1
Country: Italy | Funding: $206.3M
AAVantgarde Bio is a clinical-stage biotechnology company that develops retinal disease gene therapies. One of its drugs is intended at patients with Stargardt disease, a leading cause of vision loss in children and young adults. By delivering the full-length ABCA4 protein, AAVantgarde hopes that it can address the root cause of this eye condition. Another asset is for patients with retinitis pigmentosa associated with Usher syndrome - rare genetic disease that affects both hearing and vision. AAVantgarde says that its proprietary technology has demonstrated high transduction, protein expression and safety for both Stargardt and Usher 1B.
AAVantgarde Bio is a clinical-stage biotechnology company that develops retinal disease gene therapies. One of its drugs is intended at patients with Stargardt disease, a leading cause of vision loss in children and young adults. By delivering the full-length ABCA4 protein, AAVantgarde hopes that it can address the root cause of this eye condition. Another asset is for patients with retinitis pigmentosa associated with Usher syndrome - rare genetic disease that affects both hearing and vision. AAVantgarde says that its proprietary technology has demonstrated high transduction, protein expression and safety for both Stargardt and Usher 1B.
2
Country: Netherlands | Funding: $1.3B
uniQure has developed an industry-leading AAV gene therapy platform focused on liver and central nervous system diseases. The company's lead candidate, AMT-130, for the treatment of Huntington's disease consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene. The therapeutic goal is to inhibit the production of the mutant protein. The company is also developing candidate AMT-191 for Fabry disease (it delivers a GLA transgene to the liver to enhance GLA protein production), AMT-162 for the treatment of amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene (it is a recombinant AAVrh10 vector expressing microRNA designed to suppress SOD1 expression) and HEMGENIX - the world's first FDA-approved gene therapy for hemophilia B.
uniQure has developed an industry-leading AAV gene therapy platform focused on liver and central nervous system diseases. The company's lead candidate, AMT-130, for the treatment of Huntington's disease consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene. The therapeutic goal is to inhibit the production of the mutant protein. The company is also developing candidate AMT-191 for Fabry disease (it delivers a GLA transgene to the liver to enhance GLA protein production), AMT-162 for the treatment of amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene (it is a recombinant AAVrh10 vector expressing microRNA designed to suppress SOD1 expression) and HEMGENIX - the world's first FDA-approved gene therapy for hemophilia B.
3
Country: USA | Funding: $3.4B
Genetix Biotherapeutics (formerly Bluebird Bio) develops gene therapies for rare genetic diseases using patient's own blood stem cells. The cells are harvested, modified outside the body to add working copies of the affected gene and then reintroduced into the patient, restoring the function of the affected gene. The company specializes in the treatment of sickle cell anemia, beta thalassemia and cerebral adrenoleukodystrophy (CALD). Its unique, single-dose treatments are FDA approved and address the underlying cause of the disease and can provide significant, long-term benefits to patients. Acquired by The Carlyle Group
Genetix Biotherapeutics (formerly Bluebird Bio) develops gene therapies for rare genetic diseases using patient's own blood stem cells. The cells are harvested, modified outside the body to add working copies of the affected gene and then reintroduced into the patient, restoring the function of the affected gene. The company specializes in the treatment of sickle cell anemia, beta thalassemia and cerebral adrenoleukodystrophy (CALD). Its unique, single-dose treatments are FDA approved and address the underlying cause of the disease and can provide significant, long-term benefits to patients. Acquired by The Carlyle Group
4
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.
5
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.
6
Country: USA | Funding: $951.3M
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.
7
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.
8
Country: USA | Funding: $913M
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: $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.
10
Country: USA | Funding: $743M
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.
11
Country: USA | Funding: $646M
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.
12
Country: USA | Funding: $630.8M
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.
13
Country: USA | Funding: $595M
4D Molecular Therapeutics is unlocking the full potential of the gene therapy field. Our customized AAV vectors can deliver genes to any cell in the body to eradicate disease.
4D Molecular Therapeutics is unlocking the full potential of the gene therapy field. Our customized AAV vectors can deliver genes to any cell in the body to eradicate disease.
14
Country: USA | Funding: $581.8M
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.
15
Country: USA | Funding: $569.1M
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.
16
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.
17
Country: USA | Funding: $460.5M
Poseida Therapeutics develops differentiated allogeneic cell therapies and genetic medicines using our proprietary genetic engineering platforms. The company develops the piggyBac non-viral DNA delivery system (which enables introduction of large genetic data into the genome) and the Cas-CLOVER site-specific gene editing system (which can be used for precise site-specific deletions, insertions and knockouts in various cell types). Using this platform, the company creates CAR-T therapies with high levels of desired TSCM (stem cell-derived memory T cells) for the treatment of a range of oncological, autoimmune and rare diseases, including multiple myeloma, acute myeloid leukemia and prostate cancer. Poseida was acquired by Roche in early 2025.
