Top 50 Gene Therapy startups in USA
Apr 08, 2026
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24
1
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
2
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
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
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.
5
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.
6
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.
7
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.
8
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.
9
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.
10
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.
11
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.
12
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.
13
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.
14
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.
15
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.
16
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.
17
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.
18
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.
19
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.
20
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.
21
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.
22
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.
23
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.
24
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
25
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.
26
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.
27
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.
28
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.
29
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.
30
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.
31
Funding: $245M
AIRNA develops RNA editing technologies to create medicines for various diseases.
AIRNA develops RNA editing technologies to create medicines for various diseases.
32
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.
34
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.
35
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.
36
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.
37
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.
38
Funding: $140M
Capsida develops targeted gene therapies in serious neurodegenerative diseases.
Capsida develops targeted gene therapies in serious neurodegenerative diseases.
39
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.
40
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.
41
Funding: $123M
Epic Bio's strength is a relentless focus on ushering in a new era of gene regulation.
Epic Bio's strength is a relentless focus on ushering in a new era of gene regulation.
42
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.
43
Funding: $107.1M
XyloCor Therapeutics is a bio-pharmaceutical company intended to develop gene therapy for people with advanced coronary artery disease.
XyloCor Therapeutics is a bio-pharmaceutical company intended to develop gene therapy for people with advanced coronary artery disease.
44
Funding: $95.5M
Genprex is a clinical stage gene therapy company developing molecular therapies to better target lung cancer cells. Their new approach to treating cancer is based upon our novel proprietary technology platform, including our initial product candidate, Oncoprex immunogene therapy, or Oncoprex.
Genprex is a clinical stage gene therapy company developing molecular therapies to better target lung cancer cells. Their new approach to treating cancer is based upon our novel proprietary technology platform, including our initial product candidate, Oncoprex immunogene therapy, or Oncoprex.
45
Funding: $90M
Ascidian Therapeutics is a biotechnology company intended for medical use in medical and clinical gene therapy.
Ascidian Therapeutics is a biotechnology company intended for medical use in medical and clinical gene therapy.
46
Funding: $85M
Code Biotherapeutics is a next-generation gene therapy company pioneering the development of targeted non-viral gene therapies.
Code Biotherapeutics is a next-generation gene therapy company pioneering the development of targeted non-viral gene therapies.
47
Funding: $75M
ProFound intends to reconsider what exactly defines a protein-coding gene and how a protein is coded in the genome.
ProFound intends to reconsider what exactly defines a protein-coding gene and how a protein is coded in the genome.
48
Funding: $38M
Carbon Biosciences is a biotech company that engages in the novel parvovirus gene therapy.
Carbon Biosciences is a biotech company that engages in the novel parvovirus gene therapy.
49
Funding: $30.2M
AGT uses a proprietary gene delivery platform to rapidly develop patented gene and cell therapies that cure infectious disease, monogenic disorders, and cancers by targeting their genetic root causes.
AGT uses a proprietary gene delivery platform to rapidly develop patented gene and cell therapies that cure infectious disease, monogenic disorders, and cancers by targeting their genetic root causes.
50
Funding: $28M
CellFE is a company developing a microfluidics based device to deliver gene-editing molecules.
CellFE is a company developing a microfluidics based device to deliver gene-editing molecules.
