 |
Top 10 Gene Therapy startups
Updated: April 19, 2024 |
Gene therapy is the therapeutic delivery of nucleic acid polymers into a patient's cells as a drug to treat disease. Gene editing has been a potential therapy for many genetic, autoimmune diseases and also for immunotherapy.
1
Country: USA | Funding: $3.4B
Bluebird Bio offers products based on the transformative potential of gene therapy for patients with genetic and orphan diseases.
Bluebird Bio offers products based on the transformative potential of gene therapy for patients with genetic and orphan diseases.
2
Country: USA | Funding: $1.5B
Intellia is a leading genome editing company focused on the development of proprietary, potentially curative therapeutics utilizing a recently developed biological tool known as the CRISPR/Cas9 system. We believe that the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing diseased genes in the human body through a single treatment course. We intend to leverage our leading scientific expertise, clinical development experience and intellectual property position to unlock broad therapeutic applications of CRISPR/Cas9 genome editing and develop a potential new drug class.
Intellia is a leading genome editing company focused on the development of proprietary, potentially curative therapeutics utilizing a recently developed biological tool known as the CRISPR/Cas9 system. We believe that the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing diseased genes in the human body through a single treatment course. We intend to leverage our leading scientific expertise, clinical development experience and intellectual property position to unlock broad therapeutic applications of CRISPR/Cas9 genome editing and develop a potential new drug class.
3
Country: USA | Funding: $1.2B
ElevateBio is a biotechnology company that specializes in cell and gene-based therapies.
ElevateBio is a biotechnology company that specializes in cell and gene-based therapies.
4
Country: USA | Funding: $931.6M
Editas Medicine's mission is to translate its genome editing technology into a novel class of human therapeutics that enable precise and corrective molecular modification to treat the underlying cause of a broad range of diseases at the genetic level.
Editas Medicine's mission is to translate its genome editing technology into a novel class of human therapeutics that enable precise and corrective molecular modification to treat the underlying cause of a broad range of diseases at the genetic level.
5
Country: UK | Funding: $781.6M
Autolus utilises advanced cell programming CAR-T and manufacturing technologies and we have established a development-stage pipeline of products for the treatment of haematological malignancies and solid tumours.
Autolus utilises advanced cell programming CAR-T and manufacturing technologies and we have established a development-stage pipeline of products for the treatment of haematological malignancies and solid tumours.
6
Country: USA | Funding: $763.8M
Spark is a leader in the field of gene therapy, seeking to transform the lives of patients by developing potential one-time, life-altering treatments for debilitating genetic diseases. We initially are applying our integrated platform to treat rare diseases where no, or only palliative, therapies exist.
Spark is a leader in the field of gene therapy, seeking to transform the lives of patients by developing potential one-time, life-altering treatments for debilitating genetic diseases. We initially are applying our integrated platform to treat rare diseases where no, or only palliative, therapies exist.
7
Country: USA | Funding: $700M
Sana Biotechnology is a developer of engineered cells intended to be used as medicine for patients. It's deploying a platform that can repair and control genes in cells or replace any cell in the body.
Sana Biotechnology is a developer of engineered cells intended to be used as medicine for patients. It's deploying a platform that can repair and control genes in cells or replace any cell in the body.
8
Country: USA | Funding: $692.3M
Kriya Therapeutics is a next-generation gene therapy company focused on designing and developing transformative new treatments.
Kriya Therapeutics is a next-generation gene therapy company focused on designing and developing transformative new treatments.
9
Country: USA | Funding: $546.1M
Sangamo is a clinical stage biopharmaceutical company focused on the research, development and commercialization of engineered zinc finger DNA-binding proteins (ZFPs) as novel ZFP Therapeutics targeting various monogenic and infectious diseases with unmet medical needs.
Sangamo is a clinical stage biopharmaceutical company focused on the research, development and commercialization of engineered zinc finger DNA-binding proteins (ZFPs) as novel ZFP Therapeutics targeting various monogenic and infectious diseases with unmet medical needs.
10
Country: USA | Funding: $536.4M
Generation Bio is a biotechnology company developing a breakthrough class of genetic medicines to enable a new generation of people unaffected by inherited disease. The company’s therapies are based on its proprietary GeneWave technology, which delivers durable, high levels of gene expression and can be re-dosed to titrate to effect and to sustain impact over a lifetime.
Generation Bio is a biotechnology company developing a breakthrough class of genetic medicines to enable a new generation of people unaffected by inherited disease. The company’s therapies are based on its proprietary GeneWave technology, which delivers durable, high levels of gene expression and can be re-dosed to titrate to effect and to sustain impact over a lifetime.
11
Country: USA | Funding: $531.8M
Tessera Therapeutics is pioneering Gene Writing - a new genome engineering technology that writes therapeutic messages into the genome to treat diseases at their source.
