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Clinical Trials - Gene Therapy Trial Browser

The Gene Therapy Trial Browser represents a unique publicly accessible, free database for the benefit of users seeking information on gene therapy development. The information within integrates various sources, including clinicaltrials.gov, publications, sponsor press releases, meeting abstracts, and more to give a comprehensive overview of the gene therapy clinical trial landscape.

Showing 1-90 of 90 results in ClinicalTrials
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NCT04671433 Completed Phase3
View Report
X-Linked Retinitis Pigmentosa (XLRP)
Sponsor MeiraGTx Ocular UK Ltd (Industry)
Compound Botaretigene sparoparvovec
Therapeutic Modality Gene transfer
Target Gene RPGR
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2020-11-05 Last Modified Date: 1790226000000 Enrollment: 105 Belgium, Canada, Denmark + 8 more
NCT03588299 Completed Phase2, Phase1
View Report
Hemophilia A
Sponsor Bayer (Industry)
Compound DTX201
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2018-07-06 Last Modified Date: 1790139600000 Enrollment: 11 Bulgaria, France, Germany + 3 more
NCT01496040 Completed Phase2, Phase1
View Report
Leber Congenital Amaurosis
Sponsor Nantes University Hospital (Other)
Compound RAAV2/4.hRPE65
Therapeutic Modality Gene transfer
Target Gene RPE65
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2011-11-24 Last Modified Date: 1789362000000 Enrollment: 9 France
NCT05139316 Completed Phase3
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Glycogen Storage Disease Type Ia
Sponsor Ultragenyx Pharmaceutical Inc (Industry)
Compound GENGLYCOS
Therapeutic Modality Gene transfer
Target Gene G6PC
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Priority Review, Accelerated Approval, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-07-14 Last Modified Date: 1788843600000 Enrollment: 49 Brazil, Canada, Denmark + 6 more
NCT06237777 Completed Phase1
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Diabetic Macular Edema
Sponsor Wang Min (Other)
Compound SKG0106
Therapeutic Modality Gene transfer
Target Gene Anti-VEGF
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2024-01-11 Last Modified Date: 1787806800000 Enrollment: 3 China
NCT04046224 Completed Phase2, Phase1
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Fabry Disease
Sponsor Sangamo Therapeutics (Industry)
Compound ST-920
Therapeutic Modality Gene transfer
Target Gene GLA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2019-08-01 Last Modified Date: 1786683600000 Enrollment: 36 Australia, Canada, Germany + 4 more
NCT03634007 Completed Phase2, Phase1
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Alzheimer's Disease, Early Onset Alzheimer's Disease
Sponsor Lexeo Therapeutics (Industry)
Compound LX1001
Therapeutic Modality Gene transfer
Target Gene APOE2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track
First Submitted: 2018-08-03 Last Modified Date: 1786424400000 Enrollment: 15 United States
NCT05603312 Completed Phase2, Phase1
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Parkinson's Disease
Sponsor MeiraGTx, LLC (Industry)
Compound AAV-GAD
Therapeutic Modality Gene transfer
Target Gene GAD1/2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2022-10-12 Last Modified Date: 1786424400000 Enrollment: 14 United States
NCT01422772 Completed Phase2, Phase1
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Ischemic Heart Disease
Sponsor Helixmith Co., Ltd. (Industry)
Compound ENGENSIS
Therapeutic Modality Gene transfer
Target Gene HGF
Delivery System Not Applicable
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2011-08-21 Last Modified Date: 1784869200000 Enrollment: 9 South Korea
NCT05361031 Completed Phase2, Phase1
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Charcot-Marie-Tooth Disease 1A
Sponsor Helixmith Co., Ltd. (Industry)
Compound ENGENSIS
Therapeutic Modality Gene transfer
Target Gene HGF
Delivery System Not Applicable
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2022-03-31 Last Modified Date: 1784869200000 Enrollment: 12 South Korea
NCT03363165 Completed Phase2
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Peripheral Artery Disease
Sponsor Northwestern University (Other)
Compound ENGENSIS
Therapeutic Modality Gene transfer
Target Gene HGF
Delivery System Not Applicable
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2017-11-30 Last Modified Date: 1784869200000 Enrollment: 39 United States
NCT05176093 Completed Phase2
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Amyotrophic Lateral Sclerosis (ALS)
Sponsor Helixmith Co., Ltd. (Industry)
Compound ENGENSIS
Therapeutic Modality Gene transfer
Target Gene HGF
Delivery System Not Applicable
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2021-11-05 Last Modified Date: 1784869200000 Enrollment: 8 South Korea, United States
NCT01064440 Completed Phase2
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Critical Limb Ischemia
Sponsor Helixmith Co., Ltd. (Industry)
Compound ENGENSIS
Therapeutic Modality Gene transfer
Target Gene HGF
Delivery System Not Applicable
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2010-02-04 Last Modified Date: 1784869200000 Enrollment: 52 South Korea, United States
NCT04275323 Completed Phase3
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Critical Limb Ischemia
Sponsor Beijing Northland Biotech. Co., Ltd. (Industry)
Compound NL003
