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Top 8 Hemophilia treatment startups
Oct 10, 2024 | By Jason Kwon | 25 |
These startups develop new diagnostics and treatment technologies for Hemophilia, like genome analyses, CRISPR-based gene therapy, recombinant clotting factors, new drugs...
1
Country: Netherlands | Funding: $780.7M
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.
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: $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.
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: $124.5M
Dimension Therapeutics is developing novel, liver-directed gene therapy treatments for severe, rare genetic disorders. Dimension’s lead programs address significant medical needs for new therapies and include a wholly owned program in hemophilia B.
Dimension Therapeutics is developing novel, liver-directed gene therapy treatments for severe, rare genetic disorders. Dimension’s lead programs address significant medical needs for new therapies and include a wholly owned program in hemophilia B.
8
Country: USA | Funding: $12.3M
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.
