Gene Therapy

Image credit: National Human Genome Research Institute

Gene therapy is a technique that modifies a person’s genes to treat or cure disease.

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OrganizationCountryGene Therapy products / InnovationPreclinical / Clinical stageTreatmentCompany InfoAd/Banner/VideoCompany News
Personalize My MedicineUKPlatform for medical innovationNANAPMM keeps you informed about the latest advancements in medical innovation with a focus on personalized and precision medicine approaches to healthcareScreenshot 2023-02-20 at 09.43.05Screenshot 2023-02-20 at 08.35.30Subscribe to PMM's free monthly newsletter here!
Astellas Gene TherapiesUSAdeno-Associated Virus (AAV)-based gene therapiesNot listedAdeno-Associated Virus (AAV) based gene therapies for X-linked myotubular myopathy (XLMTM), Pompe disease, Duchenne muscular dystrophy and myotonic dystrophy
AAVogenUSAVGN7PreclinicalAVGN7 is currently being developed to treat inclusion body myositis, cancer cachexia and Duchenne muscular dystrophy, however additional disease indications are being consideredThe first row shows what your upgraded listing would look like. Click here to find out how you can upgrade your company's listing.
Abeona TherapeuticsUS The AIMª Vector, next-generation AAV capsids for AAV based gene therapies and vectors. Not listedEB-101 for Recessive Dystrophic Epidermolysis Bullosa (RDEB) ABO-102 for Sanfilippo Syndrome Type A (MPS IIIA) ABO-50X for Retinal Diseases
Adverum BiotechnologiesUSADVM-022 ADVM-062 Preclinical: ADVM-062 Clinical: ADVM-022ADVM-022, reduces the burden of frequent anti-VEGF injections, and improves vision outcomes for patients with wet age-related macular degeneration (wet AMD)
Affinia TherapeuticsUSAnc80L65, Anc80L65-trastuzumabPreclinical: Anc80L65, Anc80L65-trastuzumabAffinia Therapeutics is developing a next-generation gene therapy using a designed capsid, Anc80L65, to treat Metachromatic leukodystrophy. Anc80L65-trastuzumab, a gene therapy product candidate rationally designed to deliver a gene that programs the cells to produce trastuzumab, the antibody demonstrated as effective against this cancer
AGTCUSAGTC-501, ACHM CNGB3, ACHM CNGA3, AGTC-601Preclinical: AGTC-601 Clinical: AGTC-501, ACHM CNGB3 and ACHM CNGA3.AGTC-501 is being developed to treat XLRP. AGTC-601 a gene therapy program designed to treat Frontotemporal dementia
AkouosUSAK-OTOF Not listedAK-OTOF (OTOF-mediated Hearing Loss), potentially treats hearing, in individuals with sensorineural hearing loss, due to mutations in the otoferlin (OTOF) geneThe first row shows what your upgraded listing would look like. Click here to find out how you can upgrade your company's listing.
Alcyone TherapeuticsUSACTX-401 ACTX-101Clinical: ACTX-401 ACTX-101ACTX-401 is an AAV9-delivered gene replacement therapy for the treatment of a rare form of Spinal Muscular Atrophy (SMA) called SMA with Respiratory Distress type 1 (SMARD1), and for Charcot-Marie-Tooth type 2S (CMT2S). ACTX-101 is an AAV9-delivered X-reactivation gene therapy for the treatment of the X-linked genetic disorder Rett syndrome
AlnylamUS OnpattroNot listedOnpattro, a therapy for patients with a fatal rare disease transthyretin-mediated amyloidosis. Alnylam is currently using their novel RNAi platform to develop treatments for a very broad range of diseases including haemophilia, hypercholesterolemia, rare liver disease and hypertension
American Gene TechnologiesUS AGT103-TClinical: AGT103-TAGT103-T, an HIV therapeutic product is currently in Phase 1 clinical trials, and AGT lead candidates in Immuno-oncology and Phenylketonuria (PKU) programs are expected to move into clinical stages in a few years
AnGesUK, US and JapanHGF gene therapy product, NF-_B Decoy Oligonucleotide, DNA Vaccine, Tie2 agonists, Lonafarnib, Development of genome editingClinicalAnGes is promoting three projects: HGF gene therapy products for Chronic arterial occlusive disease, NF-_B decoy oligonucleotide for Chronic discogenic lumber back pain, and the development of DNA vaccines. Development of genome editing for Severe Congenital Neutropenia and Diseases in hematology, ophthalmology, immuno-oncology, etc.
Anjarium BiosciencesSwitzerlandHybridosome® delivery technologyNot listedNovel DNA-based vectors and proprietary Hybridosome® delivery technology, methodically combined for targeted tissue delivery. Anjarium´s non-viral gene therapy platform aims to address the underlying causes of many genetic diseases in a more predictable, repeatable, and lasting way.The first row shows what your upgraded listing would look like. Click here to find out how you can upgrade your company's listing.