Therapeutic Modality Gene transfer
Target Gene HGF
Delivery System Not Applicable
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2020-01-18 Last Modified Date: 1784869200000 Enrollment: 302 China
NCT04124042 Completed Phase2
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Osteoarthritis, Knee
Sponsor Xalud Therapeutics, Inc. (Industry)
Compound XT-150
Therapeutic Modality Gene transfer
Target Gene Recombinant IL10
Delivery System Not Applicable
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track
First Submitted: 2019-10-09 Last Modified Date: 1784869200000 Enrollment: 289 Australia, United States
NCT04469270 Completed Phase3
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Diabetic Neuropathy
Sponsor Helixmith Co., Ltd. (Industry)
Compound ENGENSIS
Therapeutic Modality Gene transfer
Target Gene HGF
Delivery System Not Applicable
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2020-06-25 Last Modified Date: 1784869200000 Enrollment: 162 United States
NCT02341807 Completed Phase2, Phase1
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Choroideremia
Sponsor Spark Therapeutics, Inc. (Industry)
Compound SPK-7001
Therapeutic Modality Gene transfer
Target Gene CHM
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2015-01-12 Last Modified Date: 1784782800000 Enrollment: 15 United States
NCT05144386 Completed Phase1
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HIV-1-infection
Sponsor Excision BioTherapeutics (Industry)
Compound EBT-101
Therapeutic Modality Gene editing
Target Gene HIV genes
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Gene excision
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track
First Submitted: 2021-11-22 Last Modified Date: 1784782800000 Enrollment: 6 United States
NCT01301443 Completed Phase1
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Neovascular (Wet) Age-Related Macular Degeneration
Sponsor Oxford BioMedica (Industry)
Compound RetinoStat
Therapeutic Modality Gene transfer
Target Gene COL18A1+PLG
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2011-02-21 Last Modified Date: 1784782800000 Enrollment: 21 United States
NCT02168686 Completed Phase2, Phase1
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Alpha-1 Antitrypsin Deficiency (AATD)
Sponsor Adverum Biotechnologies, Inc. (Industry)
Compound ADVM-043
Therapeutic Modality Gene transfer
Target Gene SERPINA1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2014-06-10 Last Modified Date: 1784782800000 Enrollment: 6 United States
NCT01801709 Completed Phase2, Phase1
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Metachromatic Leukodystrophy
Sponsor Institut National de la Santé Et de la Recherche Médicale, France (Other gov)
Compound AAVrh.10-CAG/cuARSA
Therapeutic Modality Gene transfer
Target Gene ARSA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2013-01-28 Last Modified Date: 1784696400000 Enrollment: 5 France
NCT06066008 Completed Early phase1
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X-Linked Retinoschisis
Sponsor Zhongmou Therapeutics (Industry)
Compound ZM-01
Therapeutic Modality Gene transfer
Target Gene RS1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2023-09-25 Last Modified Date: 1784696400000 Enrollment: 2 China
NCT05073133 Completed Phase4
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Spinal Muscular Atrophy
Sponsor Novartis Pharmaceuticals (Industry)
Compound ZOLGENSMA
Therapeutic Modality Gene transfer
Target Gene SMN1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-09-20 Last Modified Date: 1784696400000 Enrollment: 16 Argentina, Brazil
NCT03116113 Completed Phase2, Phase1
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X-Linked Retinitis Pigmentosa (XLRP)
Sponsor Biogen (Industry)
Compound BIIB112
Therapeutic Modality Gene transfer
Target Gene RPGR
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2017-03-29 Last Modified Date: 1784696400000 Enrollment: 50 United Kingdom, United States
NCT02240407 Completed Phase1
View Report
Glycogen Storage Disease Type 2 (Pompe Disease)
Sponsor University of Florida (Other)
Compound RAAV9-DES-hGAA
Therapeutic Modality Gene transfer
Target Gene GAA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2014-09-11 Last Modified Date: 1784696400000 Enrollment: 2 United States
NCT04418427 Completed Phase2
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Diabetic Macular Edema, Diabetic Retinopathy
Sponsor Adverum Biotechnologies, Inc. (Industry)
Compound Ixoberogene soroparvovec
Therapeutic Modality Gene transfer
Target Gene Codon optimized aflibercept
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2020-05-28 Last Modified Date: 1784696400000 Enrollment: 36 Puerto Rico, United States
NCT04323098 Completed Phase3
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Hemophilia A
Sponsor BioMarin Pharmaceutical (Industry)
Compound ROCTAVIAN
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2020-03-24 Last Modified Date: 1784696400000 Enrollment: 22 Australia, Brazil, Taiwan + 1 more
NCT02082860 Completed Phase1
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Acute Intermittent Porphyria
Sponsor Digna Biotech S.L. (Industry)
Compound RAAV2/5-PBGD
Therapeutic Modality Gene transfer
Target Gene PBGD
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2014-02-28 Last Modified Date: 1784696400000 Enrollment: 8 Spain
NCT04945772 Completed Phase2
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Retinitis Pigmentosa, Retinal Dystrophies, Retinal Degeneration
