Gene Therapy Companies and Organizations

 

75 Organization(s)

4D Molecular Therapeutics

Business Area(s): 
Description: 
Gene Therapy

Adverum Biotechnologies

Business Area(s): 
Description: 

Adverum is a clinical-stage gene therapy company targeting unmet medical needs in serious rare and ocular diseases. Through directed evolution, we generate a diverse library of millions of AAV ...

AGTC

Business Area(s): 
Description: 
Gene Therapy

Akouos

Business Area(s): 
Description: 

Akouos is a precision genetic medicine company focused on developing gene therapies that restore and preserve hearing.

American Gene Technologies

Business Area(s): 
Description: 

American Gene Technolgies is devleoping therapeutics based on lentiviral vectors to target HIV, PKU, Liver cancer, Parkinson's disease, and familial dysautonomia.

AmpliPhi Biosciences

Business Area(s): 
Description: 

Viral Vector Therapeutics, Gene Therapy

AnGes

Business Area(s): 
Description: 

Gene therapy

Angionetics

Business Area(s): 
Description: 

Angionetics Inc., is a company focused on the late-stage clinical development and commercialization of Generx®, an angiogenic gene therapy product candidate designed for medical revascularization ...

Annapurna

Business Area(s): 
Description: 
Gene Therapy for Friedrich's ataxia

Applied Viromics

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

Audentes Therapeutics

Business Area(s): 
Description: 
AAV Gene Therapy Treatments

Aum LifeTech

Business Area(s): 
Description: 
Gene silencing technology

Avalanche Biotechnologies

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

AveXis (Novartis)

Business Area(s): 
Description: 

Gene therapies for rare and orphan neurological genetic diseases

AVROBIO

Business Area(s): 
Description: 

AVROBIO, Inc., develops lentiviral-based gene therapies

BEAT BioTherapeutics

Business Area(s): 
Description: 

BEAT Biotherapeutics Corp. (BEATBio) is developing BB-R12, a novel gene therapy with the potential to revolutionize the treatment of heart failure.

Bellicum Pharmaceuticals

Business Area(s): 
Description: 

Develops next-generation product candidates in some of the most important areas of cellular immunotherapy, including hematopoietic stem cell transplantation (HSCT), CAR T cell therapy, and TCR ...

Bristol-Myers Squibb

Business Area(s): 
Description: 

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

CALiMMUNE

Business Area(s): 
Description: 
Gene Therapy

Cellectis

Business Area(s): 
Description: 

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

Cellomics

Business Area(s): 
Description: 
Lentiviral vectors

Coda Biotherapeutics

Business Area(s): 
Description: 

CODA Biotherapeutics’ chemogenetic platform aims to control the activity of cells to treat disease. With chemogenetics, the goal is to modify a target cell population using gene therapy to express ...

DalCor Pharmaceuticals

Business Area(s): 
Description: 

Genetically targeted medicine

Dimension Therapeutics

Business Area(s): 
Description: 
Gene Therapy

Discovery Genomics (Immusoft)

Business Area(s): 
Description: 
Gene Therapy

DNAlite Therapeutics

Business Area(s): 
Description: 

DNAlite Therapeutics, Inc. is an early-stage preclinical biotechnology company developing drugs for organs protected by mucus barriers.

DNAVEC

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

Errant Gene Therapeutics

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

Fibrocell Science

Business Area(s): 
Description: 

Fibrocell, a cell and gene therapy company, is focused on diseases affecting the skin and connective tissue.  By extracting fibroblast cells from a patient’s own skin, we create localized gene ...

FIT Biotech

Business Area(s): 
Description: 
Gene Therapy

Freeline Therapeutics

Business Area(s): 
Description: 

Freeline is a biopharmaceutical company focused on the development and commercialization of gene therapies for bleeding and other debilitating disorders based on a next-generation AAV gene therapy ...

GeneCure Biotechnologies

Business Area(s): 
Description: 
Gene Transfer Technology

Generation Bio

Business Area(s): 
Description: 

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

Genethon

Business Area(s): 
Description: 
Gene Therapy

GenSight Biologics

Business Area(s): 
Description: 
Ophthalmic Gene Therapy

GenVec

Business Area(s): 
Description: 
Gene Therapy

Global BioTherapeutics

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

HORAMA

Business Area(s): 
Description: 
HORAMA is developing innovative gene therapy medicinal products in the field of rare ophthalmological diseases to preserve visual autonomy of the affected patients. 

iCell Gene Therapeutics

Business Area(s): 
Description: 

Gene Therapy for Cancer

Immusoft

Business Area(s): 
Description: 

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

InvivoGen Therapeutics

Business Area(s): 
Description: 
Gene and Immunotherapies

Krystal Biotech

Business Area(s): 
Description: 

Krystal Biotech, Inc. is using gene therapy to develop effective and novel treatments for skin diseases. 

LogicBio

Business Area(s): 
Description: 

LogicBio, launched in 2016, is building on the pioneering work of gene therapy leaders from Stanford University.

The company's technology, GeneRide™, enables highly specific ...

Lysogene

Business Area(s): 
Description: 
AAV gene therapy technology

Medgenics

Business Area(s): 
Description: 

Gene Therapy or 'genomic medicine'

MeiraGTx

Business Area(s): 
Description: 

MeiraGTx is developing a potentially transformative technology to enable the use of small molecules to turn gene therapy product candidates on and off. 

Milo Biotechnology

Business Area(s): 
Description: 
Gene Therapy

MultiVir

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

Myonexus Therapeutics

Business Area(s): 
Description: 

Myonexus Therapeutics is a clinical-stage gene therapy company developing first-ever corrective gene therapies for Limb-Girdle muscular dystrophies, debilitating rare diseases ...

Nightstar Therapeutics

Business Area(s): 
Description: 

NIghtstar is a leading clinical-stage gene therapy company focused on developing and commercializing novel, one-time treatments for patients suffering from rare inherited retinal diseases that ...

NightstaRx

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

Novahep

Business Area(s): 
Description: 
Gene Therapy

Novici Biotech

Business Area(s): 
Description: 
Gene shuffling technology

Nucelis

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

Orbit Biomedica

Business Area(s): 
Description: 

 Orbit’s sub-retinal delivery system is designed to safely, precisely and consistently deliver gene and cell therapy to enable the treatment of retinal disease.

Orchard Therapeutics

Business Area(s): 
Description: 

Orchard Therapeutics is a biotechnology company incorporated in September 2015 and dedicated to being a leading global, fully integrated company delivering innovative gene therapies that transform ...

Oxford Biomedica

Business Area(s): 
Description: 
Gene-based Medicines

Poseida Therapeutics

Business Area(s): 
Description: 

Poseida Therapeutics is a spin out of Transposagen Biopharmaceuticals that utilizes best-in-class, proprietary genome editing technologies to develop targeted, life-saving therapeutics in areas of ...

Puresyn

Business Area(s): 
Description: 
Plasmid and Adenovirus Purification
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