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Gene Therapy Companies and Organizations

Companies working with Gene Therapy also work in 62 other areas:

The number of employers follows the core activity.

121 Employers in 166 Locations

4D Molecular Therapeutics

Business Area(s): 
  • Gene Therapy
    • Description: 

    The company designs, develops and commercializes transformative gene therapeutic products ...

    Abintus

    Description: 

    Abintus Bio, Inc. is a preclinical stage gene therapy company ...

    Adverum Biotechnologies

    Business Area(s): 
  • Gene Therapy
  • Ophthalmology
    • Description: 

    Adverum is a clinical-stage gene therapy company targeting unmet medical ...

    AGTC (Applied Genetics Technology Corp)

    Description: 

    AGTC is developing genetic therapies to treat patients with rare ...

    Akouos

    Business Area(s): 
  • Gene Therapy
  • Hearing
    • Description: 

    Akouos is a precision genetic medicine company focused on developing ...

    American Gene Technologies

    Business Area(s): 
  • Genetic Disease
  • Gene Therapy
    • Description: 

    American Gene Technolgies is devleoping therapeutics based on lentiviral vectors ...

    AnGes

    Business Area(s): 
  • Gene Therapy
  • COVID-19
  • Vaccines
    • Description: 

    AnGes focuses on research and development of gene medicine.

    Angionetics

    Business Area(s): 
  • Gene Therapy
  • Cardiovascular
    • Description: 

    Angionetics Inc., is a company focused on the late-stage clinical ...

    Annapurna

    Description: 
    Gene Therapy for Friedrich's ataxia

    Apic Bio

    Description: 

    Apic Bio develops gene therapy for patients with genetic ...

    Applied Viromics

    Business Area(s): 
  • Gene Therapy
    • Description: 
    custom-made recombinant viral vectors

    Aruvant Sciences

    Business Area(s): 
  • Gene Therapy
  • Blood
    • Description: 

    Aruvant Sciences is focused on developing and commercializing gene therapies ...

    AskBio

    Business Area(s): 
  • Cells
  • Gene Therapy
  • AAV
    • Description: 

    AskBio is a gene therapy company working with AAV on ...

    Audentes Therapeutics (Astellas)

    Business Area(s): 
  • Gene Therapy
    • Description: 

    AAV Gene Therapy Treatments. Bought by Astellas Dec. 2019.

    Aum LifeTech

    Business Area(s): 
  • Gene Therapy
    • Description: 
    Gene silencing technology

    Avalanche Biotechnologies

    Business Area(s): 
  • Gene Therapy
    • Description: 
    Adeno-Associated Virus Delivery, Gene Therapy

    AveXis (Novartis)

    Business Area(s): 
  • Gene Therapy
    • Description: 

    Gene therapies for rare and orphan neurological genetic diseases

    AVROBIO

    Business Area(s): 
  • Cells
  • Gene Therapy
    • Description: 

    AVROBIO, Inc., develops lentiviral-based gene therapies

    Axovant Gene Therapies

    Description: 

    Axovant Gene Therapies (formerly Axovant Biosciences) is developing gene therapies ...

    Bayside Biosciences

    Description: 

    The company provides services in bioanalytic, molecular and cell biology, ...

    Beam Therapeutics

    Description: 

    Beam Therapeutics is pioneering the use of CRISPR base editing ...

    Bellicum Pharmaceuticals

    Description: 

    Develops next-generation product candidates in some of the most important ...

    Bristol Myers Squibb

    Description: 

    A biopharmaceutical company. They discover, develop, and deliver medicines to ...

    CALiMMUNE

    Business Area(s): 
  • Gene Therapy
    • Description: 
    Gene Therapy

    Carmine Therapeutics

    Business Area(s): 
  • Gene Therapy
    • Description: 

    Carmine Therapeutics is an Esco Ventures X company that is ...

    Cellectis

    Description: 

    Designing next generation immunotherapies based on gene-edited CAR T-cells

    Cellomics

    Business Area(s): 
  • Gene Therapy
    • Description: 
    Lentiviral vectors

    Coda Biotherapeutics

    Business Area(s): 
  • Gene Therapy
  • Neurobiology
    • Description: 

    CODA seeks to treat millions of people with intractable neurological ...

    CombiGene

    Business Area(s): 
  • Gene Therapy
  • Epilepsy
    • Description: 

    CombiGene is currently conducting two gene therapeutic development projects: one ...