Sponsor Nanoscope Therapeutics Inc. (Industry)
Compound MCO-010
Therapeutic Modality Gene transfer
Target Gene MCO
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2021-06-15 Last Modified Date: 1784696400000 Enrollment: 27 Puerto Rico, United States
NCT03812263 Completed Phase2, Phase1
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Leukocyte Adhesion Defect - Type I
Sponsor Rocket Pharmaceuticals Inc. (Industry)
Compound KRESLADI
Therapeutic Modality Gene transfer
Target Gene ITGB2
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Accelerated Approval, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2019-01-18 Last Modified Date: 1784696400000 Enrollment: 9 Spain, United Kingdom, United States
NCT03569891 Completed Phase3
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Hemophilia B
Sponsor CSL Behring (Industry)
Compound HEMGENIX
Therapeutic Modality Gene transfer
Target Gene F9-R338L
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Priority Review, Orphan Drug Designation
First Submitted: 2018-06-14 Last Modified Date: 1784696400000 Enrollment: 67 Belgium, Denmark, Germany + 5 more
NCT02416622 Completed Phase2, Phase1
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X-Linked Retinoschisis
Sponsor Beacon Therapeutics (Industry)
Compound RAAV2tYF-CB-hRS1
Therapeutic Modality Gene transfer
Target Gene RS1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2015-04-02 Last Modified Date: 1784696400000 Enrollment: 27 United States
NCT01482195 Completed Phase1
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Retinal Disease, Retinitis Pigmentosa
Sponsor King Khaled Eye Specialist Hospital (Other gov)
Compound AAV2-VMD2-hMERTK
Therapeutic Modality Gene transfer
Target Gene MERTK
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2011-09-28 Last Modified Date: 1784696400000 Enrollment: 6 Saudi Arabia
NCT02781480 Completed Phase2, Phase1
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Leber Congenital Amaurosis
Sponsor MeiraGTx UK II Ltd (Industry)
Compound Cevaretigene ritoparvovec
Therapeutic Modality Gene transfer
Target Gene RPE65
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2016-04-28 Last Modified Date: 1784696400000 Enrollment: 15 United Kingdom, United States
NCT00643747 Completed Phase2, Phase1
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Leber Congenital Amaurosis-Type 2
Sponsor University College, London (Other)
Compound TgAAG76
Therapeutic Modality Gene transfer
Target Gene RPE65
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2008-03-20 Last Modified Date: 1784696400000 Enrollment: 12 United Kingdom
NCT01024998 Completed Phase1
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Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Genzyme, a Sanofi Company (Industry)
Compound RAAV2-sFLT01
Therapeutic Modality Gene transfer
Target Gene SFLT01
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2009-12-02 Last Modified Date: 1784696400000 Enrollment: 19 United States
NCT03562494 Completed Phase1
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Parkinson's Disease
Sponsor Neurocrine Biosciences (Industry)
Compound VY-AADC01
Therapeutic Modality Gene transfer
Target Gene DDC
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2018-05-16 Last Modified Date: 1784696400000 Enrollment: 14 United States
NCT05417126 Completed Phase2
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Stargardt Disease
Sponsor Nanoscope Therapeutics Inc. (Industry)
Compound MCO-010
Therapeutic Modality Gene transfer
Target Gene MCO
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2022-06-09 Last Modified Date: 1784696400000 Enrollment: 6 United States
NCT03374657 Completed Phase2, Phase1
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Retinitis Pigmentosa
Sponsor Novartis Pharmaceuticals (Industry)
Compound CPK850
Therapeutic Modality Gene transfer
Target Gene RLBP1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2017-12-11 Last Modified Date: 1784696400000 Enrollment: 12 Sweden
NCT00985517 Completed Phase2, Phase1
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Idiopathic Parkinson's Disease
Sponsor Sangamo Therapeutics (Industry)
Compound CERE-120
Therapeutic Modality Gene transfer
Target Gene NRTN
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2009-09-25 Last Modified Date: 1784696400000 Enrollment: 57 United States
NCT01161576 Completed Phase1
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CLN2 Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis
Sponsor Weill Medical College of Cornell University (Other)
Compound LX1004
Therapeutic Modality Gene transfer
Target Gene TPP1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2010-03-22 Last Modified Date: 1784696400000 Enrollment: 12 United States
NCT03758404 Completed Phase2, Phase1
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Achromatopsia
Sponsor MeiraGTx UK II Ltd (Industry)
Compound AAV-CNGA3
Therapeutic Modality Gene transfer
Target Gene CNGA3
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2018-11-27 Last Modified Date: 1784696400000 Enrollment: 11 United Kingdom, United States
NCT00876863 Completed Phase2
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Alzheimer's Disease
Sponsor Sangamo Therapeutics (Industry)
Compound CERE-110
Therapeutic Modality Gene transfer
Target Gene NGF
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2009-04-03 Last Modified Date: 1784696400000 Enrollment: 49 United States
NCT00749957 Completed Phase2, Phase1