Applied ViromicsUSProduction of AAV vectors and services for cloning genes of interest into shuttle plasmids N/AN/A
ArrowHead PharmaceuticalsUSARO-AAT, ARO-APOC3, ARO-ANG3, ARO-HSD, ARO-C3, ARO-ENaC2, ARO-MUC5AC, ARO-RAGE, ARO-MMP7,ARO-COV and ARO-DUX4 Preclinical: ARO-ENaC2, ARO-MMP7, ARO-COV and ARO-DUX4. Clinical: ARO-AAT, ARO-APOC3, ARO-ANG3, ARO-HSD, ARO-C3, ARO-MUC5AC and ARO-RAGE. ARO-AAT, developed to treat Alpha 1 Liver disease. ARO-APOC3, designed to treat Hypertriglyceridemia. ARO-ANG3 treats Dyslipidemia. ARO-HSD, designed to treat liver disease. ARO-C3 developed to treat Complement Mediated Disease.
ASC TherapeuticsUSASC-618, ASC-518, ASC-519, ASC-520Pre-Clinical: ASC-518, ASC-519, ASC-520 Clinical: ASC-618Gene Therapy Drug candidate: ASC-618 for Hemophilia A. Gene Editing Drug candidate: ASC-518 and ASC-519 for Hemophilia A and ASC-520 for beta-Thalassemia. Current pipeline is focused on blood diseases
Askepios BioPharmaceutical (AskBio)USAdeno-Associated Virus (AAV) gene therapyNot listedAAV gene therapy for Pompe disease and LGMD (2i/R9), Parkinson's disease, Multiple system atrophy and Huntington's disease, and Congestive heart disease
AviadoBioUKAVB-101PreclinicalAVB-101 is an investigational, one-time, adeno-associated virus (AAV) gene therapy for patients with Frontotemporal Dementia (FTD) with mutations in the Progranulin (GRN) gene
AvrobioUSAVR-RD-02, AVR-RD-03, AVR-RD-04 and AVR-RD-05Preclinical: AVR-RD-03, AVR-RD-05 Clinical:AVR-RD-02, AVR-RD-04AVR-RD-02 treats type 1 Gaucher disease and type 3 Gaucher disease. AVR-RD-03, gene therapy for Pompe disease. AVR-RD-04, investigational gene therapy for cystinosis. AVR-RD-05 is developed to treat mucopolysaccharidosis type II (MPS II), or Hunter syndrome
Axovia TherapeuticsUK, USAXV101, AXV102, AXV201, AXV301PreclinicalAxovia Therapeutic's novel gene therapy utilizes an adeno-associated virus (AAV9) to treat Bardet-Biedl Syndrome. Axovia Therapeutics is developing treatments for rare and devastating ciliopathies such as Bardet-Biedl Syndrome (BBS)
Bayer Germany Peboctocogene Camaparvovec (Factor VIII Gene Therapy) and gene therapies for Congestive heart failure, Pompe disease and ParkinsonÕs disease. Not listedDeveloped to treat Congestive heart failure, Pompe disease and Parkinson's disease
Beam Therapeutics USBEAM-101, BEAM-102Preclinical: BEAM-102 Clinical: BEAM-101BEAM-101 is an investigational therapy that produces base edits designed to potentially alleviate the effects of sickle cell disease. BEAM-102 is an investigational therapy designed to edit the causative hemoglobin S point mutation directly to recreate a naturally occurring normal human hemoglobin variant, HbG-Makassar
Bluebird BioUSZYNTEGLO (betibeglogene autotemcel), a one time gene therapy and My bluebird supportClinical: ZYNTEGLO¨ (betibeglogene autotemcel)ZYNTEGLO¨ treats beta thalassemia in patients who require regular red blood cell transfusions and My bluebird support, a personalized platform which supports the needs of patients who are considering the bluebird bio gene therapyThe first row shows what your upgraded listing would look like. Click here to find out how you can upgrade your company's listing.
Bluerock TherapeuticsUSCell and Gene platform, BRT-DA01Clinical: BRT-DA01CELL+ GENETM platform develops cellular treatments in the areas of neuology, cardiology, immunology, and Parkinson´s disease
CRISPR TherapeuticsUSexa-cel, CTX110, CTX112, CTX130, CTX131, VCTX210, VCTX211, VCTX212, CTX310Preclinical: CTX112, CTX131, VCTX211, VCTX212 and CTX310 Clinical: exa-cel, CTX110, CTX130 and VCTX210.Exa-cel is being developed to treat B-thalassemia and sickle cell disease. CTX110, CTX112, CTX130, CTX310 and CTX131 has applications in immuno-oncology. VCTX210 and VCTX211 is being developed to treat Type 1 diabetes mellitus and VCTX212 is being designed to treat Type 1/2 diabetes mellitus
Chimeron BioUSCB-101 CB-132 CB-137 CB-151Not listedCB-101 is an off-the-shelf personalized RNA drug designed to engage the immune system by upregulating tumor neoantigens. CB-132 and CB-137 are developed agents that target tumors. Developed ChaESAR RNA therapy for gene and metabolic disorders of the liver.
Capsida BiotherapeuticsUSAn Adeno-Associated Virus (AAV) engineering platform to generate capsids that bind only to tissues and cells a target organ. Generation 2 capsids Generation 4 capsidsN/ATissue targeted gene therapies, through Generation 2 capsids and Generation 4 capsids. Capsida technology allows the simultaneous targeting of multiple organs while still limiting the penetration of non-targeted organs
CapsigenUSTranscription-Dependent Directed Evolution (TRADE) technologyN/ATRADE technology eliminates background noise and efficiently identifies fully functional capsids designed to meet complex disease-specific transduction profiles. The TRADE platform can screen and identify capsids with enhanced levels of infectivity and transduction resulting in more potent medicines.The first row shows what your upgraded listing would look like. Click here to find out how you can upgrade your company's listing.