    DalCor Pharmaceuticals

    Business Area(s): 
  • Gene Therapy
    • Description: 

    Genetically targeted medicine

    DiNAQOR

    Business Area(s): 
  • Gene Therapy
  • Cardiovascular
    • Description: 

    DiNAQOR’s mission is to develop gene therapy solutions that transform ...

    DNAlite Therapeutics

    Business Area(s): 
  • Gene Therapy
    • Description: 

    DNAlite Therapeutics, Inc. is an early-stage preclinical biotechnology company developing ...

    DNAVEC

    Business Area(s): 
  • Gene Therapy
    • Description: 
    Viral Vectors & Gene Therapy

    Dyno Therapeutics

    Description: 

    Dyno Therapeutics is pioneering an artificial intelligence (AI) powered approach ...

    ElevateBio

    Description: 

    ElevateBio’s business model, including BaseCamp, our centralized R&D and manufacturing ...

    Emendo Biotherapeutics

    Description: 

    Emendo uses synthetic biology and genome-editing to treating diseases.

    Encoded Therapeutics

    Description: 

    Encoded identifies sequences within the human genome that control gene ...

    Errant Gene Therapeutics

    Business Area(s): 
  • Gene Therapy
    • Description: 
    Gene Therapy, Orphan Diseases

    evitria

    Description: 

    evitria specializes in CHO-based transient expression of antibodies (including bispecific ...

    Fibrocell Science (Castle Creek)

    Description: 

    Fibrocell, a cell and gene therapy company, is focused on ...

    Freeline Therapeutics

    Business Area(s): 
  • Gene Therapy
    • Description: 

    Freeline is a biopharmaceutical company focused on the development and ...

    Fujifilm Diosynth

    Description: 

    Fujifilm Diosynth Biotechnologies is a contract manufacturing organization that produces ...

    GeneCure Biotechnologies

    Business Area(s): 
  • Gene Therapy
    • Description: 
    Gene Transfer Technology

    Generation Bio

    Business Area(s): 
  • Gene Therapy
    • Description: 

    Generation Bio seeks to develop dosable genetic therapies similar to ...

    Genethon

    Business Area(s): 
  • Gene Therapy
    • Description: 
    Gene Therapy

    GenSight Biologics

    Business Area(s): 
  • Ophthalmology
  • Gene Therapy
    • Description: 
    Ophthalmic Gene Therapy

    GenVec

    Business Area(s): 
  • Gene Therapy
    • Description: 
    Gene Therapy

    Global BioTherapeutics

    Business Area(s): 
  • Gene Therapy
    • Description: 
    Gene Therapy platform technology

    Greffex

    Business Area(s): 
  • Vaccines
  • COVID-19
  • Gene Therapy
    • Description: 

    Greffex’s platform is a gene therapy delivery vehicle (vector) with ...

    Homology Medicines

    Business Area(s): 
  • Gene Therapy
  • Rare Diseases
    • Description: 

    Homology Medicines is working to use gene editing and gene ...

    HORAMA

    Business Area(s): 
  • Gene Therapy
  • Ophthalmology
    • Description: 
    HORAMA is developing innovative gene therapy medicinal products in ...

    iCell Gene Therapeutics

    Business Area(s): 
  • Gene Therapy
    • Description: 

    Gene Therapy for Cancer

    Immusoft

    Description: 

    Engineers B cells through gene therapy to produce proteins that ...

    InnoSkel

    Business Area(s): 
  • Bone
  • Gene Therapy
    • Description: 

    InnoSkel is a platform biotechnology company developing therapies ...

    InvivoGen Therapeutics

    Business Area(s): 
  • Gene Therapy
  • Immunotherapy
    • Description: 
    Gene and Immunotherapies

    IVERIC bio

    Business Area(s): 
  • Gene Therapy
    • Description: 

    IVERIC bio is a gene therapy focused company developing innovative ...

    Kriya Therapeutics

    Business Area(s): 
  • Gene Therapy
    • Description: 

    Kriya is a next-generation gene therapy company.

    Krystal Biotech

    Business Area(s): 
  • Gene Therapy
  • Skin
    • Description: 

    Krystal Biotech, Inc. is using gene therapy to develop effective ...

    Kytopen

    Description: 

    Kytopen is an MIT startup developing their proprietary Flowfect TM ...

    LEXEO Therapeutics

    Business Area(s): 
  • Gene Therapy
    • Description: 

    LEXEO Therapeutics is a New York City based clinical stage ...

    Locana

    Business Area(s): 
  • Gene Therapy
    • Description: 

    Locana is creating RNA-targeting gene therapies to treat devastating ...

