Gene Therapy
This page covers all Gene Therapy drugs tracked by Drug Landscape: marketed products and active clinical-stage compounds, targeting SMN1 gene, RPE65 gene, p53 (tumor suppressor protein).
Targets
SMN1 gene · RPE65 gene · p53 (tumor suppressor protein) · HGF gene · ABCD1 gene · ABCA3 gene · COL7A1 gene · Dystrophin gene · TGF-β3 (Transforming Growth Factor Beta 3) · CFTR gene
Marketed (4)
- Onasemnogene Abeparvovec-brve · Novartis Pharmaceuticals · Neuroscience
ITVISMA delivers a functional SMN1 gene using an AAV9 vector to promote SMN protein expression in motor neurons. - voretigene neparvovec-rzyl · Spark Therapeutics, Inc. · Ophthalmology
Voretigene neparvovec-rzyl is a gene therapy that delivers a functional copy of the RPE65 gene into retinal cells to restore vision in patients with inherited retinal dystrophy caused by RPE65 mutations. - Onasemnogene Abeparvovec-xioi · Novartis Gene Therapies · Neurology
Onasemnogene abeparvovec-xioi is a gene therapy that delivers a functional copy of the SMN1 gene via an adeno-associated virus vector to restore survival motor neuron protein production in patients with spinal muscular atrophy. - p53 gene therapy · Shenzhen SiBiono GeneTech Co.,Ltd · Oncology
This gene therapy delivers functional p53 tumor suppressor gene into cancer cells to restore apoptosis and cell cycle arrest.
Phase 3 pipeline (9)
- TissueGene-C · Kolon TissueGene, Inc. · Liver disease
TissueGene-C is a gene therapy that uses a viral vector to deliver the human hepatocyte growth factor (HGF) gene to the liver, promoting tissue repair and regeneration. - Topical Beremagene Geperpavec · Krystal Biotech, Inc. · Dermatology
Beremagene geperpavec is a gene therapy that uses a viral vector to deliver a gene to the skin, which then produces a protein to treat the condition. - AAV2-hRPE65v2,voretigene neparvovec-rzyl · Spark Therapeutics, Inc. · Ophthalmology
AAV2-hRPE65v2 is a gene therapy that delivers a functional copy of the human RPE65 gene into retinal pigment epithelium cells to restore vision in patients with inherited retinal dystrophy caused by RPE65 mutations. - EB-101 · Abeona Therapeutics, Inc · Neurology
EB-101 is a gene therapy that delivers a functional copy of the ABCD1 gene to restore adrenoleukodystrophy protein function in patients with cerebral adrenoleukodystrophy. - MDR-101 · Medeor Therapeutics, Inc. · Pulmonology / Rare Genetic Lung Disease
MDR-101 is a gene therapy that delivers a functional copy of the ABCA3 gene to restore surfactant production in patients with ABCA3-deficient interstitial lung disease. - KB803 · Krystal Biotech, Inc. · Dermatology / Rare Genetic Disorders
KB803 is a gene therapy that delivers a functional COL7A1 gene to restore type VII collagen production in patients with recessive dystrophic epidermolysis bullosa. - SRP-4045 · Sarepta Therapeutics, Inc. · Muscular dystrophy
SRP-4045 is a micro-dystrophin gene therapy that aims to treat Duchenne muscular dystrophy by providing a functional copy of the dystrophin gene. - TG-C · Kolon TissueGene, Inc. · Orthopedics / Regenerative Medicine
TG-C is a gene therapy that delivers TGF-β3 to cartilage tissue to promote cartilage regeneration and repair. - SGT-003 · Solid Biosciences Inc. · Muscular dystrophy
SGT-003 is a gene therapy that aims to deliver a functional dystrophin gene to muscle cells.
Phase 2 pipeline (9)
- 4D-710 · 4D Molecular Therapeutics · Cystic Fibrosis
4D-710 is a gene therapy that uses an adeno-associated virus to deliver a functional copy of the CFTR gene to the lungs, correcting the underlying genetic defect in cystic fibrosis. - AAV2-GDNF gene therapy · Brain Neurotherapy Bio, Inc. · Neurology
AAV2-GDNF gene therapy delivers a viral vector carrying the GDNF gene to the brain, where it is expressed and promotes the survival and growth of dopaminergic neurons. - AB-1002 · AskBio Inc · Genetic disorders
AB-1002 is a gene therapy that aims to treat certain genetic disorders by delivering a healthy copy of the gene. - ABO-101 · Abeona Therapeutics, Inc · Hematology/Oncology
ABO-101 is a gene therapy that delivers a functional copy of the FANCC gene to correct Fanconi anemia complementation group C deficiency. - AAV OPTIRPE65 · MeiraGTx UK II Ltd · Ophthalmology
Gene therapy targeting RPE65 - CTI-1601 · Larimar Therapeutics, Inc. · Neurology
CTI-1601 is a gene therapy that aims to restore motor neuron function in patients with amyotrophic lateral sclerosis (ALS). - CBLB612 · BioLab 612 LLC · Respiratory
CBLB612 is a gene therapy that aims to treat certain conditions by delivering a healthy copy of the CFTR gene to cells. - AVTX-801 · Eva Morava-Kozicz · Genetics
AVTX-801 is a gene therapy that aims to treat certain genetic disorders by replacing or repairing faulty genes. - AAV2-hRPE65v2 · Spark Therapeutics, Inc. · Ophthalmology
AAV2-hRPE65v2 is a gene therapy that uses a viral vector to deliver a healthy copy of the RPE65 gene to the retina, treating inherited retinal dystrophy.
Phase 1 pipeline (9)
- AAV - CNGB3 · MeiraGTx UK II Ltd · Ophthalmology
Gene therapy targeting the CNGB3 gene - AAV RPE65 · MeiraGTx UK II Ltd · Ophthalmology
Gene therapy for RPE65-mediated inherited retinal dystrophy - AVXS-101 · Novartis Gene Therapies · Rare Disease
Gene therapy for spinal muscular atrophy - AAV-hTERT · Libella Gene Therapeutics · Rare Disease
AAV-mediated delivery of the hTERT gene - AAV9.SLC6A1 Gene Therapy · Emily de los Reyes · Neurology
AAV9-mediated delivery of the SLC6A1 gene to the brain - AAV2hAQP1 · MeiraGTx, LLC · Ophthalmology
AAV2-mediated gene therapy targeting aquaporin-1 - AAV8.hLCA5 · Opus Genetics, Inc · Ophthalmology
Gene therapy for Leber congenital amaurosis 10 (LCA10) - AAV2-hCHM · Spark Therapeutics, Inc. · Ophthalmology
Gene therapy for Leber congenital amaurosis - AAV2-GDNF · Brain Neurotherapy Bio, Inc. · Neurology
AAV2-mediated delivery of glial cell line-derived neurotrophic factor