Carbon BiosciencesUSCGT-001Not listedCGT-001, utilizes the CBN-1000 vector which has demonstrated highly specific lung airway tropism, significantly increased packaging capacity and the ability to be readministered in animal models. CGT-001 is designed to enable airway delivery of the full length CFTR gene for Cystic Fibrosis, an approach that has been previously unachievable with current generation viral vectors.
Carmine TherapeuticsUSRed Cell EV Gene Therapy (REGENT®) platformNot listedRed Cell EV Gene Therapy (REGENT®) platform generates a pipeline of next-generation disease-modifying gene therapies for a broad spectrum of diseases
Castle Creek BiosciencesUSD-Fi Gene Therapy, LV-FAH Gene Therapy, FCX-007Clinical: FCX-007D-Fi Gene Therapy for Dystrophic Epidermolysis Bullosa (DEB) LV-FAH Gene Therapy for Hereditary Tyrosinemia Type 1 (HT1) FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa
Cellectis France UCART123, UCART22, UCARTCS1, UCART19, ALLO-501 and ALLO-715ClinicalIn development: UCART123 for acute myeloid leukemia (AML), UCART22 for relapsed and refractory B-cell acute lymphoblastic leukemia (R/R B-ALL), UCARTCS1 for CS1/SLAMF7-expressing hematologic malignancies, UCART19 for CD19-expressing hematological malignancies, engineered using TALEN® gene editing technology, and ALLO-501 and ALLO-715 for patients with relapsed/refractory multiple myeloma
cGMPnowUSGMP Manufacturing & QC Laboratory Equipment, Automation & Controls (IT/OT), Commissioning & Qualification, and Procurement services N/AN/AThe first row shows what your upgraded listing would look like. Click here to find out how you can upgrade your company's listing.
Coave TherapeuticsFrance CTx-TFEB, CTx-PDE6bClinical: CTx-PDE6bCTx-TFEB: optimized gene therapy to treat a-synucleinopathies CTx-PDE6b: A clinical stage AAV based gene therapy for treating PDE6b IRD
Coda BiotherapeuticsUSCODA´s engineered ligand-gated ion channelsN/ACODA´s engineered ligand-gated ion channels are selectively activated by orally bioavailable small molecules, and interact with the receptor in a dose-dependent manner to control the aberrant activity of neurons responsible for many neurological disorders. The gene encoding the engineered receptor can be delivered to dysfunctional neurons by AAV vectors. Their first two clinical applications are neuropathic pain and focal epilepsy.
CombiGeneSwedenCOZY02, CG01, CGT2Not listedCOZY02 gene therapy treatment for severe chronic pain conditions CG01 is an AAV-based gene therapy with the potential to improve the quality of life for patients with drug-resistant focal epilepsy for whom there is currently no effective treatment. CombiGene´s CGT2 project is to develop a gene therapy treatment for partial lipodystrophy, a very rare disease that today completely lacks adequate treatment.
4D Molecular TherapeuticsUSOpthalmology: 4D-150, 4D-125, 4D-110 Cardiology: 4D-310 Pulmonology: 4D-710Clinical4D-150, treatment for adults with Neovascular (wet) Age-Related Macular Degeneration (wet AMD). 4D-125, develops treatments for patients with XLRP related to mutations in the RPGR gene. 4D-110 develops treatments for patients with choroideremia related to mutations in the CHM gene. 4D-310, is an ongoing Phase 1/2 clinical trial in adult patients with classic (severe) Fabry disease. 4D-710: ongoing clinical trial for cystic fibrosis patients, ineligible for CFTR modulator therapy or who have discontinued therapy due to adverse effects.
Deep Genomics CanadaSteric Blocking Oligonucleotide (SBO) Therapies AI Workbench 1.0, AI Workbench 2.0, AI Workbench 3.0 N/ASteric Blocking Oligonucleotide (SBO) Therapies AI Workbench 1.0 corrects RNA splicing, to restore protein expression. AI Workbench 2.0, seven mechanisms to increase expression and reduce the work needed to identify efficacious compounds. AI Workbench 3.0 is in development, to support target identification and drug discovery for more common, complex diseases involving multiple genes. The first row shows what your upgraded listing would look like. Click here to find out how you can upgrade your company's listing.
DiNAQORSwitzerlandMonogenic Cardiomyopathies: BMD-293, DINA-003, DINA-002, DINA-004/5, DINA-006. Monogenic Renal Diseases: DINAK-001, DINAK-002, DINAK-003, DINAK-004Preclinical: DINA-002, DINA-004/5, DINA-006 and DINAK-001 Clinical: BMD-293BMN293 is under phase 1 clinical trial to treat Hypertropic Cardiomyopathy. DINA-002 is being deveoped to treat Arrhythmogenic Cardiomyopathy. DINA-004/5 and DINA-006 is being developed to treat Dilated Cardiomyopathy. Dinak-001 is being developed to treat Autosomal Dominant Polycystic Kidney Diseases.