View Report
Leber Congenital Amaurosis-Type 2
Sponsor Beacon Therapeutics (Industry)
Compound RAAV2-CB-hRPE65
Therapeutic Modality Gene transfer
Target Gene RPE65
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2008-09-08 Last Modified Date: 1784696400000 Enrollment: 12 United States
NCT02132130 Completed Phase2, Phase1
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Unilateral OR Bilateral Severe to Profound Hearing Loss
Sponsor Novartis Pharmaceuticals (Industry)
Compound CGF166
Therapeutic Modality Gene transfer
Target Gene ATOH1
Vector Type Adenovirus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2014-05-05 Last Modified Date: 1784696400000 Enrollment: 22 United States
NCT02247843 Completed Phase2, Phase1
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Sickle Cell Disease
Sponsor Donald B. Kohn, M.D. (Other)
Compound LentiG-βAS3-FB (contains anti-sickling mutations: T87Q, G16D, E22A)
Therapeutic Modality Gene transfer
Target Gene HBB
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2014-09-20 Last Modified Date: 1784696400000 Enrollment: 4 United States
NCT02556736 Completed Phase2, Phase1
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Advanced Retinitis Pigmentosa
Sponsor AbbVie (Industry)
Compound RST-001
Therapeutic Modality Gene transfer
Target Gene ChR2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Orphan Drug Designation
First Submitted: 2015-09-21 Last Modified Date: 1784696400000 Enrollment: 14 United States
NCT05811351 Completed Phase2
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Geographic Atrophy
Sponsor Janssen Research & Development, LLC (Industry)
Compound JNJ-1887
Therapeutic Modality Gene transfer
Target Gene CD59
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2023-03-07 Last Modified Date: 1784696400000 Enrollment: 305 Australia, Belgium, Canada + 14 more
NCT00821340 Completed Phase1
View Report
Leber Congenital Amaurosis-Type 2
Sponsor Hadassah Medical Organization (Other)
Compound RAAV2-hRPE65
Therapeutic Modality Gene transfer
Target Gene RPE65
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2009-01-12 Last Modified Date: 1784696400000 Enrollment: 3 Israel
NCT03964792 Completed Phase2, Phase1
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Sickle Cell Disease
Sponsor Assistance Publique - Hôpitaux de Paris (Other)
Compound DREPAGLOBE
Therapeutic Modality Gene transfer
Target Gene HBB
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2019-05-14 Last Modified Date: 1784696400000 Enrollment: 6 France
NCT05903794 Completed Phase1
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Exegenesis Bio (Industry)
Compound EXG102-031
Therapeutic Modality Gene transfer
Target Gene ABD-VEGFR
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2023-06-06 Last Modified Date: 1784696400000 Enrollment: 12 United States
NCT02317887 Completed Phase2, Phase1
View Report
X-Linked Retinoschisis
Sponsor VegaVect, Inc. (Industry)
Compound RS1 AAV Vector
Therapeutic Modality Gene transfer
Target Gene RS1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2014-12-16 Last Modified Date: 1784696400000 Enrollment: 12 United States
NCT03207009 Completed Phase3
View Report
Beta-Thalassemia Major
Sponsor Genetix Biotherapeutics Inc. (Industry)
Compound ZYNTEGLO
Therapeutic Modality Gene transfer
Target Gene HBB
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2017-06-29 Last Modified Date: 1784696400000 Enrollment: 19 France, Germany, Greece + 3 more
NCT00976352 Completed Phase2, Phase1
View Report
Glycogen Storage Disease Type 2 (Pompe Disease)
Sponsor University of Florida (Other)
Compound AAV1-CMV-hGAA
Therapeutic Modality Gene transfer
Target Gene GAA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2009-07-13 Last Modified Date: 1784696400000 Enrollment: 9 United States
NCT05386680 Completed Phase3
View Report
Spinal Muscular Atrophy
Sponsor Novartis Pharmaceuticals (Industry)
Compound ITVISMA
Therapeutic Modality Gene transfer
Target Gene SMN1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Orphan Drug Designation
First Submitted: 2022-05-18 Last Modified Date: 1784696400000 Enrollment: 27 Australia, Belgium, Canada + 6 more
NCT03001310 Completed Phase2, Phase1
View Report
Achromatopsia
Sponsor MeiraGTx UK II Ltd (Industry)
Compound AAV-CNGB3
Therapeutic Modality Gene transfer
Target Gene CNGB3
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2016-11-22 Last Modified Date: 1784696400000 Enrollment: 23 United Kingdom, United States
NCT00428935 Completed Phase1
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor Nationwide Children's Hospital (Other)
Compound RAAV2.5-CMV-mini-Dystrophin
Therapeutic Modality Gene transfer
Target Gene Micro-dystrophin
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2007-01-26 Last Modified Date: 1784696400000 Enrollment: 6 United States
NCT03300453 Completed Phase2, Phase1
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Sanfilippo Syndrome B
Sponsor UniQure Biopharma B.V. (Industry)
Compound AMT-110
Therapeutic Modality Gene transfer
Target Gene NAGLU
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2016-06-15 Last Modified Date: 1784696400000 Enrollment: 4 France
NCT03533673 Completed Phase1
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Pompe Disease
Sponsor AskBio Inc (Industry)
Compound ACTUS-101
Therapeutic Modality Gene transfer
Target Gene GAA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track
