News

The latest in features and updates that are relevant to sickle cell research, from publications around the world.

Graphite Bio Doses First Patient with Investigational Gene Editing Therapy GPH101 for Sickle Cell Disease

19 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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Pfizer to Acquire Global Blood Therapeutics for $5.4 Billion to Enhance Presence in Rare Hematology

19 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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Intellia Therapeutics Presents Preclinical Proof of Concept for CRISPR-based In Vivo Editing of Bone Marrow at Keystone eSymposium

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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Inhaled nitric oxide for treating pain crises in people with sickle cell disease

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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Researchers have found that new sickle cell disease gene therapies depend on choosing the right laboratory mice.

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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Evidence Unclear About Superiority of Exchange Transfusion Types in Sickle Cell Disease

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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Fulcrum Therapeutics Announces Proof-of-Concept for FTX-6058 in Sickle Cell Disease Based on Initial Data from the Ongoing Phase 1b Trial

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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Opioid Use in Vaso-Occlusive Crisis During Intravenous Opioid Drug Shortage

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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