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Initial CRISPR Gene Editing Therapy has been Approved. What is the Domestic Progress? Yefenghong/PharmaSources
December 14, 2023
Vertex Pharmaceuticals and CRISPR Therapeutics announced that their jointly developed CRISPR gene editing therapy CASGEVY has been conditionally approved.
FDA approves new treatment for adults with serious rare blood disease fda.drug
May 17, 2021
FDA has approved Empaveli (pegcetacoplan) injection to treat adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare, life-threatening blood disease. Empaveli is the first PNH treatment that binds to compliment protein C3.
BMS partners with Sensyne Health for rare blood disease research pharmatimes
October 26, 2020
Clinical AI company Sensyne Health has signed a research collaboration agreement with Bristol Myers Squibb to apply machine learning for rare blood disease research.
FDA Approves First Drug to Treat Group of Rare Blood Disorders americanpharmaceuticalreview
October 09, 2020
The U.S. Food and Drug Administration (FDA) has approved Nucala (mepolizumab) for adults and children aged 12 years and older with hypereosinophilic syndrome (HES) for six months or longer without another identifiable non-blood related cause of the ...
Pitt and Bayer to advance research for heart, lung and blood disease indications pharmaceutical-technology
January 13, 2017
The University of Pittsburgh (Pitt) and Bayer have collaborated to advance research for heart, lung and blood disease indications.