^
    20h
    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
    3d
    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
    3d
    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
    5d
    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
    7d
    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
    16d
    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
    18d
    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
    18d
    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
    18d
    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
    22d
    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
    22d
    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
    22d
    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
    23d
    Evaluating Safety and Efficacy of Lentiviral-transduced CD34+ HSCs in Β-thalassaemia Patients. (clinicaltrials.gov)
    P1, N=8, Recruiting, Shenzhen Hemogen
    New P1 trial
    29d
    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)
    29d
    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
    29d
    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
    30d
    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)
    1m
    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
    1m
    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)
    1m
    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
    1m
    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
    1m
    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)
    1m
    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
    1m
    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
    1m
    Safety and Proof-of-Concept (POC) Study with AMT-130 in Adults with Early Manifest Huntington's Disease (clinicaltrials.gov)
    P1/2, N=36, Active, not recruiting, UniQure Biopharma B.V. | Recruiting --> Active, not recruiting
    Enrollment closed
    |
    NEFL (Neurofilament Light Chain)
    1m
    AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom) (clinicaltrials.gov)
    P1/2, N=12, Recruiting, NGGT INC. | Not yet recruiting --> Recruiting
    Enrollment open
    1m
    AATD: A Phase 1/2 Study of NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease (clinicaltrials.gov)
    P1/2, N=30, Not yet recruiting, Intellia Therapeutics
    New P1/2 trial
    1m
    Acclaim-2: Quaratusugene Ozeplasmid (Reqorsa) in Combination with Pembrolizumab in Previously Treated Non-Small Lung Cancer (clinicaltrials.gov)
    P1/2, N=180, Active, not recruiting, Genprex, Inc. | Trial completion date: Nov 2028 --> Dec 2025 | Trial primary completion date: Nov 2027 --> Dec 2024
    Trial completion date • Trial primary completion date • Combination therapy
    |
    EGFR (Epidermal growth factor receptor) • PD-L1 (Programmed death ligand 1) • ALK (Anaplastic lymphoma kinase)
    |
    EGFR mutation • ALK mutation
    |
    Keytruda (pembrolizumab) • docetaxel • Cyramza (ramucirumab) • Reqorsa (quaratusugene ozeplasmid)
    2ms
    AUDIOGENE: A Phase I/II Clinical Trial with SENS-501 in Children Suffering from Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations (clinicaltrials.gov)
    P1/2, N=12, Recruiting, Sensorion | Not yet recruiting --> Recruiting | Trial completion date: Apr 2031 --> Jul 2031 | Trial primary completion date: Apr 2027 --> Jul 2027
    Enrollment open • Trial completion date • Trial primary completion date
    2ms
    Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis (clinicaltrials.gov)
    P1/2, N=22, Active, not recruiting, Ocugen | Recruiting --> Active, not recruiting | N=124 --> 22
    Enrollment closed • Enrollment change
    |
    CEP290 (Centrosomal Protein 290)
    |
    CEP290 mutation
    2ms
    A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) (clinicaltrials.gov)
    P1/2, N=8, Active, not recruiting, Adrenas Therapeutics Inc | Recruiting --> Active, not recruiting | N=25 --> 8 | Trial completion date: Nov 2028 --> Feb 2029 | Trial primary completion date: Dec 2024 --> Feb 2029
    Enrollment closed • Enrollment change • Trial completion date • Trial primary completion date • Gene therapy
    2ms
    A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD ) (clinicaltrials.gov)
    P1/2, N=12, Recruiting, Prime Medicine, Inc. | Not yet recruiting --> Recruiting
    Enrollment open • Gene therapy
    2ms
    A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease (clinicaltrials.gov)
    P2, N=12, Active, not recruiting, Rocket Pharmaceuticals Inc. | Recruiting --> Active, not recruiting
    Enrollment closed • Gene therapy
    |
    LAMP2 (Lysosomal Associated Membrane Protein 2)
    2ms
    reiMMAgine: A Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of SEL-302 in Pediatric Subjects With MMA (clinicaltrials.gov)
    P1/2, N=0, Withdrawn, Selecta Biosciences, Inc. | Suspended --> Withdrawn
    Trial withdrawal
    2ms
    Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD) (clinicaltrials.gov)
    P1, N=4, Active, not recruiting, Novartis Pharmaceuticals | Trial completion date: Sep 2026 --> Oct 2024 | Trial primary completion date: Aug 2025 --> Oct 2024
    Trial completion date • Trial primary completion date
    2ms
    Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations (clinicaltrials.gov)
    P1/2, N=11, Terminated, Neurophth Therapeutics Inc | N=18 --> 11 | Trial completion date: Dec 2029 --> Jun 2024 | Recruiting --> Terminated | Trial primary completion date: Dec 2024 --> Jun 2024; Due to the sponsor circumstances and external reasons
    Enrollment change • Trial completion date • Trial termination • Trial primary completion date • Gene therapy
    2ms
    A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMA (clinicaltrials.gov)
    P=N/A, N=18, Not yet recruiting, Hangzhou Jiayin Biotech Ltd
    New trial
    2ms
    Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients (clinicaltrials.gov)
    P=N/A, N=25, Enrolling by invitation, Forge Biologics, Inc | Not yet recruiting --> Enrolling by invitation
    Enrollment open • Gene therapy
    2ms
    BEYOND-9: A Trial to Learn if REGV131-LNP1265 is Safe and Works to Help the Body Make Clotting Factor in Pediatric, Adolescent and Adult Patients With Hemophilia B (clinicaltrials.gov)
    P1/2, N=130, Recruiting, Regeneron Pharmaceuticals | Not yet recruiting --> Recruiting
    Enrollment open
    2ms
    Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT) (clinicaltrials.gov)
    P1, N=9, Active, not recruiting, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine | Recruiting --> Active, not recruiting | Trial completion date: Dec 2024 --> Oct 2028 | Trial primary completion date: Jan 2024 --> Oct 2024
    Enrollment closed • Trial completion date • Trial primary completion date • Gene therapy
    2ms
    A Study to Determine Eligibility for CAH-301 (A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [NCT04783181]) (clinicaltrials.gov)
    P=N/A, N=65, Completed, Adrenas Therapeutics Inc | Recruiting --> Completed | N=300 --> 65 | Trial completion date: Dec 2024 --> Aug 2024 | Trial primary completion date: Dec 2024 --> Aug 2024
    Trial completion • Enrollment change • Trial completion date • Trial primary completion date • Gene therapy
    2ms
    A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) (clinicaltrials.gov)
    P1/2, N=43, Recruiting, Solid Biosciences Inc. | Trial completion date: May 2030 --> May 2031 | Trial primary completion date: May 2026 --> May 2027
    Trial completion date • Trial primary completion date • Gene therapy