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BioWorld - Sunday, June 28, 2026
Home » Topics » Disease categories and therapies » Hematologic

Hematologic
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Keeping stem cells quiescent enables greater ultimate potency

March 10, 2020

BioCryst initiates proof-of-concept trial of BCX-9930 in PNH

March 6, 2020

Keros’ pedal-down TGF-beta push touts safety, draws $56M series C

March 5, 2020
By Randy Osborne
Keros Therapeutics Inc. CEO Jasbir Seehra told BioWorld that he plans to use at his new company lessons learned as co-founder of Acceleron Pharma Inc., where work with receptors in the TGF-beta superfamily “taught me the potential of the biology and those molecules, but also the limitations” with regard to safety that need to be surmounted.
Read More

Omeros reports new data from pivotal trial of narsoplimab for HSCT-TMA

March 3, 2020

From Ixinity and beyond: Buzz for Aptevo as bispecific pure play ‘cleaner’

Feb. 28, 2020
By Randy Osborne
Aptevo Therapeutics Inc.’s chief scientific officer, Jane Gross, told BioWorld that the sale of the firm’s marketed recombinant hemophilia B therapeutic, Ixinity, to Medexus Pharmaceuticals Inc. allowed for a “cleaner message” to Wall Street. “It was a little difficult to explain having a commercial asset and an R&D pipeline,” she said.
Read More
John Hood at the Biocom Global Life Sciences
Sometimes it takes an advocate (or four)

Backstory of the FDA lifting its clinical hold and eventual approval of Inrebic

Feb. 28, 2020
By Brian Orelli
SAN DIEGO – At Biocom's 10th Annual Global Life Sciences Partnering Conference, a panel of players intimately familiar with last year's approval of myelofibrosis treatment Inrebic (fedratinib) explained the backstory of how they got the JAK2 kinase inhibitor off an FDA clinical hold, wrangled the rights to the drug back from the big pharma owner that had acquired the drug from Targegen Inc. and eventually helped the drug gain FDA approval after selling the rights to another large company.
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Phase I data for novel lentiviral vector therapy for children with Fanconi anemia

Feb. 28, 2020

Bluebird bio presents phase I/II data on LentiGlobin for sickle cell disease

Feb. 25, 2020
Blood sample, DNA

Biomarin’s BLA for Valrox gene therapy for hemophilia A accepted by FDA

Feb. 24, 2020
By Peter Winter
Valrox (valoctocogene roxaparvovec) from San Rafael Calif-based Biomarin Pharmaceutical Inc. moved one step closer to entering the U.S. market, with the company reporting that that the FDA had accepted for priority review the BLA for its investigational AAV5 gene therapy for adults with hemophilia A.
Read More

Phase IIb results presented for dalcinonacog alfa in hemophilia B patients

Feb. 12, 2020
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