    Locana Bio

    Business Area(s): 
  • RNA
  • Gene Therapy
    • Description: 

    Develops an RNA-targeting gene therapy platform.

    LogicBio

    Description: 

    LogicBio, launched in 2016, is building on the pioneering work ...

    Lysogene

    Business Area(s): 
  • Gene Therapy
    • Description: 
    AAV gene therapy technology

    MeiraGTx

    Business Area(s): 
  • Gene Therapy
  • Small Molecules
    • Description: 

    MeiraGTx is developing a potentially transformative technology to enable the ...

    Mekonos

    Description: 

    Developing industrial scale genome editing for therapeutics and therefore making ...

    Metagenomi

    Business Area(s): 
  • Gene Editing
  • Gene Therapy
    • Description: 

    Working to discover new gene editing systems for gene therapy

    Milo Biotechnology

    Business Area(s): 
  • Gene Therapy
    • Description: 
    Gene Therapy

    MultiVir

    Business Area(s): 
  • Gene Therapy
    • Description: 
    Gene therapy cancer treatment

    Myonexus Therapeutics

    Business Area(s): 
  • Gene Therapy
  • Muscle
    • Description: 

    Myonexus Therapeutics is a clinical-stage gene therapy company developing ...

    Neurogene

    Business Area(s): 
  • Gene Therapy
  • Rare Diseases
    • Description: 

    Neurogene is focused on developing gene therapies for rare, devastating ...

    Nightstar Therapeutics

    Business Area(s): 
  • Gene Therapy
  • Ophthalmology
    • Description: 

    NIghtstar is a leading clinical-stage gene therapy company focused on ...

    NightstaRx

    Business Area(s): 
  • Gene Therapy
    • Description: 
    Gene therapies for retinal dystrophies

    Novahep

    Business Area(s): 
  • Gene Therapy
    • Description: 
    Gene Therapy

    Novici Biotech

    Description: 

    Gene shuffling technology

    Nucelis

    Business Area(s): 
  • Gene Therapy
    • Description: 
    Site-Specific Gene Modification System

    Obsidian Therapeutics

    Business Area(s): 
  • Gene Therapy
  • Small Molecules
    • Description: 

    Developing controllable cell and gene therapies. Obsidian’s proprietary cytoDRiVE™ platform ...

    Orbit Biomedica (Gyroscope Therapeutics)

    Business Area(s): 
  • Gene Therapy
  • Vision
    • Description: 

    Orbit’s sub-retinal delivery system is designed to safely, precisely ...

    Orchard Therapeutics

    Business Area(s): 
  • Gene Therapy
    • Description: 

    Orchard Therapeutics is a biotechnology company incorporated in September 2015 ...

    Oxford BioMedica

    Business Area(s): 
  • Gene Therapy
  • Cell Therapies
    • Description: 

    Oxford BioMedica uses their LentiVector® delivery platform to develop gene ...

    Passage Bio

    Business Area(s): 
  • Gene Therapy
    • Description: 

    Gene therapy platform developing therapies for CNS-targeted rare diseases

    Poseida Therapeutics

    Description: 

    Poseida Therapeutics is a spin out of Transposagen Biopharmaceuticals that ...

    Precigen

    Description: 

    Precigen (fomerly Intrexon, Nasdaq: PGEN) is a dedicated discovery and ...

    Prevail Therapeutics

    Description: 

    Prevail Therapeutics is developing disease-modifying gene therapies for patients with ...

    PTC Therapeutics

    Description: 

    PTC is a biopharmaceutical company focused on the discovery and ...

    Puresyn

    Business Area(s): 
  • Gene Therapy
    • Description: 
    Plasmid and Adenovirus Purification

    Redpin Therapeutics

    Description: 

    Redpin Therapeutics is a privately held, preclinical stage gene therapy ...

    ReGenX Biosciences

    Business Area(s): 
  • Gene Therapy
    • Description: 
    Gene Therapy, Biologics

    Regenxbio

    Business Area(s): 
  • Gene Therapy
    • Description: 

    REGENXBIO is seeking to improve lives through the curative potential ...

    Rejuvenate Bio

    Business Area(s): 
  • Gene Therapy
  • Pets
    • Description: 

    The company is working on gene therapy for mitral valve ...

    Renova Therapeutics

    Business Area(s): 
  • Gene Therapy
    • Description: 
    Gene Therapy

    Renovacor

    Business Area(s): 
  • Gene Therapy
  • Cardiovascular
    • Description: 

    Renovacor, headquartered in Philadelphia, is a preclinical‑stage biopharmaceutical company focused ...