Poseida Therapeutics develops differentiated allogeneic cell therapies and genetic medicines using our proprietary genetic engineering platforms. The company develops the piggyBac non-viral DNA delivery system (which enables introduction of large genetic data into the genome) and the Cas-CLOVER site-specific gene editing system (which can be used for precise site-specific deletions, insertions and knockouts in various cell types). Using this platform, the company creates CAR-T therapies with high levels of desired TSCM (stem cell-derived memory T cells) for the treatment of a range of oncological, autoimmune and rare diseases, including multiple myeloma, acute myeloid leukemia and prostate cancer. Poseida was acquired by Roche in early 2025.
18
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.
19
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.
20
Country: USA | Funding: $445.4M
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.
21
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.
22
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.
23
Country: USA | Funding: $383.5M
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.
24
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.
25
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.
26
Country: UK | Funding: $365.1M
Beacon Therapeutics is an ophthalmic gene therapy company to treat blinding retinal illnesses and restore and enhance eyesight.
Beacon Therapeutics is an ophthalmic gene therapy company to treat blinding retinal illnesses and restore and enhance eyesight.
27
Country: USA | Funding: $360M
LEXEO Therapeutics develops gene therapy for the world's most devastating diseases
LEXEO Therapeutics develops gene therapy for the world's most devastating diseases
28
Country: USA | Funding: $325M
Obsidian Therapeutics develops precision cell and gene therapies to expand the capabilities of adoptive cancer immunotherapy. The company has developed the cytoDRiVE platform, which leverages drug-responsive domains (DRDs) to control protein function using an FDA-approved small molecule, and is continually expands its library of identified DRDs of varying sizes and purposes. The company's lead drug, OBX-115, is an experimental therapy using tumor-infiltrating lymphocytes genetically modified to produce a membrane-bound (non-secreted) cytokine (IL15). Obsidian is conducting a multicenter clinical trial for the treatment of advanced melanoma and non-small cell lung cancer.
Obsidian Therapeutics develops precision cell and gene therapies to expand the capabilities of adoptive cancer immunotherapy. The company has developed the cytoDRiVE platform, which leverages drug-responsive domains (DRDs) to control protein function using an FDA-approved small molecule, and is continually expands its library of identified DRDs of varying sizes and purposes. The company's lead drug, OBX-115, is an experimental therapy using tumor-infiltrating lymphocytes genetically modified to produce a membrane-bound (non-secreted) cytokine (IL15). Obsidian is conducting a multicenter clinical trial for the treatment of advanced melanoma and non-small cell lung cancer.
29
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.
30
Country: USA | Funding: $310M
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.
31
Country: USA | Funding: $304.5M
Arbor Biotechnologies is an operator of a bio-discovery company intended to provide human diagnostic development services.
Arbor Biotechnologies is an operator of a bio-discovery company intended to provide human diagnostic development services.
32
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.
33
Country: USA | Funding: $277.5M
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.
34
Country: USA | Funding: $265.2M
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.
35
Country: USA | Funding: $245M
AIRNA develops RNA editing technologies to create medicines for various diseases.
AIRNA develops RNA editing technologies to create medicines for various diseases.
36
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.
37
Country: USA | Funding: $237.6M
Atsena Therapeutics develops blindness gene therapy
Atsena Therapeutics develops blindness gene therapy
38
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.
39
Country: USA | Funding: $221M
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.
40
Country: USA | Funding: $215M
Tune Therapeutics develops epigenomic controls that dial gene expression up or down to potentially thwart cancers, genetic diseases and aging.
Tune Therapeutics develops epigenomic controls that dial gene expression up or down to potentially thwart cancers, genetic diseases and aging.
41
Country: Spain | Funding: $193.9M
Splice Bio is a biotech company developing gene therapies based on a platform that could bypass the cargo capacity of adeno-associated vectors (AAVs).
Splice Bio is a biotech company developing gene therapies based on a platform that could bypass the cargo capacity of adeno-associated vectors (AAVs).
42
Country: UK | Funding: $192.5M
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: 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.
44
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.
45
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.
46
Country: USA | Funding: $162.7M
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: UK | Funding: £125M
Purespring Therapeutics is an AAV gene therapy company focused on the kidney globally.
Purespring Therapeutics is an AAV gene therapy company focused on the kidney globally.
48
Country: USA | Funding: $140M
Capsida develops targeted gene therapies in serious neurodegenerative diseases.
Capsida develops targeted gene therapies in serious neurodegenerative diseases.
49
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.
50
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.
