1
Country: USA | Funding: $1.7B
Intellia is a leading genome editing company specializing in the development of proprietary, potentially curative therapeutics using CRISPR/Cas9 technology. Intellia's co-founder Jennifer Daudna, along with Emmanuelle Charpentier, were awarded the 2020 Nobel Prize in Chemistry for their pioneering work in CRISPR. The company's pipeline includes treatments for hereditary angioedema, transthyretin amyloidosis with polyneuropathy and cardiomyopathy, hemophilia, acute myeloid leukemia, and B-cell malignancies.
Intellia is a leading genome editing company specializing in the development of proprietary, potentially curative therapeutics using CRISPR/Cas9 technology. Intellia's co-founder Jennifer Daudna, along with Emmanuelle Charpentier, were awarded the 2020 Nobel Prize in Chemistry for their pioneering work in CRISPR. The company's pipeline includes treatments for hereditary angioedema, transthyretin amyloidosis with polyneuropathy and cardiomyopathy, hemophilia, acute myeloid leukemia, and B-cell malignancies.
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: $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.
4
Country: USA | Funding: $585.8M
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions.
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions.
5
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.
6
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.
7
Country: USA | Funding: $211.5M
Haplomics is a biotech company focused on the treatment and cure of hemophilia. Scientists at Haplomics are pursuing three promising initiatives to control and potentially eliminate this cruel disease: determining why 30% of hemophilia patients develop inhibitors to treatment; developing a new technique to overcome this allergic response; and repairing patients’ defective DNA, curing them for life.
Haplomics is a biotech company focused on the treatment and cure of hemophilia. Scientists at Haplomics are pursuing three promising initiatives to control and potentially eliminate this cruel disease: determining why 30% of hemophilia patients develop inhibitors to treatment; developing a new technique to overcome this allergic response; and repairing patients’ defective DNA, curing them for life.
8
Country: USA | Funding: $81.3M
Eagle Pharmaceuticals is a company focused on developing and commercializing injectable products in the critical care and oncology areas.
Eagle Pharmaceuticals is a company focused on developing and commercializing injectable products in the critical care and oncology areas.