First Submitted: 2018-04-13 Last Modified Date: 1784696400000 Enrollment: 7 United States
NCT00979238 Completed Phase1
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Hemophilia B
Sponsor St. Jude Children's Research Hospital (Other)
Compound ScAAV2/8-LP1-hFIXco
Therapeutic Modality Gene transfer
Target Gene F9
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2009-09-16 Last Modified Date: 1784696400000 Enrollment: 14 United Kingdom, United States
NCT03173521 Completed Phase2, Phase1
View Report
Mucopolysaccharidosis Type VI
Sponsor Fondazione Telethon (Other)
Compound AAV2/8.TBG.hARSB
Therapeutic Modality Gene transfer
Target Gene ARSB
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2017-04-12 Last Modified Date: 1784696400000 Enrollment: 9 Italy, Turkey (Türkiye)
NCT04240314 Completed Phase2, Phase1
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor Megan Waldrop (Other)
Compound AT702
Therapeutic Modality Gene transfer
Target Gene DMD (exon 2)
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Exon skipping/splice editor
First Submitted: 2020-01-22 Last Modified Date: 1784696400000 Enrollment: 3 United States
NCT02652767 Completed Phase3
View Report
Leber Hereditary Optic Neuropathy (LHON)
Sponsor GenSight Biologics (Industry)
Compound LUMEVOQ
Therapeutic Modality Gene transfer
Target Gene ND4 m.11778G>A
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2016-01-07 Last Modified Date: 1784696400000 Enrollment: 39 France, Germany, Italy + 2 more
NCT03333590 Completed Phase2, Phase1
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor Kevin Flanigan (Other)
Compound RAAVrh74.MCK.GALGT2
Therapeutic Modality Gene transfer
Target Gene GALGT2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2017-11-01 Last Modified Date: 1784696400000 Enrollment: 2 United States
NCT02161380 Completed Phase1
View Report
Leber Hereditary Optic Neuropathy (LHON)
Sponsor Byron Lam (Other)
Compound ScAAV2-P1ND4v2
Therapeutic Modality Gene transfer
Target Gene ND4
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2014-06-06 Last Modified Date: 1784696400000 Enrollment: 28 United States
NCT02725580 Completed Phase2, Phase1
View Report
Variant Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN6 Batten Disease)
Sponsor Emily de los Reyes (Other)
Compound AT-GTX-501
Therapeutic Modality Gene transfer
Target Gene CLN6
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2016-03-16 Last Modified Date: 1784696400000 Enrollment: 13 United States
NCT03852498 Completed Phase3
View Report
Cerebral Adrenoleukodystrophy (CALD)
Sponsor Genetix Biotherapeutics Inc. (Industry)
Compound SKYSONA
Therapeutic Modality Gene transfer
Target Gene ABCD1
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2019-02-13 Last Modified Date: 1784696400000 Enrollment: 35 France, Germany, Italy + 3 more
NCT03496012 Completed Phase3
View Report
Choroideremia
Sponsor Biogen (Industry)
Compound BIIB111
Therapeutic Modality Gene transfer
Target Gene CHM
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2018-03-07 Last Modified Date: 1784696400000 Enrollment: 169 Canada, Denmark, Finland + 5 more
NCT05265767 Completed Phase1
View Report
Hemophilia A
Sponsor Christian Medical College, Vellore, India (Other)
Compound CD68-ET3-LV
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2022-02-16 Last Modified Date: 1784696400000 Enrollment: 6 India
NCT01621867 Completed Phase2
View Report
Cystic Fibrosis
Sponsor Imperial College London (Other)
Compound PGM169/GL67A
Therapeutic Modality Gene transfer
Target Gene CFTR
Delivery System Endocytosis
Mechanism of Action Functional gene replacement
First Submitted: 2012-06-14 Last Modified Date: 1784610000000 Enrollment: 130 United Kingdom
NCT00598481 Completed Phase2
View Report
Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Sponsor Fondazione Telethon (Other)
Compound STRIMVELIS
Therapeutic Modality Gene transfer
Target Gene ADA
Vector Type Retrovirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2008-01-10 Last Modified Date: 1784610000000 Enrollment: 12 Israel, Italy
NCT00494195 Completed Phase1
View Report
Limb-Girdle Muscular Dystrophy, Type 2D/R3
Sponsor Nationwide Children's Hospital (Other)
Compound RAAV1.tMCK.human-alpha-sarcoglycan
Therapeutic Modality Gene transfer
Target Gene SGCA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2007-06-27 Last Modified Date: 1784610000000 Enrollment: 6 United States
NCT01344798 Completed Phase1
View Report
Limb-Girdle Muscular Dystrophy, Type 2C/R5
Sponsor Genethon (Other)
Compound AAV1.DES.hSGCG
Therapeutic Modality Gene transfer
Target Gene SGCG
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2011-03-17 Last Modified Date: 1784610000000 Enrollment: 9 France
NCT04917874 Completed Phase3
View Report
Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Sponsor Krystal Biotech, Inc. (Industry)
Compound VYJUVEK
Therapeutic Modality Gene transfer
Target Gene COL7A1
Vector Type Herpesvirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Priority Review, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-06-02 Last Modified Date: 1784610000000 Enrollment: 47 United States
NCT00272857 Completed Phase1
View Report
Fanconi Anemia Complementation Group A
Sponsor Boston Children's Hospital (Other)
Compound MSCV-FANCA
Therapeutic Modality Gene transfer
Target Gene FANCA
Vector Type Retrovirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2006-01-04 Last Modified Date: 1784610000000 Enrollment: 3 United States
NCT00028236 Completed Phase1
View Report
