1
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.
2
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.
3
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.
4
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.
5
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.
6
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.
7
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.
