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: 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.
3
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.
4
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.
5
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.
6
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.
7
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.
8
Country: Netherlands | Funding: $855.7M
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.
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.
