The latest in features and updates that are relevant to sickle cell research, from publications around the world.
06 Jan 2023
Graphite Bio has cited a “serious adverse event in the first patient dosed” with its gene therapy nulabeglogene autogedtemcel (nula-cel) and paused its lead program.
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20 Sep 2022
The free program helps individuals become more care-prepared through information, resources, connections, counseling and support.
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16 Sep 2022
A recent analysis of Medicaid claims data found that individuals with sickle cell disease are seeing hematologists at a lower rate than patients with other chronic genetic diseases.
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07 Sep 2022
Danish drugmaker Novo Nordisk will spend $1.1 billion to acquire Forma Therapeutics, a 15-year-old biotechnology company that formed to discover cancer medicines but now has an experimental drug in late-stage testing for treating sickle cell disease.
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31 Aug 2022
President of the United States of America, by virtue of the authority vested in him by the Constitution and the laws of the United States, proclaimed September 2022 as National Sickle Cell Awareness Month.
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23 Aug 2022
Interim results in Ardent trial for sickle cell disease showed no significant difference in median annualized rate of vaso-occlusive crises in high-dose group versus placebo in an intent-to-treat population
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23 Aug 2022
African health ministers today launched a campaign to ramp up awareness, bolster prevention and care to curb the toll of sickle cell disease, one of the most common illnesses in the region but which receives inadequate attention.
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11 Aug 2022
First patient has been dosed with GPH101, now called nulabeglogene autogedtemcel (nula-cel), in the company’s Phase 1/2 CEDAR trial in people with sickle cell disease (SCD). Nula-cel is an investigational gene editing therapy designed to directly correct the genetic mutation that causes SCD and definitively cure the disease.
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08 Aug 2022
Pfizer will acquire GBT, a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments that provide hope to underserved patient communities, starting with sickle cell disease (SCD).
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16 Jul 2022
Gene editing of HSCs in vivo via a non-viral delivery system offers the potential to transform the treatment of sickle cell disease (SCD) and other inherited blood disorders by overcoming the complexity and safety risks of ex vivo approaches.
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08 Jul 2022
Can inhaled nitric oxide relieve pain crises in people with sickle cell disease and does it have other beneficial effects (such as lessening the severity of pain?)
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07 Jul 2022
Scientists from St. Jude Children’s Research Hospital, US, have been investigating a promising new sickle cell treatment by editing genes to switch on the production of healthy, fetal hemoglobin in adult red blood cells.
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10 Jun 2022
For patients with sickle cell disease (SCD), exchange transfusion has benefits over simple transfusion (ST), but a new meta-analysis and systematic review suggests there is insufficient evidence to say which type of exchange transfusion is superior.
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10 Jun 2022
Initial data from the first subjects to receive FTX-6058, an oral HbF inducer, showed a rapid and robust induction of HbF, and subjects achieved increases of up to 6.3% over baseline.
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04 Jun 2022
Patients with sickle cell disease (SCD) are particularly vulnerable, as high amounts of IV opioid medications are standard therapy during vaso-occlusive crises (VOC). [The] institution responded to the crises by implementing IV to oral (PO) conversions of opioid therapies and encouraging multimodal pain management with non-opioid medications.
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