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BioWorld - Friday, December 19, 2025
Home » Topics » Hematologic, BioWorld

Hematologic, BioWorld
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Durable goods? Biomarin ‘rox’ in hemophilia A with two-year gene therapy data

Jan. 10, 2022
By Randy Osborne
Having unveiled more data from the ongoing, global phase III Gener8-1 study with Roctavian (valoctocogene roxaparvovec, also known as valrox), Biomarin Pharmaceutical Inc. remains on track to file a regulatory submission with the FDA in the second quarter of this year for the gene therapy to treat adults with severe hemophilia A. The EMA is already reviewing a marketing authorization application.
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Conference data for Dec. 17, 2021: ASH

Dec. 17, 2021
New and updated preclinical and clinical data presented by biopharma firms at the American Society of Hematology annual meeting, including: Century, Forma.
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Conference data for Dec. 16, 2021: ASH

Dec. 16, 2021
New and updated preclinical and clinical data presented by biopharma firms at the American Society of Hematology annual meeting, including: Beyondspring, Coimmune, Geron, JW, Novartis, Oncolytic, Oncternal, Oric, Oryzon, Ose, Pfizer, Poseida, Precigen, Rheos, Roche, Rocket, Sangamo, Sanofi, Seagen, Secura, Sierra, Syndax, Talaris, Tessa, TG, Viracta, Wugen, Xencor.
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Beth Psaila, co-founder, Alethiomics

Alethiomics raises $8M in seed finance to bring multi-omics analysis to myeloproliferative neoplasms

Dec. 15, 2021
By Cormac Sheridan
DUBLIN – Fans of “His Dark Materials” and the “Book of Dust,” Philip Pullman’s series of fantasy novels set in Oxford, U.K. and more remote corners of the earth, are familiar with alethiometers. In the hands of a highly select number of characters, these complex, compass-like devices can interrogate a mysterious substance called “dust” – an elementary particle associated with consciousness – and provide trusted answers to the most crucial questions.
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Conference data for Dec. 15, 2021: ASH

Dec. 15, 2021
New and updated preclinical and clinical data presented by biopharma firms at the American Society of Hematology annual meeting, including: 2seventy, Actinium, Agios, Allogene, Antegene, Apellis, Aptose, Ascentage, Atar, Bantam, Bioinvent, Daiichi, Harpoon, Iaso, Innocare, Innovent, Janssen, Karyopharm, Keros, Kymera, Lantern, Legend, Magenta, Mereo, Mustang, Nektar, Neximmune, Nextcure, Nkarta.
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ASH 2021: For fitusiran, efficacy is impressive but safety’s not there yet

Dec. 14, 2021
By Anette Breindl
Two trials presented at the annual meeting of the American Society of Hematology (ASH) showed that treatment with the antithrombin inhibitor fitusiran reduced the bleeding rate in patients with severe hemophilia by roughly 90%, regardless of whether they had type A or B, and whether they had developed antibodies to recombinant clotting factors.
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Conference data for Dec. 14, 2021: ASH

Dec. 14, 2021
New and updated preclinical and clinical data presented by biopharma firms at the American Society of Hematology annual meeting, including: Actinium, Adagene, Affimed, Agios, Allogene, Annexon, Aprea, Arcellx, Astrazeneca, Autolus, Beigene, Editas, Emmaus, Epizyme, Equillium, Fate, Freeline, Gamida, GBT, Genentech, Glycomimetics, GSK, Hutchmed, I-Mab, Imara, Kite, Kura, Sutro.
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Hand holding "Acquisition" piece over puzzle

CSL to acquire Vifor for $11.7B to expand renal and iron deficiency pipelines

Dec. 14, 2021
By Tamra Sami
PERTH, Australia – In the second multi-billion-dollar pharma merger this week, CSL Ltd. has announced plans to buy Vifor Pharma Ltd. for an equity value of $11.7 billion. It’s also the second largest biopharma merger of 2021, a whisker above Merck & Co Inc.’s $11.5 billion acquisition of Acceleron Pharma Inc. in November, although way off Astrazeneca plc’s $39 billion buy of Alexion Pharmaceuticals Inc. in July.
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Conference data for Dec. 13, 2021: ASH

Dec. 13, 2021
New and updated preclinical and clinical data presented by biopharma firms at the American Society of Hematology annual meeting, including: Aleta, Allovir, Alx, Beigene, Bio-Path, Bluebird, BMS, Carsgen, Catamaran, CDR-Life, Cellectis, Celularity, Corvus, CTI, Epizyme, Forma, GBT, Genentech, Harpoon, Ichnos, IGM, Imago, Immunicum, Immunogen, Incyte, Janssen, Jazz, Kite, Loxo, Maat, Morphosys, Neoleukin, Novartis, Orca, Precision, Protagonist, Regeneron, Samsung, Sana, Schrodinger, Servier, Silence, Syndax, Tessa, Vincerx, X4.
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Neurons

ASH 2021: Blood disorder CHIPs away Alzheimer's disease risk

Dec. 13, 2021
By Anette Breindl
Clonal hematopoiesis of indeterminate potential (CHIP) was associated with a 35% reduced risk of Alzheimer's disease (AD) in studies presented at the plenary session of the American Society of Hematology (ASH) annual meeting on Sunday.
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