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BioWorld - Thursday, December 18, 2025
Home » Topics » Disease categories and therapies » Hematologic

Hematologic
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Biotest adds additional patient group to ongoing phase III AdFIrst study of BT-524

April 8, 2021

Fireball in gene therapy? Wall Street boosters like Rocket’s trajectory

April 7, 2021
By Randy Osborne
Cambridge, Mass.-based Agios Therapeutics Inc.’s encouraging phase III data from a pair of trials with allosteric activator mitapivat in pyruvate kinase deficiency brought more attention to the space, where Rocket Pharmaceuticals Inc. – at a much earlier stage – is trying a gene therapy called RP-L301.
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FDA clears initiation of first-in-human study of CRISPR_SCD-001 for sickle cell disease

March 31, 2021

Phase II trial of IONIS-PKK-LRx as treatment for HAE meets endpoints

March 30, 2021

Japanese approval of additional indication for Riona

March 25, 2021

Kinase links innate immunity to MDS

March 24, 2021

Biotest presents FVIII-based recombinant proteins for hemophilia A

March 24, 2021

BioCryst presents successful data from proof-of-concept trial of BCX-9930 in PNH

March 23, 2021

Cellphire initiates phase II study of Thrombosomes for bleeding in patients with thrombocytopenia

March 22, 2021

New results presented for oral formulation of milvexian

March 18, 2021
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