Top 6 Hemophilia treatment startups in USA

Oct 10, 2024 | By Jason Kwon

1
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.
2
Funding: $585.8M
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions.
3
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.
4
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.
5
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.
6
Funding: $12.3M
Eagle Pharmaceuticals is a company focused on developing and commercializing injectable products in the critical care and oncology areas.
Editor: Jason Kwon
Jason Kwon is a senior editor for MedicalStartups. He has previously covered the pharmaceutical and medical research industries for FDAnews and worked as a head of marketing for medical startup Sonic Therapeutics. Before that, he co-founded a startup consulting business for emerging entrepreneurial hubs in Asia. Jason graduated from St. Bonaventure University’s journalism school. In his free time, Jason enjoys yoga, watching movie trailers, traveling to places where he can't get cell service. You can contact Jason at jaskwon(at)medicalstartups(dot)com