^
    6d
    Impact of COL7A1 Gene Therapy on SCC Recurrence in RDEB Skin (clinicaltrials.gov)
    P2, N=16, Not yet recruiting, Stanford University
    New P2 trial • Gene therapy
    9d
    HGB-210: A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease (clinicaltrials.gov)
    P3, N=35, Active, not recruiting, bluebird bio | Recruiting --> Active, not recruiting
    Enrollment closed • Gene therapy • Viral vector
    9d
    Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene (clinicaltrials.gov)
    P3, N=97, Completed, Janssen Research & Development, LLC | Active, not recruiting --> Completed
    Trial completion • Gene therapy
    9d
    Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials (clinicaltrials.gov)
    P3, N=175, Recruiting, Novartis Pharmaceuticals | N=260 --> 175 | Trial completion date: Oct 2039 --> Jun 2030 | Trial primary completion date: Oct 2039 --> Jun 2030
    Enrollment change • Trial completion date • Trial primary completion date
    10d
    A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP) (clinicaltrials.gov)
    P2, N=42, Recruiting, Janssen Research & Development, LLC | Not yet recruiting --> Recruiting
    Enrollment open • Gene therapy
    15d
    A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa (clinicaltrials.gov)
    P3, N=4, Recruiting, Janssen Pharmaceutical K.K. | Trial completion date: Feb 2030 --> May 2030
    Trial completion date
    16d
    Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN) (clinicaltrials.gov)
    P1/2, N=28, Recruiting, Biocad | N=12 --> 28
    Enrollment change
    17d
    An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency (OTC-HOPE) (clinicaltrials.gov)
    P1/2, N=13, Recruiting, iECURE, Inc. | Active, not recruiting --> Recruiting
    Enrollment open
    20d
    the Safety and Efficacy Evaluation of HGI-001 Injection in Patients With Transfusion-Dependent β-Thalassemia (clinicaltrials.gov)
    P1, N=3, Recruiting, Shenzhen Hemogen | Trial completion date: Aug 2025 --> Dec 2025 | Trial primary completion date: Aug 2024 --> Dec 2025
    Trial completion date • Trial primary completion date
    23d
    BEYOND-9: A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B (clinicaltrials.gov)
    P1/2, N=130, Recruiting, Regeneron Pharmaceuticals | Trial completion date: Aug 2032 --> Dec 2032 | Trial primary completion date: Aug 2032 --> Dec 2032
    Trial completion date • Trial primary completion date
    23d
    Safety and Efficacy Study of NGGT001 in Bietti Crystalline Corneoretinal Dystrophy Subjects (clinicaltrials.gov)
    P1/2, N=12, Active, not recruiting, NGGT (Suzhou) Biotechnology Co., Ltd.
    New P1/2 trial
    28d
    Study to Evaluate the Efficacy and Safety of VGR-R01 Gene Therapy in Patients with Bietti Crystalline Dystrophy (clinicaltrials.gov)
    P3, N=45, Not yet recruiting, Shanghai Vitalgen BioPharma Co., Ltd.
    New P3 trial • Gene therapy
    28d
    MAGNOLIA: An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients with Hemophilia B (clinicaltrials.gov)
    P3, N=24, Not yet recruiting, Biocad
    New P3 trial
    1m
    Phase 3, Open-label Study to Assess the Efficacy and Safety of a Single Lumbar Intrathecal Administration of MELPIDA in Individuals with Hereditary Spastic Paraplegia Type 50 (SPG50) Versus Matched Prospective Concurrent Controls. (clinicaltrials.gov)
    P3, N=24, Not yet recruiting, Elpida Therapeutics SPC
    New P3 trial
    1m
    MyPEAK-1: Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM (clinicaltrials.gov)
    P1/2, N=30, Recruiting, Tenaya Therapeutics | Phase classification: P1 --> P1/2 | N=15 --> 30 | Trial completion date: Dec 2029 --> Aug 2032 | Trial primary completion date: Dec 2025 --> Aug 2027
    Phase classification • Enrollment change • Trial completion date • Trial primary completion date
    1m
    SUNRISE-FA: Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia (clinicaltrials.gov)
    P1/2, N=8, Active, not recruiting, Lexeo Therapeutics | Recruiting --> Active, not recruiting
    Enrollment closed • Gene therapy
    1m
    CT-AMT-130-02: Safety and Efficacy of AMT-130 in European Adults with Early Manifest Huntington's Disease (clinicaltrials.gov)
    P1/2, N=14, Active, not recruiting, UniQure Biopharma B.V. | Recruiting --> Active, not recruiting
    Enrollment closed
    |
    CHI3L1 (Chitinase 3-like 1) • GFAP (Glial Fibrillary Acidic Protein) • NEFL (Neurofilament Light Chain)
    1m
    Safety and Efficacy of RRG001 Gene Therapy in Subjects With Neovascular Age-related Macular Degeneration (nAMD) (clinicaltrials.gov)
    P1/2, N=48, Recruiting, Shanghai Refreshgene Technology Co., Ltd. | Trial primary completion date: Dec 2027 --> Apr 2026
    Trial primary completion date • Gene therapy
    1m
    Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects (clinicaltrials.gov)
    P1/2, N=18, Recruiting, NGGT (Suzhou) Biotechnology Co., Ltd.