DNAgoUSGenetic test Products: Genetic Cancer test kit, Rare Disease test kit, Genetic test kit, Full Wellness and Ancesty kit. N/ADNAGO provides diagnostic testing for patients, providing patients with a quick solution to help manage their lifestyle. Diagnostic tests includes all rare genetic diseases, including Cystic fibrosis, Fabry disease, Epilepsy, Hemophilia, all types of hereditary cancers.
Editas Medicine USINVIVO Gene Edited Medicines: EDIT-101, EDIT-103 and EDIT-102 EXVIVO Gene Edited Medicines: EDIT-301Clinical: EDIT-301EDIT-101: Leber Congenital Amaurosis 10, EDIT-103: Rhodopsin-Associated Autosomal Dominant Retinitis Pigmentosa and EDIT-102: Usher Syndrome 2A. EDIT-301: Sickle Cell Disease and EDIT-301: Transfusion-Dependent Beta-Thalassemia
eGenesisUSHuCo kidney, HuCo heart, HuCo islet cells, HuCo liver perfusionN/ATreatment for patients with organ failure and other debilitating diseases
Elevate BioUSHighPassBio, AlloVirNot listedHighPassBio for advancing novel targeted T cell immunotherapies to manage post-hematopoietic stem cell transplant leukemic relapse. AlloVir, Cell and Gene Therapy and T cell immunotherapies targeting BK Virus, Cytomegalovirus, Adenovirus, Epstein-Barr and Human Herpes virus.The first row shows what your upgraded listing would look like. Click here to find out how you can upgrade your company's listing.
Emendo BiotherapeuticsUSOMNI™ technology platform and a Program performing allele specific editingN/AEmendo´s OMNI™ technology platform enables extreme precision gene editing customized to any target. Allele Specific Editing to Cure Dependent Severe Congenital Neutropenia (SCN)
Encoded TherapeuticsUSETX101, Envision is an ongoing observational study of infants and children with SCN1A+ Dravet syndrome. Endeavour is a first-in-human investigational trial designed to study safety and efficacy of ETX101 in young children with SCN1A+ Dravet syndrome.ClinicalETX101, their lead program, is specifically designed to address the underlying cause of Dravet syndrome, the most common developmental and epileptic encephalopathy.
Exegenesis BioUS, SingaporeExegenesis Bio's gene therapy pipeline focuses on the CNS, Ocular, and Liver diseases. They have also made developments in treating Spinal Muscular Atrophy, Parkinson's Disease, Wet Age-related macular degeneration, Bietti crystalline dystrophy (BCD), Hemophilia A (HA) and Phenylketonuria (PKU). AAVarta®, Constellation®, Progress®.Not listedAAVarta® is an in-silico evolution discovery platform, utilizing cutting-edge AI technologies to design novel AAV capsids. Constellation® is an AI aided gene expression cassette design platform achieving tissue-specific regulation through the development of de-targeting microRNAs and synthetic promoters. Progress® is a data-driven protein engineering platform.
Forge BiologicsUSGene Therapies: FBX-101, FBX-201. The RESKUE TrialClinical: FBX-101FBX-101 is being developed to treat Krabbe Disease. The RESKUE Trial - a Phase 1/2 Clinical Trial for Patients with Krabbe Disease. FBX-201 is being developed for rare monogenic diseases.
Freeline Therapeutic USFLT180a FLT190 FLT201 AAV Vector Platform and Manufacturing platformClinical: FLT190 and FLT201FLT180a: next-generation AAV gene therapy for haemophilia B patients. FLT190, developed for the treatment of Fabry disease. FLT201, developed for the treatment of type 1 Gaucher disease. The first row shows what your upgraded listing would look like. Click here to find out how you can upgrade your company's listing.
Fujifilm DiosynthUK, USViral vector platforms, Triple transfection plasmid technologyN/AN/A
GenSight Biologics S.A.FranceGS011 GS030Preclinical: GS011 Clinical: GS030GS011 treats Leber hereditary optic neuropathy. GS030 treats Retinitis Pigmentosa, Dry AMD and Geographic Atrophy
GeneCure BiotechnologiesUSSimVecª, HIVAX GeneCure is currently developing both preventative and therapeutic vaccines against a number of human diseases: Therapeutic HIV-1 vaccine, Prophylactic HIV-1 vaccine, Therapeutic HCV vaccine, Prophylactic HCV vaccine and Therapeutic HBV vaccine. Preclinical: Prophylactic HIV-1 vaccine, Prophylactic HCV vaccine and Therapeutic HBV vaccine. Clinical: Therapeutic HIV-1 vaccine, Therapeutic HCV vaccine, GeneCure has developed a patented gene transfer technology (SimVecª) based on a primate lentivirus, the simian immunodeficiency virus. HIVAX is a patented replication-defective HIV-1 vaccine which is capable of stimulating both cellular and antibody immune responses in both animal models and humans.