Tessera Therapeutics is pioneering Gene Writing - a new genome engineering technology that writes therapeutic messages into the genome to treat diseases at their source.
12
Country: USA | Funding: $460.5M
Poseida Therapeutics utilizes best-in-class genome engineering capabilities to develop targeted lifesaving therapeutics.
Poseida Therapeutics utilizes best-in-class genome engineering capabilities to develop targeted lifesaving therapeutics.
13
Country: USA | Funding: $459.7M
Synthego is a provider of synthetic RNA solutions for CRISPR Genome Engineering.
Synthego is a provider of synthetic RNA solutions for CRISPR Genome Engineering.
14
Country: USA | Funding: $457M
Metagenomi is using the power of metagenomics and machine learning to discover novel genome editing systems.
Metagenomi is using the power of metagenomics and machine learning to discover novel genome editing systems.
15
Country: USA | Funding: $428M
Taysha Gene Therapies is a developer of treatments to eradicate severe & life-threatening monogenic diseases of the central nervous system.
Taysha Gene Therapies is a developer of treatments to eradicate severe & life-threatening monogenic diseases of the central nervous system.
16
Country: USA | Funding: $419.3M
Verve is focused on discovering and developing therapies that safely edit the genomes of adults to confer protection against coronary.
Verve is focused on discovering and developing therapies that safely edit the genomes of adults to confer protection against coronary.
17
Country: USA | Funding: $407M
The mission of CRISPR Therapeutics is to develop transformative gene-based medicines for patients with serious diseases. Our therapeutic approach is to cure diseases at the molecular level using the breakthrough gene editing technology called CRISPR-Cas9. With our multi-disciplinary team of world-renowned academics, drug developers and clinicians, we are uniquely positioned to translate CRISPR-Cas9 technology into human therapeutics.
The mission of CRISPR Therapeutics is to develop transformative gene-based medicines for patients with serious diseases. Our therapeutic approach is to cure diseases at the molecular level using the breakthrough gene editing technology called CRISPR-Cas9. With our multi-disciplinary team of world-renowned academics, drug developers and clinicians, we are uniquely positioned to translate CRISPR-Cas9 technology into human therapeutics.
18
Country: USA | Funding: $381M
Maze Therapeutics is a operator of a biotechnology firm intended to focused on translating genetic insights into new medicines.
Maze Therapeutics is a operator of a biotechnology firm intended to focused on translating genetic insights into new medicines.
19
Country: USA | Funding: $379.5M
Passage Bio is a fully integrated gene therapy company with a mission to develop a portfolio of five life-transforming AAV-delivered therapeutics for the treatment of rare monogenic central nervous system diseases.
Passage Bio is a fully integrated gene therapy company with a mission to develop a portfolio of five life-transforming AAV-delivered therapeutics for the treatment of rare monogenic central nervous system diseases.
20
Country: USA | Funding: $374M
Voyager Therapeutics is a clinical stage gene therapy company developing life-changing treatments for severe diseases of the central nervous system (CNS). Gene therapy has the potential to transform the treatment of CNS and other diseases by providing a one-time therapy that dramatically improves patients' lives.
Voyager Therapeutics is a clinical stage gene therapy company developing life-changing treatments for severe diseases of the central nervous system (CNS). Gene therapy has the potential to transform the treatment of CNS and other diseases by providing a one-time therapy that dramatically improves patients' lives.
21
Country: USA | Funding: $345.4M
CytomX is unlocking the potential of antibody therapeutics in oncology by developing a novel therapeutic antibody class of highly targeted Probody therapeutics.
CytomX is unlocking the potential of antibody therapeutics in oncology by developing a novel therapeutic antibody class of highly targeted Probody therapeutics.
22
Country: USA | Funding: $317.7M
Caribou Biosciences is a pioneer in the revolutionary field of CRISPR-Cas genome editing. Our proprietary technology puts us at the forefront of the development of new medical therapies and bio-based products which offer profound benefits to both human health and society as a whole.
Caribou Biosciences is a pioneer in the revolutionary field of CRISPR-Cas genome editing. Our proprietary technology puts us at the forefront of the development of new medical therapies and bio-based products which offer profound benefits to both human health and society as a whole.
23
Country: USA | Funding: $305.8M
ArsenalBio is focused on integrating technologies such as CRISPR-based genome engineering, scaled and high throughput target identification, synthetic biology, and machine learning to advance a new paradigm to discover and develop in immune cell therapies.
ArsenalBio is focused on integrating technologies such as CRISPR-based genome engineering, scaled and high throughput target identification, synthetic biology, and machine learning to advance a new paradigm to discover and develop in immune cell therapies.
24
Country: UK | Funding: $302.1M
Freeline Therapeutics is a biopharmaceutical company specialising in developing liver directed gene therapies for bleeding disorders.