    New P1/2 trial
    1m
    GATEWAY: A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease (clinicaltrials.gov)
    P1/2, N=4, Active, not recruiting, Vivet Therapeutics SAS | Recruiting --> Active, not recruiting | N=16 --> 4 | Trial completion date: Mar 2029 --> Jun 2029
    Enrollment closed • Enrollment change • Trial completion date
    1m
    HOLOGENE 5: Open-label, Pivotal Clinical Trial to Confirm Efficacy and Safety of Autologous Grafts Containing Stem Cells Genetically Modified for Epidermis Restoration in Patients With Junctional Epidermolysis Bullosa (clinicaltrials.gov)
    P2/3, N=2, Active, not recruiting, Holostem Terapie Avanzate s.r.l. | Recruiting --> Active, not recruiting | Trial completion date: Jun 2024 --> Dec 2024
    Enrollment closed • Trial completion date
    1m
    A Multicenter, Single-Arm, Open-Label Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Intrathecal of SNUG01 in Patients with Amyotrophic Lateral Sclerosis (ALS) (ChiCTR2400090090)
    P=N/A, N=7, Not yet recruiting, Peking University Third Hospital; Peking University Third Hospital
    New trial
    1m
    RST-001 Phase I/II Trial for Advanced Retinitis Pigmentosa (clinicaltrials.gov)
    P1/2, N=14, Completed, AbbVie | Active, not recruiting --> Completed
    Trial completion • Metastases
    2ms
    AATD: NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease (clinicaltrials.gov)
    P1/2, N=30, Recruiting, Intellia Therapeutics | Not yet recruiting --> Recruiting
    Enrollment open
    2ms
    Oral Hydroxychloroquine (HCQ) for Retinitis Pigmentosa Caused by P23H- Rhodopsin (RHO) (clinicaltrials.gov)
    P1/2, N=8, Terminated, University of Michigan | N=12 --> 8 | Trial completion date: Jan 2026 --> Aug 2024 | Recruiting --> Terminated | Trial primary completion date: Jan 2026 --> Aug 2024; Unable to recruit additional participants after multiple extensions to planned enrollment period.
    Enrollment change • Trial completion date • Trial termination • Trial primary completion date
    |
    hydroxychloroquine
    2ms
    JAG-201-CL-101: JAG201 Gene Therapy Study in Children & Adults with SHANK3 Haploinsufficiency (clinicaltrials.gov)
    P1/2, N=6, Not yet recruiting, Jaguar Gene Therapy, LLC
    New P1/2 trial
    2ms
    A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients with Glycogen Storage Disease Type Ia (GSDIa) (clinicaltrials.gov)
    P3, N=52, Recruiting, Ultragenyx Pharmaceutical Inc | Active, not recruiting --> Recruiting
    Enrollment open
    2ms
    A Long-term Extension Study of JNJ-81201887 (AAVCAGsCD59) Parent Studies in Participants With Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD) (clinicaltrials.gov)
    P2, N=311, Recruiting, Janssen Research & Development, LLC | Trial completion date: Aug 2029 --> Dec 2029
    Trial completion date
    2ms
    A Study to Evaluate Intravitreal JNJ-81201887 (AAVCAGsCD59) Compared to Sham Procedure for the Treatment of Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD) (clinicaltrials.gov)
    P2, N=305, Active, not recruiting, Janssen Research & Development, LLC | Recruiting --> Active, not recruiting
    Enrollment closed
    |
    prednisone
    2ms
    Study of ARO-DUX4 in Adult Patients With Facioscapulohumeral Muscular Dystrophy Type 1 (clinicaltrials.gov)
    P1, N=60, Recruiting, Arrowhead Pharmaceuticals | Trial completion date: Jun 2025 --> Sep 2025
    Trial completion date
    2ms
    Evaluating Safety and Efficacy of Lentiviral-transduced CD34+ HSCs in Β-thalassaemia Patients. (clinicaltrials.gov)
    P1, N=8, Recruiting, Shenzhen Hemogen
    New P1 trial
    2ms
    Acclaim-1: Quaratusugene Ozeplasmid (Reqorsa) and Osimertinib in Patients With Advanced Lung Cancer Who Progressed on Osimertinib (clinicaltrials.gov)
    P1/2, N=158, Recruiting, Genprex, Inc. | Trial completion date: Dec 2027 --> Mar 2029 | Trial primary completion date: Dec 2026 --> Mar 2028
    Trial completion date • Trial primary completion date • Combination therapy • Metastases
    |
    EGFR (Epidermal growth factor receptor)
    |
    EGFR mutation
    |
    cisplatin • Tagrisso (osimertinib) • carboplatin • Reqorsa (quaratusugene ozeplasmid)
