Hospital, Capital Medical University, 100730, Beijing, China.
Shiyi YinBeijing Tongren Eye Center, Beijing Key Laboratory of Intraocular Tumor Diagnosis and Treatment, Beijing Ophthalmology & Visual Sciences Key Lab, Medical Artificial Intelligence Research and Verification Key Laboratory of the Ministry of Industry and Information Technology, Beijing Tongren Hospital, Capital Medical University, 100730, Beijing, China.ORCID 0000-0001-9119-0302
Huili WangChigenovo Co., Ltd., 102206, Beijing, China.
Heping LiChigenovo Co., Ltd., 102206, Beijing, China.
Junle LiuChigenovo Co., Ltd., 102206, Beijing, China.
Jingyuan ZhuBeijing Tongren Eye Center, Beijing Key Laboratory of Intraocular Tumor Diagnosis and Treatment, Beijing Ophthalmology & Visual Sciences Key Lab, Medical Artificial Intelligence Research and Verification Key Laboratory of the Ministry of Industry and Information Technology, Beijing Tongren Hospital, Capital Medical University, 100730, Beijing, China.
Qiong YangBeijing Tongren Eye Center, Beijing Key Laboratory of Intraocular Tumor Diagnosis and Treatment, Beijing Ophthalmology & Visual Sciences Key Lab, Medical Artificial Intelligence Research and Verification Key Laboratory of the Ministry of Industry and Information Technology, Beijing Tongren Hospital, Capital Medical University, 100730, Beijing, China.
Ying ShaBeijing Tongren Eye Center, Beijing Key Laboratory of Intraocular Tumor Diagnosis and Treatment, Beijing Ophthalmology & Visual Sciences Key Lab, Medical Artificial Intelligence Research and Verification Key Laboratory of the Ministry of Industry and Information Technology, Beijing Tongren Hospital, Capital Medical University, 100730, Beijing, China.
Chuan ZhangBeijing Tongren Eye Center, Beijing Key Laboratory of Intraocular Tumor Diagnosis and Treatment, Beijing Ophthalmology & Visual Sciences Key Lab, Medical Artificial Intelligence Research and Verification Key Laboratory of the Ministry of Industry and Information Technology, Beijing Tongren Hospital, Capital Medical University, 100730, Beijing, China.
Yuhang YangBeijing Tongren Eye Center, Beijing Key Laboratory of Intraocular Tumor Diagnosis and Treatment, Beijing Ophthalmology & Visual Sciences Key Lab, Medical Artificial Intelligence Research and Verification Key Laboratory of the Ministry of Industry and Information Technology, Beijing Tongren Hospital, Capital Medical University, 100730, Beijing, China.
Xuan YangBeijing Tongren Eye Center, Beijing Key Laboratory of Intraocular Tumor Diagnosis and Treatment, Beijing Ophthalmology & Visual Sciences Key Lab, Medical Artificial Intelligence Research and Verification Key Laboratory of the Ministry of Industry and Information Technology, Beijing Tongren Hospital, Capital Medical University, 100730, Beijing, China.
Xifang ZhangBeijing Tongren Eye Center, Beijing Key Laboratory of Intraocular Tumor Diagnosis and Treatment, Beijing Ophthalmology & Visual Sciences Key Lab, Medical Artificial Intelligence Research and Verification Key Laboratory of the Ministry of Industry and Information Technology, Beijing Tongren Hospital, Capital Medical University, 100730, Beijing, China.
Xiuli ZhaoBeijing Tongren Eye Center, Beijing Key Laboratory of Intraocular Tumor Diagnosis and Treatment, Beijing Ophthalmology & Visual Sciences Key Lab, Medical Artificial Intelligence Research and Verification Key Laboratory of the Ministry of Industry and Information Technology, Beijing Tongren Hospital, Capital Medical University, 100730, Beijing, China.
Likun WangDepartment of Biomedical Informatics, School of Basic Medical Sciences, Peking University Health Science Center, 100191, Beijing, China. wanglk@pku.edu.cn.ORCID 0000-0002-0933-2808
Liping YangDepartment of Ophthalmology, Peking University Third Hospital, Beijing Key Laboratory of Restoration of Damaged Ocular Nerve, Peking University Third Hospital, 100191, Beijing, China. alexlipingyang@bjmu.edu.cn.
Wenbin WeiBeijing Tongren Eye Center, Beijing Key Laboratory of Intraocular Tumor Diagnosis and Treatment, Beijing Ophthalmology & Visual Sciences Key Lab, Medical Artificial Intelligence Research and Verification Key Laboratory of the Ministry of Industry and Information Technology, Beijing Tongren Hospital, Capital Medical University, 100730, Beijing, China. weiwenbintr@163.com.ORCID 0000-0003-2386-0989
Beijing Tongren Hospital · CNPeking University · CN
Funding
Beijing Municipal Science and Technology Commission Z181100001818003Beijing Municipal Science and Technology Commission Z201100005520045Beijing Municipal Science and Technology Commission Z231100004823021Capital Health Research and Development of Special 2020-1-2052National Natural Science Foundation of China (National Science Foundation of China) 82141128National Natural Science Foundation of China (National Science Foundation of China) 82220108017National Natural Science Foundation of China (National Science Foundation of China) 82371074Natural Science Foundation of Beijing Municipality (Beijing Natural Science Foundation) J230031
Abstract
Bietti crystalline corneoretinal dystrophy is an inherited retinal disease caused by mutations in CYP4V2, which results in blindness in the working-age population, and there is currently no available treatment. Here, we report the results of the first-in-human clinical trial (NCT04722107) of gene therapy for Bietti crystalline corneoretinal dystrophy, including 12 participants who were followed up for 180-365 days. This open-label, single-arm exploratory trial aimed to assess the safety and efficacy of a recombinant adeno-associated-virus-serotype-2/8 vector encoding the human CYP4V2 protein (rAAV2/8-hCYP4V2). Participants received a single unilateral subretinal injection of 7.5 × 10
Indexed as
Corneal Dystrophies, HereditaryCytochrome P450 Family 4DependovirusGenetic TherapyRetinal DiseasesAdultFemaleGenetic VectorsHumansMaleMiddle AgedVisual AcuityCYP4V2 protein, humanCytochrome P450 Family 4
Identifiers
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LicenceCC BY
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Registered trials
NCT04722107ClinicalTrials.gov
NCT05832684ClinicalTrials.gov
NCT06743646ClinicalTrials.gov
NCT07307469ClinicalTrials.gov
NCT07653971ClinicalTrials.gov
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.