X-linked Severe Combined Immunodeficiency (XSCID)
Sponsor National Institute of Allergy and Infectious Diseases (NIAID) (NIH)
Compound GALV MFGS-gc retrovirus
Therapeutic Modality Gene transfer
Target Gene IL2RG
Vector Type Retrovirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2001-12-17 Last Modified Date: 1784610000000 Enrollment: 3 United States
NCT03734588 Completed Phase2, Phase1
View Report
Hemophilia A
Sponsor Spark Therapeutics, Inc. (Industry)
Compound SPK-8016
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2018-11-06 Last Modified Date: 1784610000000 Enrollment: 4 United States
NCT01494805 Completed Phase2, Phase1
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Lions Eye Institute, Perth, Western Australia (Other)
Compound RAAV.sFlt-1
Therapeutic Modality Gene transfer
Target Gene Soluble VEGFR1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2011-12-14 Last Modified Date: 1784610000000 Enrollment: 40 Australia
NCT01410019 Completed Phase2, Phase1
View Report
X-linked Severe Combined Immunodeficiency (XSCID)
Sponsor Assistance Publique - Hôpitaux de Paris (Other)
Compound Autologous CD34+ cells transduced with the self-inactivating (SIN) GAMMARETROVIRAL vector pSRS11.EFS.IL2RG.pre
Therapeutic Modality Gene transfer
Target Gene IL2RG
Vector Type Retrovirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2011-06-21 Last Modified Date: 1784610000000 Enrollment: 5 France
NCT01643330 Completed Phase2
View Report
Ischemic Cardiomyopathy, Non-ischemic Cardiomyopathy, Heart Failure, Cardiomyopathies
Sponsor Celladon Corporation (Industry)
Compound AAV1-CMV-Serca2a
Therapeutic Modality Gene transfer
Target Gene SERCA2A
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2012-07-16 Last Modified Date: 1784610000000 Enrollment: 250 Belgium, Denmark, Germany + 7 more
NCT01519349 Completed Phase1
View Report
Becker Muscular Dystrophy, Sporadic Inclusion Body Myositis
Sponsor Nationwide Children's Hospital (Other)
Compound RAAV1.CMV.huFollistatin344
Therapeutic Modality Gene transfer
Target Gene FST (FS344)
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2012-01-23 Last Modified Date: 1784610000000 Enrollment: 15 United States
NCT01054339 Completed Phase2
View Report
Alpha-1 Antitrypsin Deficiency (AATD)
Sponsor Beacon Therapeutics (Industry)
Compound AGTC-0106
Therapeutic Modality Gene transfer
Target Gene SERPINA1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2010-01-21 Last Modified Date: 1784610000000 Enrollment: 9 Ireland, United States
NCT02354781 Completed Phase2, Phase1
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor Jerry R. Mendell (Other)
Compound RAAV1.CMV.huFollistatin344
Therapeutic Modality Gene transfer
Target Gene FST (FS344)
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2015-01-26 Last Modified Date: 1784610000000 Enrollment: 3 United States
NCT00891306 Completed Phase3, Phase2
View Report
Familial Lipoprotein Lipase Deficiency
Sponsor Amsterdam Molecular Therapeutics (Industry)
Compound GLYBERA
Therapeutic Modality Gene transfer
Target Gene LPL
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2009-04-30 Last Modified Date: 1784610000000 Enrollment: 5 Canada
NCT05398029 Completed Phase1
View Report
Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease
Sponsor Verve Therapeutics, Inc. (Industry)
Compound VERVE-101
Therapeutic Modality Gene editing
Target Gene PCSK9
Delivery System Endocytosis
Mechanism of Action Gene inactivation
First Submitted: 2022-05-19 Last Modified Date: 1784610000000 Enrollment: 13 New Zealand, United Kingdom
NCT00787059 Completed Phase2, Phase1
View Report
Congestive Heart Failure
Sponsor Hammond, H. Kirk, M.D. (Indiv)
Compound RT-100
Therapeutic Modality Gene transfer
Target Gene ADCY6
Vector Type Adenovirus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track
First Submitted: 2008-11-06 Last Modified Date: 1784610000000 Enrollment: 56 United States
NCT00372320 Completed Phase1
View Report
Parotid Salivary Dysfunction
Sponsor National Institute of Dental and Craniofacial Research (NIDCR) (NIH)
Compound AdhAQP1
Therapeutic Modality Gene transfer
Target Gene AQP1
Vector Type Adenovirus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2006-09-02 Last Modified Date: 1784610000000 Enrollment: 17 United States
NCT00927134 Completed Phase2, Phase1
View Report
X-Linked Chronic Granulomatous Disease
Sponsor University of Zurich (Other)
Compound Retroviral SF71-gp91phox transduced CD34+ cells
Therapeutic Modality Gene transfer
Target Gene CYBB
Vector Type Retrovirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2009-06-22 Last Modified Date: 1784610000000 Enrollment: 2 Switzerland
NCT06291961 Completed Phase1
View Report
Beta-Thalassemia Major
Sponsor CorrectSequence Therapeutics Co., Ltd (Industry)
Compound CS-101
Therapeutic Modality Gene editing
Target Gene BCL11A
Delivery System Electroporation
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2024-02-23 Last Modified Date: 1781240400000 Enrollment: 9 China
NCT03432364 Completed Phase2, Phase1
View Report
Beta-Thalassemia Major
Sponsor Sangamo Therapeutics (Industry)
Compound ST-400
Therapeutic Modality Gene editing
Target Gene BCL11A
Delivery System Electroporation
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Orphan Drug Designation
First Submitted: 2018-02-01 Last Modified Date: 1773723600000 Enrollment: 5 United States
Visual summary of all 90 matching trials (across every page). Click a slice or bar to filter the results by it.