Freeline Therapeutics is a biopharmaceutical company specialising in developing liver directed gene therapies for bleeding disorders.
25
Country: UK | Funding: $269.5M
Gyroscope is an ophthalmology company developing gene therapies for the treatment of eye diseases linked to an unbalanced complement system
Gyroscope is an ophthalmology company developing gene therapies for the treatment of eye diseases linked to an unbalanced complement system
26
Country: USA | Funding: $241.8M
Askbio develops meds for range of genetic diseases targeting central nervous system, muscle, respiratory, and heart tissues.
Askbio develops meds for range of genetic diseases targeting central nervous system, muscle, respiratory, and heart tissues.
27
Country: Canada | Funding: $236.7M
Deep Genomics brings together world-leading expertise in machine learning and genome biology. We’re inventing a new generation of computational technologies that predict what will happen within a cell when DNA is altered by genetic variation, whether natural or therapeutic. Our systems predict the molecular effects of genetic variation, opening a new and exciting path to discovery for disease diagnostics and therapies.
Deep Genomics brings together world-leading expertise in machine learning and genome biology. We’re inventing a new generation of computational technologies that predict what will happen within a cell when DNA is altered by genetic variation, whether natural or therapeutic. Our systems predict the molecular effects of genetic variation, opening a new and exciting path to discovery for disease diagnostics and therapies.
28
Country: USA | Funding: $225.2M
Precision BioSciences is a genome editing company dedicated to improving life. Our team seeks to solve – not just treat, but solve – significant problems in oncology, genetic disease, agriculture, and beyond via its proprietary ARCUS genome editing platform.
Precision BioSciences is a genome editing company dedicated to improving life. Our team seeks to solve – not just treat, but solve – significant problems in oncology, genetic disease, agriculture, and beyond via its proprietary ARCUS genome editing platform.
29
Country: USA | Funding: $210M
Cabaletta Bio has adapted clinically-validated and FDA-approved CAR T cell technology to target B cell-mediated autoimmune diseases.
Cabaletta Bio has adapted clinically-validated and FDA-approved CAR T cell technology to target B cell-mediated autoimmune diseases.
30
Country: USA | Funding: $207.5M
Korro Bio provides an efficient and selective method for RNA editing, leveraging natural processes common to all multi-cellular organisms.
Korro Bio provides an efficient and selective method for RNA editing, leveraging natural processes common to all multi-cellular organisms.
31
Country: China | Funding: $190M
JW is focused on revolutionizing medicine by re-engaging the body's immune system to treat cancer.
JW is focused on revolutionizing medicine by re-engaging the body's immune system to treat cancer.
32
Country: USA | Funding: $187M
Homology Medicines is a genetic medicines company translating proprietary, cutting-edge gene editing and gene therapy technology into cures for patients with rare diseases. It is taking a slightly different tack from other players in the gene-editing domain with its adeno-associated virus (AAV) technology AMEnDR. It’s a different approach to other techniques such as CRISPR/cas9 or zinc finger nucleases
Homology Medicines is a genetic medicines company translating proprietary, cutting-edge gene editing and gene therapy technology into cures for patients with rare diseases. It is taking a slightly different tack from other players in the gene-editing domain with its adeno-associated virus (AAV) technology AMEnDR. It’s a different approach to other techniques such as CRISPR/cas9 or zinc finger nucleases
33
Country: USA | Funding: $185M
LEXEO Therapeutics develops gene therapy for the world's most devastating diseases
LEXEO Therapeutics develops gene therapy for the world's most devastating diseases
34
Country: USA | Funding: $183.5M
Neurogene develops gene therapies for inherited neurological diseases, initially focusing on a form of Batten disease.
Neurogene develops gene therapies for inherited neurological diseases, initially focusing on a form of Batten disease.
35
Country: China | Funding: $179.7M
EdiGene is a biotechnology company that develops genome editing technologies.
EdiGene is a biotechnology company that develops genome editing technologies.
36
Country: Netherlands | Funding: $167M
uniQure is delivering on the promise of gene therapy, single treatments with potentially curative results. We have developed a modular platform to rapidly bring new disease modifying therapies to patients with severe disorders. We believe our multiple partnerships and the regulatory approval of our lead product Glybera in the European Union for a subset of patients with LPLD provides validation for our approach.
uniQure is delivering on the promise of gene therapy, single treatments with potentially curative results. We have developed a modular platform to rapidly bring new disease modifying therapies to patients with severe disorders. We believe our multiple partnerships and the regulatory approval of our lead product Glybera in the European Union for a subset of patients with LPLD provides validation for our approach.
37
Country: USA | Funding: $165M
Obsidian Therapeutics is a developer of a next-generation cell and gene therapies created to extend adoptive immunotherapy.