    2ms
    Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia (clinicaltrials.gov)
    P1, N=25, Recruiting, Weill Medical College of Cornell University | N=10 --> 25
    Enrollment change • Gene therapy
    |
    AFP (Alpha-fetoprotein)
    |
    prednisone
    2ms
    A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP) (clinicaltrials.gov)
    P2, N=42, Not yet recruiting, Janssen Research & Development, LLC
    New P2 trial • Gene therapy
    2ms
    ASCENT: Pivotal 2 Study of RGX-314 Gene Therapy in Participants With nAMD (clinicaltrials.gov)
    P3, N=660, Recruiting, AbbVie | N=465 --> 660 | Trial completion date: Dec 2025 --> Nov 2026 | Trial primary completion date: Feb 2025 --> Nov 2025
    Enrollment change • Trial completion date • Trial primary completion date • Gene therapy
    |
    Eylea (aflibercept intravitreal)
    2ms
    A Long-term Extension Study of JNJ-81201887 (AAVCAGsCD59) Parent Studies in Participants With Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD) (clinicaltrials.gov)
    P2, N=311, Recruiting, Janssen Research & Development, LLC
    New P2 trial
    2ms
    Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy. (PubMed, N Engl J Med)
    At a median follow-up of 6 years after lentiviral gene therapy, most patients with early cerebral adrenoleukodystrophy and MRI abnormalities had no major functional disabilities. However, insertional oncogenesis is an ongoing risk associated with the integration of viral vectors. (Funded by Bluebird Bio; ALD-102 and LTF-304 ClinicalTrials.gov numbers NCT01896102 and NCT02698579, respectively.).
    Journal • Gene therapy
    |
    CD34 (CD34 molecule)
    2ms
    Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy. (PubMed, N Engl J Med)
    Hematologic cancer developed in a subgroup of patients who were treated with eli-cel; the cases are associated with clonal vector insertions within oncogenes and clonal evolution with acquisition of somatic genetic defects. (Funded by Bluebird Bio; ALD-102, ALD-104, and LTF-304 ClinicalTrials.gov numbers, NCT01896102, NCT03852498, and NCT02698579, respectively.).
    Journal • Gene therapy
    |
    KRAS (KRAS proto-oncogene GTPase) • NRAS (Neuroblastoma RAS viral oncogene homolog) • CDKN2A (Cyclin Dependent Kinase Inhibitor 2A) • RUNX1 (RUNX Family Transcription Factor 1) • CDKN2B (Cyclin Dependent Kinase Inhibitor 2B) • WT1 (WT1 Transcription Factor) • CD34 (CD34 molecule) • MECOM (MDS1 And EVI1 Complex Locus) • PRDM16 (PR/SET Domain 16)
    |
    KRAS mutation • NRAS mutation • WT1 mutation
    2ms
    An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy (clinicaltrials.gov)
    P1/2, N=6, Recruiting, Lantu Biopharma | Trial completion date: Jul 2027 --> Oct 2027 | Trial primary completion date: Jul 2027 --> Oct 2027
    Trial completion date • Trial primary completion date • Gene therapy
    3ms
    DeltaRex-G, tumor targeted retrovector encoding a CCNG1 inhibitor, for CAR-T cell therapy induced cytokine release syndrome. (PubMed, Front Mol Med)
    DeltaRex-G received United States Food and Drug Administration Emergency Use Authorization to treat Covid-19-induced acute respiratory distress syndrome, which is due to hyperactivated immune cells. A brief administration of DeltaRex-G would inhibit a certain proportion of hyperactive chimeric T cells, consequently reducing cytokine release while retaining chimeric T cell efficacy.
    Journal • CAR T-Cell Therapy
    |
    CCNG1 (Cyclin G1)
    |
    DeltaRex-G (retroviral expression vectors bearing inhibitory genes)
    3ms
    Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study) (clinicaltrials.gov)
    P1/2, N=20, Recruiting, Taysha Gene Therapies, Inc. | Phase classification: P1 --> P1/2
    Phase classification
    3ms
    Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Intravitreal (IVT) Injection of ABI-110 (AAV2.N54-VEGF Trap) in Subjects With Neovascular (Wet) Age-Related Macular Degeneration (wAMD), Including Symptomatic Macular Polypoidal Choroidal Vasculopathy (PCV) (clinicaltrials.gov)
    P1/2, N=18, Recruiting, Avirmax Biopharma Inc | Enrolling by invitation --> Recruiting
    Enrollment status