Generation BioUSceDNA (closed-ended DNA) Cell-targeted lipid nanoparticle (ctLNP), non viral delivery system Capsid-free manufacturing process N/AN/A
GenethonFranceGene Therapy Products: GNT 0004, GNT 0006, GNT 0007, GNT 0008, Preclinical: GNT 0006, GNT 0007 and GNT 0008. Clinical: GNT 0004GNT 0004 for Duchenne Muscular Dystrophy. GNT 0006 has been developed to treat Limb girdle muscular dystrophies. GNT 0007 for Gamma sarco-glycanopathy and GNT 0008 for Calpa•nopathy. Gene therapy products have been developed for Wiskott-Aldrich syndrome, Fanconi's anemia, Crigler-Najjar syndrome, Pompe disease, Cori-Forbes Disease and Leber Hereditary Optic Neuropathy. The first row shows what your upgraded listing would look like. Click here to find out how you can upgrade your company's listing.
GenprexUS REQORSA Immunogene Therapy, through an ONCOPREX Nanoparticle Delivery System. Gene Therapy products: GPX-002 and GPX-003 through an AAV vector. Preclinical: GPX-002 and GPX-003 through an AAV vector. Clinical: REQORSA Immunogene Therapy, through an ONCOPREX Nanoparticle Delivery SystemLead product candidate REQORSA Immunogene Therapy for Non-Small Cell Lung Cancer and Small cell lung cancer. Preclinical diabetes candidates, GPX-002 for Type 1 Diabetes and GPX-003 for Type 2 Diabetes.
Global BioTherapeuticsUSGene therapy designed to avoid viremia, ensure no cytokine storm, no apoptosis, no liver inflammation, no significant liver damage and sustained transgene expressionN/AGBT's Platform Technology has the potential to treat Hemophilia, Rheumatoid Arthritis, Diabetes, Chronic Pain and HIV / AIDS. Potential areas of therapeutic use include Alpha-1-antitrypsin deficiency, Cancer, Multiple Sclerosis and Inborn Errors of Metabolism.
Graphite BioUSNula-cel formerly known as GPH101 and GPH102.Preclinical: GPH102 Clinical: Nula-cel Nula-cel is our investigational, next-generation, gene-edited autologous stem cell-based therapy that is currently in development for sickle cell disease (SCD). GPH102 is our research program for the treatment of beta-thalassemia.
Gyroscope Therapeutics, A Novatris CompanyUK, USGyroscopeÕs lead investigational gene therapy, GT005ClinicalGyroscope´s lead investigational gene therapy, GT005, is currently being evaluated in Phase II clinical trials for the treatment of geographic atrophy (GA), the advanced form of dry AMD
Halo LabsUSAura™ family systems Gene Therapy products include AURA+ and AURA GTN/AN/AThe first row shows what your upgraded listing would look like. Click here to find out how you can upgrade your company's listing.
Haya Therapeutics SwitzerlandDiscoverHAYA™, HTX-001Preclinical: HTX-001 DiscoverHAYA™ is a proprietary drug discovery engine that enables us to discover and generate a pipeline of lncRNA targeting anti-fibrotic candidates for many tissues including lung, kidney, liver and the tumor microenvironment. HTX-001 for the inhibition of cardiac fibrosis_
Homology MedicinesUSGene Editing (Nuclease Free): HMI-103 and in human stem cells. Gene Therapy products: HMI-102, HM-203, HM-204Preclinical: HM-204 Clinical: HMI-103, HMI-102 and HM-203Homology is conducting its Phase 1, open label, dose-escalation clinical trial (called the pheEDIT study) with the HMI-103 gene editing candidate for patients with PKU. HMI-102 gene therapy for adult PKU patients. HMI-203 Gene Therapy for Patients with Hunter syndrome Homology is conducting a Phase 1 open-label dose-escalation clinical trial (called the juMPStart study). HMI-204 is being developed to treat Metachromatic Leukodystrophy.
Horizon DiscoveryPin-point™ Base Editing platform, CRISPRmod product platform, Dharmacon™ reagentsN/AN/A
Intellia TherapeuticsUSNTLA-2001, NTLA-2002, NTLA-2003, NTLA-3001, NTLA-6001Preclinical: NTLA-2003, NTLA-3001, NTLA-6001 Clinical: NTLA-2001, NTLA-2002NTLA-2001 is being developed to treat Transthyretin (ATTR) Amyloidosis. NTLA-2002 is being designed to treat HAE. NTLA-3001 is Intellia´s first and wholly owned CRISPR/Cas9-mediated in vivo targeted gene insertion development candidate.
iECUREUSInvivo gene insertion: ARCUS® Dual AAV with ARCUS NucleaseN/APrograms currently focused on developing in vivo gene insertion therapies for treatment of rare pediatric liver diseases. Dual Adeno-Associated Virus (AAV) with ARCUS Nuclease have been developed to treat OTC, Citrullinemia Type 1 and PKU. Their gene editing technology is being developed to treat ornithine transcarbamylase (OTC) deficiency and citrullinemia type 1 (argininosuccinate synthase or ASS deficiency).
InnoSkelFranceGene Therapy product: INS-101 PreclinicalINS-101, a gene therapy for type II collagen disorders, which has demonstrated strong efficacy in preclinical studies
IVERIC bioUSAdeno-Associated Virus (AAV) gene therapy products: Mini-CEP290, mini-ABCA4, mini-USH2A*PreclinicalIn development: Mini-CEP290 for Leber congenital amaurosis type 10, mini-ABCA4 for  Stargardt disease, and mini-USH2A* to treat Usher syndrome type 2A
Jaguar Gene Therapy USGene Therapy products: JAG101, JAG201, JAG301PreclinicalJAG101, an investigational gene therapy that aims to deliver a gene replacement solution to address the root cause of Type 1 galactosemia. JAG201 is being developed for disorders with SHANK3 mutation or deletion. JAG301 is being developed to treat type 1 diabetes.