Obsidian Therapeutics is a developer of a next-generation cell and gene therapies created to extend adoptive immunotherapy.
38
Country: USA | Funding: $162.5M
Akouos is a new biotechnology company focused on restoring and preserving hearing.
Akouos is a new biotechnology company focused on restoring and preserving hearing.
39
Country: USA | Funding: $139.5M
Gemini Therapeutics is a precision medicine company focused on genetically-defined dry age-related macular degeneration (AMD) and associated rare genetic diseases.Their therapeutic candidates are matched to molecular abnormalities found in patients with high clinical need and our broad multimodal pipeline includes monoclonal antibodies, recombinant proteins and gene therapies.
Gemini Therapeutics is a precision medicine company focused on genetically-defined dry age-related macular degeneration (AMD) and associated rare genetic diseases.Their therapeutic candidates are matched to molecular abnormalities found in patients with high clinical need and our broad multimodal pipeline includes monoclonal antibodies, recombinant proteins and gene therapies.
40
Country: USA | Funding: $140M
Capsida develops targeted gene therapies in serious neurodegenerative diseases.
Capsida develops targeted gene therapies in serious neurodegenerative diseases.
41
Country: USA | Funding: $135M
SalioGen advances in genetic therapies using its Exact DNA Integration Technology platform, a mammal-derived genome engineering technology.
SalioGen advances in genetic therapies using its Exact DNA Integration Technology platform, a mammal-derived genome engineering technology.
42
Country: UK | Funding: $132.5M
PsiOxus aims to be the world’s leading cancer gene therapy company, delivering medicines of value to patients with cancer.
PsiOxus aims to be the world’s leading cancer gene therapy company, delivering medicines of value to patients with cancer.
43
Country: USA | Funding: $129M
Prevail Therapeutics is a biotechnology company focused on developing novel gene therapies for patients with Parkinson’s disease and other neurodegenerative diseases.
Prevail Therapeutics is a biotechnology company focused on developing novel gene therapies for patients with Parkinson’s disease and other neurodegenerative diseases.
44
Country: France | Funding: $127.6M
SparingVision is a biotechnology company focused on the discovering and development of an innovative, therapeutic approach for treatment of blinding inherited retinal diseases such as retinitis pigmentosa.
SparingVision is a biotechnology company focused on the discovering and development of an innovative, therapeutic approach for treatment of blinding inherited retinal diseases such as retinitis pigmentosa.
45
Country: USA | Funding: $124.5M
Dimension Therapeutics is developing novel, liver-directed gene therapy treatments for severe, rare genetic disorders. Dimension’s lead programs address significant medical needs for new therapies and include a wholly owned program in hemophilia B.
Dimension Therapeutics is developing novel, liver-directed gene therapy treatments for severe, rare genetic disorders. Dimension’s lead programs address significant medical needs for new therapies and include a wholly owned program in hemophilia B.
46
Country: USA | Funding: $112.7M
Meira Gene Therapy is argeting inherited retinal diseases with therapies designed to halt vision loss.
Meira Gene Therapy is argeting inherited retinal diseases with therapies designed to halt vision loss.
47
Country: USA | Funding: $111M
Foghorn Therapeutics is developing therapies based on a system that directs which genes our cells express, and when, where, and in what order.
Foghorn Therapeutics is developing therapies based on a system that directs which genes our cells express, and when, where, and in what order.
48
Country: USA | Funding: $109.9M
Mirna is a clinical-stage biopharmaceutical company developing a broad pipeline of leading microRNA-based oncology therapeutics, the first to demonstrate clinical proof of concept for microRNA replacement therapy.
Mirna is a clinical-stage biopharmaceutical company developing a broad pipeline of leading microRNA-based oncology therapeutics, the first to demonstrate clinical proof of concept for microRNA replacement therapy.
49
Country: USA | Funding: $109M
Dyno Therapeutics is a biotechnology company that uses artificial intelligence (AI) to gene therapy.
Dyno Therapeutics is a biotechnology company that uses artificial intelligence (AI) to gene therapy.
50
Country: USA | Funding: $101.5M
Edge Therapeutics is a clinical-stage biotechnology company that discovers, develops and seeks to commercialize novel, hospital-based therapies capable of transforming treatment paradigms in the management of acute, life-threatening neurological conditions. Our product candidates utilize our proprietary Precisa development platform that are based on specific physical and chemical properties that enable targeted delivery to the exact site of the injury, and provide a controlled and sustained drug release to the affected area.
Edge Therapeutics is a clinical-stage biotechnology company that discovers, develops and seeks to commercialize novel, hospital-based therapies capable of transforming treatment paradigms in the management of acute, life-threatening neurological conditions. Our product candidates utilize our proprietary Precisa development platform that are based on specific physical and chemical properties that enable targeted delivery to the exact site of the injury, and provide a controlled and sustained drug release to the affected area.