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BioWorld - Sunday, April 26, 2026
Home » Topics » Clinical

Clinical
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Sickle cell disease
ASH 2019

‘Multiple ways’ of fighting once-incurable sickle cell disease

Dec. 10, 2019
By Anette Breindl
ORLANDO, Fla. – At the 61st ASH annual meeting late-breaking abstracts session, researchers from Boston Children’s Hospital reported that three adult patients who had received an autologous transplant of gene-edited hematopoietic stem cells lacking BCL11A produced high levels of functional hemoglobin and had reduced disease symptoms for at least eight months after transplantation. 
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ASH 2019

Sanofi's sutimlimab delivers benefit for CAD patients in phase III study

Dec. 10, 2019
By Michael Fitzhugh
ORLANDO, Fla. – New research on sutimlimab, an investigational complement pathway inhibitor under development by Sanofi SA's Bioverativ unit, showed substantial benefits for people with the rare autoimmune disorder cold agglutinin disease (CAD).
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Red blood cells
ASH 2019

Forty Seven’s data eat up the market, with positive results in MDS and AML

Dec. 9, 2019
By Lee Landenberger
ORLANDO, Fla. – Data from two studies from Forty Seven Inc. announced at the American Society of Hematology (ASH) annual meeting moved the market in a major way Monday as the company stock (NASDAQ:FTSV) soared 111% to close at $30.43, up $15.99.
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ASH 2019

Constellation shares fall after myelofibrosis update, despite progress

Dec. 9, 2019
By Michael Fitzhugh
ORLANDO, Fla. – Preliminary data from a phase II test of Constellation Pharmaceuticals Inc.'s CPI-0610 in patients with myelofibrosis (MF), previewed to market adulation in November, got a full airing Monday at the American Society of Hematology (ASH) annual meeting.
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CTAD 2019

Small Alzheimer’s drug companies look to advance quickly into late-stage

Dec. 9, 2019
By Brian Orelli
SAN DIEGO – Smaller companies looking to move their Alzheimer’s disease drugs into late-stage testing as quickly as possible are eschewing cognitive endpoints that can take years to readout for biomarkers and functional assays of brain activity.
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ASH 2019

Janssen’s Imbruvica improves survivability

Dec. 9, 2019
By Lee Landenberger
ORLANDO, Fla. – Not long past the opening of the American Society of Hematology’s (ASH) massive annual conference, Janssen Biotech Inc. released upbeat follow-up survivability data on Imbruvica (ibrutinib) from two studies and an integrated analysis evaluating Imbruvica in previously untreated patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
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ASH 2019

Autolus’ CAR T therapy is well-tolerated

Dec. 9, 2019
By Lee Landenberger
ORLANDO, Fla. – New phase I/II data from Autolus Therapeutics plc announced at the American Society of Hematology’s (ASH) annual conference show that AUTO-3, the first bicistronic CAR T targeting CD19 and CD22 followed by an anti-PD1, was well-tolerated in a phase I/II study.
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ASH 2019

Kura Oncology plans pivotal trial in rare form of PTCL

Dec. 9, 2019
By Michael Fitzhugh
ORLANDO, Fla. – Kura Oncology Inc., encouraged by new data on tipifarnib and positive feedback from the FDA, said Sunday that next year it will initiate a single-arm, phase II registration-directed trial of the candidate in angioimmunoblastic T-cell lymphoma (AITL), a rare and often aggressive form of peripheral T-cell lymphoma (PTCL).  
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ASH 2019

Genentech showcases new data on CD20-CD3 bispecific for tough-to-treat lymphomas

Dec. 9, 2019
By Michael Fitzhugh
ORLANDO, Fla. - Chimeric antigen receptor T (CAR T) cells have proved to be a major advance in treating patients with refractory B-cell malignancies so far. But, often, in approaching those issues "we solve one and we create another," Stephen Schuster, a doctor at the Abramson Cancer Center at the University of Pennsylvania in Philadelphia, told reporters at the 61st American Society of Hematology (ASH) annual meeting Saturday.
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‘Fergene’ therapy in BCG-stubborn bladder cancer, Instiladrin instills phase III confidence

Dec. 6, 2019
By Randy Osborne
The “remarkably appealing” route of administration and every-three-month dosing put Ferring Pharmaceuticals SA’s nadofaragene firadenovec (rAd-IFN/Syn3, also known as Instiladrin) in strong position for approval in high-grade Bacillus Calmette-Guérin (BCG) unresponsive non-muscle invasive bladder cancer (NMIBC).  
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