    RetroSense Therapeutics

    Business Area(s): 
  • Gene Therapy
    • Description: 
    Gene Therapy

    Rocket Pharmaceuticals

    Business Area(s): 
  • Gene Therapy
  • Stem Cells
    • Description: 

    Rocket's lentiviral-based programs aim to enable transduction of patients’ stem ...

    Sangamo Therapeutics

    Description: 

    Sangamo was founded in 1995 as Sangamo BioSciences, Inc. in ...

    Sarepta Therapeutics

    Business Area(s): 
  • Biologics
  • Gene Therapy
    • Description: 

    Sarepta Therapeutics is a biopharmaceutical company focused on developing innovative ...

    Scout Bio

    Description: 

    Scout Bio is working to harness gene therapy to treat ...

    Scribe Therapeutics

    Business Area(s): 
  • CRISPR
  • Gene Therapy
    • Description: 

    Scribe is building and applying a suite of CRISPR technologies ...

    Shape Therapeutics

    Business Area(s): 
  • Gene Therapy
  • Drug Delivery
    • Description: 

    ShapeTX is developing Gene Therapies using proprietary RNA technologies.

    Sixfold Bioscience

    Description: 

    Develop safe and effective drug delivery systems for cell and ...

    SmartPharm Therapeutics

    Business Area(s): 
  • Gene Therapy
  • COVID-19
  • Vaccines
    • Description: 

    SmartPharm Therapeutics uses gene-encoded therapeutics for a non-viral form of ...

    SparingVision

    Business Area(s): 
  • Gene Therapy
  • Vision
    • Description: 

    SparingVision is a biotechnology company focused on the discovery and ...

    Spark Therapeutics

    Business Area(s): 
  • Gene Therapy
    • Description: 

    Spark Therapeutics makes Luxturna, a form of gene therapy that ...

    Specific Biologics

    Business Area(s): 
  • Gene Therapy
    • Description: 

    Specific Biologics Inc. (SBI) is developing gene editing technologies to ...

    StrideBio

    Business Area(s): 
  • Gene Therapy
    • Description: 

    StrideBio, Inc. is a gene therapy company focused on creating ...

    SwanBio Therapeutics

    Business Area(s): 
  • Gene Therapy
    • Description: 

    SwanBio Therapeutics is developing therapies for the treatment of neurological ...

    Takara Bio

    Description: 

    Gene Therapy, Agribiotech, Biotech Lab Technology

    Taysha Gene Therapies

    Business Area(s): 
  • Gene Therapy
  • Neurobiology
    • Description: 

    At Taysha Gene Therapies, we’re on a mission to eradicate ...

    Tenaya Therapeutics

    Description: 

    Tenaya Therapeutics is a company with a mission to ...

    Tessera Therapeutics

    Business Area(s): 
  • Gene Therapy
    • Description: 

    Tessera Therapeutics is pioneering Gene Writing™—a new genome engineering technology ...

    TrakCel

    Business Area(s): 
  • Gene Therapy
  • Cell Therapies
    • Description: 
    Gene & Cell Therapy Services

    Transgene

    Business Area(s): 
  • Gene Therapy
    • Description: 
    Viral Vector Therapeutics, Gene Therapy

    UCB

    Description: 

    Multinational pharmaceutical company. Disease areas include neurology and immunology.

    Ultragenyx

    Description: 

    The company is a biopharmaceutical company focused on the development ...

    uniQure

    Business Area(s): 
  • Gene Therapy
  • Rare Diseases
    • Description: 

    The company is working on gene therapy cures for hemophilia ...

    Universal Cells (Astellas)

    Business Area(s): 
  • Stem Cells
  • Gene Therapy
    • Description: 

    Uses recombinant Adeno-associated virus to engineer and edit genomes and ...

    Vectalys

    Business Area(s): 
  • Gene Therapy
    • Description: 
    Gene Delivery, Services

    Vigene

    Description: 

    The company specializes in viral vector-based gene delivery. The company ...

    Vivet Therapeutics

    Business Area(s): 
  • Gene Therapy
    • Description: 

    Vivet Therapeutics is focused on optimizing gene therapy through a ...

    Voyager Therapeutics

    Business Area(s): 
  • Neurobiology
  • Gene Therapy
    • Description: 

    Voyager’s mission is to develop and deliver life-changing therapies to ...

    XyloCor Therapeutics

    Business Area(s): 
  • Gene Therapy
  • Cardiovascular
    • Description: 

    XyloCor Therapeutics is focused on improving the lives of people ...

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