Krystal BiotechUSB-VEC, KB105, KB104, KB407, KB408, JN301 (previously known as KB301)Preclinical: KB104 and KB408 Clinical: B-VEC, KB105, KB407 and JN301Krystal Biotech uses STAR-D technology to develop topical treatments for rare or orphan dermatological indications
Kriya TherapeuticsUSComputational engine SIRVE™, cutting edge GMP manufacturing facility, R&D engine and a portfolio of gene therapiesN/ASIRVE™ integrates large volumes of data generated by Kriya´s engineering and screening platforms. R&D engine accelerates the design and development of products. Developing gene therapies for the treatment of diseases of high unmet need, with a focus on metabolic disorders, neurology and ophthalmology
LogicBioUSGeneRideT™, sAAVy™N/AGeneRide™ is designed to transform gene editing with higher levels of precision and durability to target different needs based diseases. Novel capsid platform, sAAVy™ is designed to enable the delivery of gene therapy and gene editing vectors with more potent expression and tissue specificity
MeiraGTxUSAAV-RPGR, AAV-RPE65, AAV-CNGB3, AAV-CNGA3, AAV-AIPL, A007, A008, A006, Pre Clinical: A007, A008 and A006 Clinical: AAV-RPGR, AAV-RPE65, AAV-CNGB3 and AAV-CNGA3.MeiraGTx has six programs in clinical development, including Phase 1/2 clinical stage programs in Achromatopsia, X-Linked Retinitis Pigmentosa, RPE65-Deficiency, radiation-induced xerostomia, and Parkinson's disease
MetagenomiUSAI-enabled discovery process and have a vast array of active editing systems N/ACurrent pipeline includes development of novel in vivo genetic medicines for Hemophilia A and Cystic Fibrosis
Neurophth TherapeuticsChina, USGene Therapy products: NFS-01, NFS-02, NFS-04, NFS-05. and NFS-10Preclinical: NFS-02, NFS-04, NFS-05 and NFS-10 Clinical: NFS-01In development: NFS-01 and NFS-02 for Leber´s Hereditary Optic Neuropathy. NFS-04 for Optic Neuropathy (Glaucoma, etc.), NFS-05 for Autosomal Dominant Optic Atrophy, and NFS-10 is being developed to treat Vascular Retinopathy (wAMD, DME, etc.)
Novartis AG (Was AveXis)USMore than 20 projects in gene therapy pipeline and around 162 projects in clinical pipelineMultiple therapies and projects in Preclinical and Clinical stages.Development of gene therapies for Cardiovascular health, Renal health, Metabolic health and in Hematology, Immunology, Neuroscience, Opthalmology, Solid Tumours, and Respiratory and allergies.The first row shows what your upgraded listing would look like. Click here to find out how you can upgrade your company's listing.
Opus GeneticsUSGene Therapy programs: OPGx-001, OPGX-002, OPGX-003Preclinical: OPGX-002, OPGX-003 Clinical: OPGx-001Development of treatments delivered sub-retinally to address mutations in genes that cause different forms of Leber congenital amaurosis (LCA)
Orchard Therapeutics (ORTX)UKOTL-200 is an ex vivo autologous hematopoietic stem cell gene therapy. OTL-203 is an ex vivo autologous gene therapy. Strimvelis is a gammaretroviral vector-based gene therapy. OTL-103 is an ex vivo autologous gene therapy.Preclinical: OTL-103 Clinical: OTL-200, OTL-203, StrimvelisHematopoietic stem cell (HSC), gene therapy approach: OTL-200 is the only approved treatment for Metachromatic leukodystrophy (MLD). OTL-203 treatment for Mucopolysaccharidosis type I (MPS-I). Strimvelis treatment for patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID). OTL103 treatment for Wiskott Aldrich syndrome (WAS).
Oxford BioMedicaUKOXB-302, OXB-40X*, OXB-40Y*, OXB-40Z*, AXO-Lenti-PDPreclinical: OXB-302, OXB-40X*, OXB-40Y*, OXB-40Z* Clinical: AxoÐLentiÐPD^OXB-302 is a gene-based immunotherapy targeting blood cancer. OXB-40X*, OXB-40Y*, and OXB-40Z* developed for an undisclosed liver indication. AXO-Lenti-PD is a gene-based treatment for Parkinson's disease.
Passage BioUSPBGM01, PBKR03, PBML04, PBFT02ClinicalGene therapy treatments in development: PBGM01 for GM1 gangliosidosis. PBKR03 for  Krabbe disease. PBML04 for metachromatic leukodystrophy. PBFT02 for Frontotemporal dementia.
Poseida Therapeutics USGene Therapy programs: P-OTC-101 and P-FVIII-101Not listedP-OTC-101 is a liver-directed gene therapy combining piggyBac technology with Adeno-Associated Virus (AAV) for the in vivo treatment of OTC deficiency. PiggyBac Factor VIII is a liver-directed gene therapy combining piggyBac technology with our nanoparticle delivery technology for the in vivo treatment of Hemophilia A.The first row shows what your upgraded listing would look like. Click here to find out how you can upgrade your company's listing.
