 |
Top 10 Gene Therapy startups in USA
Mar 20, 2025 | By Jason Kwon | 24 |
1
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.
2
Funding: $543M
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.
3
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.
4
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
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
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.
7
Funding: $865M
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
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.
9
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.
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: $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.
13
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.
14
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.
15
Funding: $496M
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.
16
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.
17
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.
18
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.
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.