Therapeutic Modality
Current Phase
Recruitment Status
Funder Type
Vector Type
Conditions Studied
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Top Locations

Clinical Trials Daily Digest

4 Updates

Updates in the last 14 days

NCT06467344
Last update post date Previous: 2025-12-02 Current: 2026-10-07 api
Number of locations Previous: 10 Current: 11 api
Vector type Previous: AAV Current: Adeno-associated virus curator
Dose 3 Previous: Undisclosed high dose Current: curator
Dose 2 Previous: Undisclosed medium dose Current: curator
Dose 1 Previous: Undisclosed low dose Current: Undisclosed dose escalation, 3 levels curator
Editor type Previous: none Current: curator
Eligibility criteria Previous: null Current: Key inclusion criteria: * presence of mutations i... api
Indication doid Previous: DOID:0050817 Current: DOID:0050817,DOID:0061241 curator
1791349200000
Stargardt Disease, Cone Rod Dystrophy, Juvenile Macular Degeneration
Ascidian Therapeutics, Inc Recruiting
Rare Pediatric Disease designation granted 4/25/24
NCT01306019
Last update post date Previous: 2026-10-06 Current: 2026-10-13 api
Description Previous: = 2 years of age who have clinically significant defects of immunity despite prior haploidentical hematopoietic stem cell transplant, And who lack an hla-matched sibling donor. our current gene transfer treatment protocol can be regarded as a salvage/rescue protocol. prior successful retroviral gene transfer treatment instead of bone marrow transplant (bmt) in paris and london for 20 infants with xscid has provided proof of principle for efficacy. however, A major safety concern is the occurrence of 5 cases of leukemia at 3-5 years after treatment triggered in part by vector insertional mutagenesis activation of lmo2 and other dna regulatory genes by the strong enhancer present in the long-terminal repeat (ltr) of the moloney leukemia virus (mlv)- based vector. furthermore, Previous studies of gene transfer treatment of older xscid patients with mlv- based vectors demonstrated the additional problem of failure of adequate expansion of gene corrected t- lymphocytes to the very high levels seen in infants. to reduce or eliminate this leukemia risk, And possibly enhance performance sufficiently to achieve benefit in older xscid patients, We have generated a lentivector with improved safety and performance features. we have generated a self-inactivating (sin) lentiviral vector that is devoid of all viral transcription elements; that contains a short form of the human elongation factor 1a (ef1a) internal promoter to expres......">This is a phase i/ii non-randomized clinical trial... Current: = 2 years of age who have clinically significant defects of immunity despite prior haploidentical hematopoietic stem cell transplant, And who lack an hla-matched sibling donor. our current gene transfer treatment protocol can be regarded as a salvage/rescue protocol. prior successful retroviral gene transfer treatment instead of bone marrow transplant (bmt) in paris and london for 20 infants with xscid has provided proof of principle for efficacy. however, A major safety concern is the occurrence of 5 cases of leukemia at 3-5 years after treatment triggered in part by vector insertional mutagenesis activation of lmo2 and other dna regulatory genes by the strong enhancer present in the long-terminal repeat (ltr) of the moloney leukemia virus (mlv)- based vector. furthermore, Previous studies of gene transfer treatment of older xscid patients with mlv- based vectors demonstrated the additional problem of failure of adequate expansion of gene corrected t- lymphocytes to the very high levels seen in infants. to reduce or eliminate this leukemia risk, And possibly enhance performance sufficiently to achieve benefit in older xscid patients, We have generated a lentivector with improved safety and performance features. we have generated a self-inactivating (sin) lentiviral vector that is devoid of all viral transcription elements; that contains a short form of the human elongation factor 1a (ef1a) internal promoter to expres...">This is a phase i/ii non-randomized clinical trial... api
Vector type Previous: VSV-G Current: Lentivirus curator
Editor type Previous: none Current: curator
Eligibility criteria Previous: null Current: * inclusion criteria: * a proven mutation in the c... api
1791262800000
X-linked Severe Combined Immunodeficiency (XSCID)
National Institute of Allergy and Infectious Diseases (NIAID) Recruiting
Developed in partnership with St. Jude, and licensed to Mustang Bio, Mustang Bio discontinued this program
NCT06092034