Prevail TherapeuticsUSAAV9-based gene therapies: PR001, PR006ClinicalPR001 is being developed to treat Parkinson's disease with GBA1 mutations, type 2 Gaucher disease, and type 1 Gaucher disease. PR006 is being developed to treat Frontotemporal dementia with GRN mutations.
Prime MedicineUSPrime EditingN/ANext generation editing treats disease-causing mutations in a variety of organisms, organs, and cell types.
Regenxbio Inc.USRGX-314, RGX-202, RGX-121, RGX-111, RGX-181, RGX-381Preclinical: RGX-181, RGX-381 Clinical: RGX-314, RGX-202, RGX-121, RGX-111RGX-314 for Wet Age-related macular degeneration (AMD). RGX-202 for Duchenne Muscular dystrophy. RGX-121 for Hunter Syndrome, and RGX-111 for Hurler Syndrome. RGX-181 and RGX-381 are being developed to treat Batten disease.
Rejuvenate BioUSRJB-01, RJB-02 and RJB-03PreclinicalRJB-01 is expected to have applications in cardiac, metabolic, renal areas and in animal health. RJB-02 is expected to be pivotal in the treatment of joints. RJB-03 has potential applications in cardiac, neuro, muscle, metabolic, joint, and animal health.
Renova TherapeuticsUSRT-100 gene therapy and RT-200 gene therapyPreclinical: RT-200 gene therapy Clinical: RT-100 gene therapyRT-100 gene therapy is being developed to treat Heart Failure and RT-200 gene therapy is being developed to treat Type 2 diabetesThe first row shows what your upgraded listing would look like. Click here to find out how you can upgrade your company's listing.
Rocket PharmaceuticalsUSAAV RP-A501, LV RP-L102, LV RP-L201, LV RP-L301ClinicalLV RP-L102 is being developed to treat Fanconi Anemia. LV RP-L201 is being designed to treat Leukocyte Adhesion Deficiency-1 and LV RP-L301 is being developed to treat Pyruvate Kinase Deficiency. Adeno-Associated Vector (AAV) RP-A501 is being developed to treat Danon Disease.
Sangamo TherapeuticsUSSB-525 TX200 ST-920 SAR445136 ST-400Not listedSangamo Therapeutics has partnered with Pfizer to develop its lead program SB-525, a gene therapy developed to treat hemophilia A. The company is also conducting phase 1/2 studies of TX200, a gene-edited cell therapy for preventing rejection of transplanted kidneys in patients with end-stage renal disease, ST-920 for Fabry disease, SAR445136 for sickle cell disease, and ST-400 for β-thalassemia.
Sarepta Therapeutics IncUSGALGGT2, GNT 0004, SRP-9001, SRP-9003, SRP-9004, and SRP-6004ClinicalIn development: GALGGT2, GNT 0004, and SRP-9001 for Duchenne Muscular Dystrophy. SRP-9003 for LGMD2E/R4 β-sarcoglycan, SRP-9004 for LGMD2D/R3 α-sarcoglycan and SRP-6004 for LGMD2B/R2 Dysferlin
Sio Gene TherapiesUSAAV-based gene therapiesNot listedClinical stage treatments include Adeno-Associated Virus (AAV) based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases. Gene therapies for highly prevalent conditions such as Parkinson´s disease are also in development.
Solid BiosciencesUSNeuromuscular: SGT-003 Duchenne, AVB-202 Friedreich's ataxia Cardiac: AVB-401 BAG3 DCMPreclinical SGT-003 is a next generation Adeno-Associated Virus (AAV) gene therapy candidate developed to treat Duchenne. AVB-202 is a novel gene transfer product candidate being developed for the treatment of Friedrich´s ataxia. AVB-401 is a novel gene transfer product candidate being developed for the treatment of BAG3 mediated dilated cardiomyopathy (BAG3).The first row shows what your upgraded listing would look like. Click here to find out how you can upgrade your company's listing.
Spark Therapeutics USGene Therapy Programs: SPK-8011, SPK-8016, Fidanacogene elaparvovec, previously SPK-9001 and SPK-3006ClinicalSPK-8011 and SPK-8016 are investigational gene therapies for Hemophilia A. Fidanacogene elaparvovec, previously SPK-9001, is an investigational bio-engineered Adeno-Associated Virus (AAV) vector utilizing a high-activity F9 transgene for Hemophilia B, or factor IX deficiency. SPK-3006 is an investigational gene therapy for the potential treatment of Pompe disease.
SQZ BiotechnologiesUSOncology: SQZ APCs, SQZ eAPCs, SQZ AACs Auto-immunity: SQC TACs Infectious disease: SQZ eAPCs Preclinical: SQC TACs, SQZ eAPCs Clinical: SQZ APCs, SQZ eAPCs, SQZ AACsSQZ APCs, SQZ eAPCs, SQZ AACs has applications in oncology. SQC TACs is being developed to treat Celiac Disease and Type 1 Diabetes and SQZ eAPCs is being developed to treat Chronic Hepatitis B Virus.
