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BioWorld - Thursday, June 11, 2026
Home » Topics » Disease categories and therapies » Hematologic

Hematologic
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Holding out for a hero: Protagonist takes a market hit

Dec. 3, 2019
By Lee Landenberger
Protagonist Therapeutics Inc. stock (NASDAQ:PTGX) nosedived 47% on Tuesday after the company released preliminary results from its phase II open-label study of PTG-300, an injectable hepcidin mimetic to treat patients with transfusion-dependent beta-thalassemia.  
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Funding supports LUNAC-led project to develop next-generation anticoagulant

Dec. 3, 2019

Akari announces trial design for pivotal phase III study of nomacopan in pediatric HSCT-TMA

Dec. 3, 2019

Shenzhen Salubris Pharmaceuticals identifies new PAR-4 antagonists

Dec. 2, 2019

La Jolla re-evaluates its development of LJPC-401 due to mixed clinical results

Nov. 27, 2019
ASH 2019

World's biggest hematology confab to feature new therapies, big-picture research

Nov. 26, 2019
By Michael Fitzhugh
With nearly 5,000 abstracts on tap for this December's American Society of Hematology (ASH) meeting in Orlando, Fla., organizers of the annual conference have highlighted a multitude of new advances in the event's programming.
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GBT’s Oxbryta gains nod in SCD; one-two punch for patients with Novartis win

Nov. 26, 2019
By Randy Osborne

Less than two weeks after giving the go-ahead to Novartis AG for Adakveo (crizanlizumab) to reduce the frequency of vaso-occlusive crises (VOCs) in adult and pediatric patients ages 16 and older with sickle cell disease (SCD), the FDA cleared – well ahead of its Feb. 26, 2020, PDUFA date – Oxbryta (voxelotor), from Global Blood Therapeutics Inc. (GBT), for SCD in adults and pediatric patients ages 12 and up. 


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FDA grants accelerated approval to Oxbryta for sickle cell disease

Nov. 26, 2019

Mixed clinical results jolt La Jolla Pharma

Nov. 25, 2019
By Lee Landenberger
La Jolla Pharmaceutical Co. discontinued its study LJ401-BT01 due to lack of efficacy and will reassess further development of LJPC-401 (synthetic human hepcidin) based on recent mixed clinical results.
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Preliminary clinical data on CRISPR/Cas9 gene-editing therapy CTX-001 in severe hemoglobinopathies

Nov. 20, 2019
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