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Home » sickle cell disease

Articles Tagged with ''sickle cell disease''

Bluebird suspends sickle cell studies amid blood cancer concerns

Feb. 16, 2021
By Michael Fitzhugh
Shares of Bluebird Bio Inc. (NASDAQ:BLUE) fell 37.8% to $28.44 on Feb. 16 as the company temporarily suspended two trials of its experimental gene therapy for sickle cell disease, Lentiglobin (BB-1111), while investigating one unexpected case of acute myeloid leukemia (AML) and another of myelodysplastic syndrome among participants in a phase I/II study of the candidate, called HGB-206. A second patient experienced MDS in 2018.
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Sickle cells

Imara wraps up a phase IIa in sickle cell with mixed results

Jan. 6, 2021
By Lee Landenberger
Imara Inc. reported a mixed bag of phase IIa study data for its lead candidate, IMR-687, for treating sickle cell disease in adults, bringing an end to a study that the company found cumbersome and that eventually stung its stock.
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Agios’ anemia treatment hits phase III primary endpoint

Dec. 3, 2020
By Lee Landenberger
Despite challenges tossed at the phase III study of mitapivat from Agios Pharmaceuticals Inc. by COVID-19, top-line data showed the oral therapy hit its primary endpoint in treating adults with pyruvate kinase (PK) deficiency who don’t receive regular transfusions.
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FDA vector objector, Bluebird SCD filing pushed out a year

Nov. 5, 2020
By Randy Osborne
Shares of Bluebird Bio Inc. (NASDAQ:BLUE) sank 16.6%, or $9.72, to close at $48.83 as Wall Street reacted to news that the U.S. regulatory filing for Lentiglobin in sickle cell disease (SCD) will be delayed. Previously expected in the second half of next year, the filing won’t happen until late 2022.
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SCD bid kaput, Cyclerion pedals forth with sGC approach in CNS

Oct. 14, 2020
By Randy Osborne
Cyclerion Therapeutics Inc.’s phase II blowup with sickle cell disease (SCD) candidate olinciguat ended its development, and attention turned to the Cambridge, Mass.-based firm’s earlier-stage effort with IW-6463, a drug in the same class for central nervous system (CNS) disorders.
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Sickle cells

New medicines in the pipeline to treat sickle cell disease

Sep. 18, 2020
By Peter Winter
The CDC estimates that sickle cell disease affects well over 100,000 Americans, with the disease occurring most often in African Americans. September has been designated as National Sickle Cell Awareness month designed to focus attention on the ongoing research in this field and the need for new treatments.
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DNA illustration

Graphite Bio raises $45M series A for targeted DNA integration

Sep. 17, 2020
By Cormac Sheridan
DUBLIN – Versant Ventures, the founding investor of Crispr Therapeutics AG, is teaming up with one of the academic founders of that company, Stanford University’s Matt Porteus, to start a new gene editing firm, Graphite Bio Inc., which is focused on targeted integration of DNA to achieve a therapeutic benefit.
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Dollar arrows pointing upward

Out to launch: IPOs from Repare and Forma post strong debuts

June 19, 2020
By Lee Landenberger
Against a backdrop of general economic uncertainty bolstered in part by a strong biopharma market, two IPOs launched June 19 and ended their first trading day well above their high-end asking prices. Repare Therapeutics Inc., which identifies synthetic lethal precision oncology targets for drug candidates, and Forma Therapeutics Holdings Inc., which is developing therapeutics to treat rare hematologic diseases and cancers, launched IPOs with an anticipated combined value of $497.6 million in gross proceeds.
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Syros, GBT in multimillion-dollar deal to treat sickle cell, beta-thalassemia

Dec. 18, 2019
By Lee Landenberger
Syros Pharmaceuticals Inc. and Global Blood Therapeutics Inc. (GBT) agreed to a discovery, development and commercialization deal to treat sickle cell disease (SCD) and beta-thalassemia. GBT, of South San Francisco, Calif., will pay Syros $20 million up front and fund up to $40 million in preclinical research for at least three years, with the goal of identifying targets and discovering drugs to induce fetal hemoglobin.
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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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