StrideBioUSSTRX-330, STRX-110, STRX-210, STRX-230Not listedSTRX-330 is being developed to treat Arrhythmogenic Right Ventricular Cardiomyopathy. STRX-110 is being developed to treat Friedreich's Ataxia. STRX-210 developed to treat Niemann Pick Disease Type C. STRX-230 developed to treat Rett Syndrome.
Taysha Gene TherapiesUSNeurodegenerative diseases: TSHA-120, TSHA-118 Neurodevelopmental disorders: TSHA-102 Genetic Epilepsies: TSHA-105Preclinical: TSHA-105 Clinical: TSHA-120, TSHA-118, TSHA-102TSHA-120, AAV9 gene therapy program developed for the treatment of giant axonal neuropathy. TSHA-102 is being developed for the treatment of Rett syndrome. TSHA-118 is being developed for the treatment of CLN1 disease, also known as infantile Batten disease, a rapidly progressing rare lysosomal storage disease with no approved treatment.
Tessera TherapeuticsUS RNA-based GENE WRITINGª DNA-based GENE WRITINGªN/AGene writing technology writes therapeutic messages into the genome to treat previously untreatable diseasesThe first row shows what your upgraded listing would look like. Click here to find out how you can upgrade your company's listing.
Tune TherapeuticsUSGenetic tuning platform, TEMPOTMN/ATEMPOTM, is able to control the way genes behave in a graded, tunable, and reversible manner, as compared to the all-or-nothing, irreversible interventions (deletions and knockouts) common to most gene editing approaches. TEMPO can silence specific genes without risking collateral damage to targeted (or non-targeted) DNA and has the unique capacity to tune and modulate specific genes as needed.
uniQureNetherlandsGene therapy programs: AMT_061 (has been approved), AMT_191, AMT_130, AMT_260, AMT_210, AMT_240 and AMT_161 Preclinical: AMT_191, AMT_260, AMT_210, AMT_240 and AMT_161 Clinical: AMT_061 and AMT_130Programs treat the following: Hemophilia B etranacogene dezaparvovec (AMT_061), Fabry Disease (AMT_191), Huntington´s Disease (AMT_130), Temporal Lobe Epilepsy (AMT_260), ParkinsonÕs Disease (AMT_210), Autosomal Dominant Alzheimer´s Disease (AMT_240) and Amyotrophic Lateral Sclerosis (AMT_161)
UltragenyxUSThe Pinnacle PCLª Platform: Optimizing AAV Gene Therapy, UX111, DTX401, DTX301, UX701, UX055, UX810Preclinical: UX055, UX810 Clinical: UX111, DTX401, DTX301, UX701,Gene Therapy programs treat the following: Mucopolysaccharidosis Type IIIA (UX111), Glycogen Storage Disease Type Ia (DTX401), Ornithine Transcarbamylase Deficiency (DTX301), Wilson Disease (UX701), CDKL5 Deficiency Disorder (UX055) and Duchenne Muscular Dystrophy (UX810)
Vertex PharmaceuticalsUSVX-522, CRISPR/Cas9Clinical: VX-522 VX-522, CFTR mRNA delivered to the lung via nanoparticles to treat the underlying cause of CF lung disease. CRISPR Therapeutics are helping them develop treatments for cystic fibrosis.
Verve TherapeuticsUS VERVE-101/ABE-PCSK9, ANGPTL3Preclinical: ANGPTL3 Clinical: VERVE-101/ABE-PCSK9VERVE-101, is designed to be a single-course in vivo liver gene editing treatment for Heterozygous Familial Hypercholesterolemia. ANGPTL3 Program has recently emerged as a new and promising target for severe hyperlipidemia.The first row shows what your upgraded listing would look like. Click here to find out how you can upgrade your company's listing.
VinetiUSVineti´s Personalized Therapy Management¨N/AVineti is a clinical-to-commercial cloud-based platform for cell and gene therapy supply chain management
Vivet TherapeuticsFranceVTX-801, VTX-802, VTX-803, VTX-804Preclinical: VTX-802, VTX-803, VTX-804 Clinical: VTX-801VTX-801 developed to treat Wilson's disease. VTX-802 & VTX-803 Progressive Familial Intrahepatic Cholestasis (PFIC) VTX-804 developed to treat Citrullinemia type I
VizgenUSMerfish and the Mescope platformN/AN/A
Voyager TherapeuticsUSVoyager´s SOD1 knockdown program, FXN Gene Therapy and GBA1 Gene TherapyPreclinicalVoyager´s SOD1 knockdown program aims to address ALS caused by SOD1 mutations FXN Gene Therapy (Gene Replacement) for Friedreich's ataixa. GBA1 Gene Therapy (Gene Replacement) for Parkinson's and other neurological diseases.
XyloCor TherapeuticsUSGene Therapies: XC001 and XC002Preclinical: XC002 Clinical: XC001 XC001 is an investigational gene therapy for patients with refractory angina. Gene therapy with XC001 has the potential treat Coronary Artery Bypass Graft Surgery, which may reduce the incidence of incomplete revascularization. XC002 is being developed for the regeneration of cardiac tissue in patients who have poorly functioning hearts due to heart attacks and heart failure.The first row shows what your upgraded listing would look like. Click here to find out how you can upgrade your company's listing.
Yposkesi FranceTwo platforms for AAV and lentiviral vector manufacturingNot listedN/A