Recent updates Previous: Clinical hold lifted August 2025 Current: Clinical hold lifted August 2025. Sponsor expects ... curator
News and press releases Previous: null Current: LinkName:Rocket Pharmaceuticals Announces FDA Alig... curator
Last update post date Previous: 2026-05-04 Current: 2026-10-06 api
Estimated completion date Previous: 2032-04 Current: 2032-06 api
Eligibility criteria Previous: Key inclusion criteria: 1. documentation of a pat... Current: Key inclusion criteria: 1. documentation of a pat... api
Enrollment count Previous: 14 Current: 18 api
Intervention description Previous: Rp-a501 is a gene therapy product consisting of a ... Current: Rp-a501 is a gene therapy product consisting of an... api
Description Previous: This is a single arm phase 2 trial to evaluate the... Current: This is a single arm phase 2 trial to evaluate the... api
Vector type Previous: AAV9 Current: Adeno-associated virus curator
Editor type Previous: none Current: curator
1791262800000
Danon Disease
Rocket Pharmaceuticals Inc. Recruiting
Clinical hold lifted August 2025
NCT06389877
Dose 2 Previous: Part B: 30mg (n=3); 60mg (n=2) Current: Part B: 30mg (n=3); 60mg (n=5) curator
Clinical publications Previous: null Current: LinkName:(Presentation) First-in-human in vivo gen... curator
Last update post date Previous: 2026-03-20 Current: 2026-10-05 api
Eligibility max age Previous: 70 years Current: 75 years api
Eligibility criteria Previous: Part a: inclusion criteria: * males or females 1... Current: Part a: key inclusion criteria: * age 18-70 year... api
Number of locations Previous: 11 Current: 12 api
Enrollment count Previous: 106 Current: 192 api
Description Previous: This is a phase 1/2, Multicenter, Open-label, Dose... Current: This is a phase 1/2, Multicenter, Open-label study... api
Preclinical publications Previous: LinkName:(Presentation) BEAM-302 Base editing as a... Current: LinkName:(Presentation) BEAM-302 Base editing as a... curator
Preclinical publications Previous: LinkName:(Corporate Presentation) BEAM-302: Target... Current: null curator
Preclinical publications Previous: LinkName:(Poster) BEAM-302 decreases hepatic aggre... Current: LinkName:(Poster) BEAM-302 decreases hepatic aggre... curator
Vector type Previous: LNP Current: curator
Editor type Previous: ABE Current: ABE mRNA curator
Clinical publications Previous: null Current: LinkName:(Presentation) The potential for base edi... curator
Drug product type Previous: Encapsulated RNP Current: Encapsulated mRNA curator
Delivery system Previous: Lipid encapsulation Current: Endocytosis curator
Fda designation Previous: Orphan Drug Designation, Regenerative Medicine Adv... Current: Orphan Drug Designation, Regenerative Medicine Adv... curator
Recent updates Previous: Data from Parts A and B of Phase 1/2 trial expecte... Current: Data from Parts A and B of Phase 1/2 trial release... curator
Dose 4 Previous: 75mg Current: curator
Dose 3 Previous: 60mg Current: Pivotal dose selected: 60mg curator
Dose 1 Previous: 15mg Current: Part A: 15mg (n=3); 30mg (n=3); 60mg (n=6); 75mg (... curator
News and press releases Previous: null Current: LinkName:Corporate Presentation - January 2026;Lin... curator
News and press releases Previous: null Current: LinkName:Beam Therapeutics Announces Compelling Up... curator
Estimated completion date Previous: 2027-08 Current: 2030-05 curator
Locations Previous: New zealand, Netherlands, United kingdom, Australi... Current: New zealand, Netherlands, United states, Ireland, ... curator
News and press releases Previous: null Current: LinkName:Beam Therapeutics Reports Fourth Quarter ... curator
1791176400000
Alpha-1 Antitrypsin Deficiency (AATD)
Beam Therapeutics Inc. Recruiting
Data from Parts A and B of Phase 1/2 trial released in March 2026, Beam intends to pursue an accelerated approval for BEAM-302
Disclaimer: The information on this dashboard has been collected for the convenience of patients and researchers. The SCGE team are not medical doctors and cannot provide medical advice. Please discuss with your provider the risks/benefits of participating in a clinical trial, and do not send us your personal medical information. The information contained within this table does not make use of any confidential or privileged information-all data is collected from publicly available sources. The SCGE makes no comment as to the efficacy and safety of the items listed, as these are not known at the time of publication. For the most up to date information, or to inquire about enrollment, please refer to clinicaltrials.gov or the Sponsor's website for contact information.