The latest in features and updates that are relevant to sickle cell research, from publications around the world.
18 Sep 2023
Lyric Porter has been a patient at the University of Chicago Medical Center since the day she was born. In 2019, after graduating from college, Porter learned of a clinical trial from her physicians at UChicago Medicine. Along with the other patients in the clinical trial, she saw a decrease in the number of sickle cell crises she experienced — a positive sign for her quality of life going forward.
Learn more01 Sep 2023
The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on FTX-6058, an investigational oral treatment for sickle cell disease (SCD) that’s being developed by Fulcrum Therapeutics.
Learn more31 Jul 2023
Donovan Peoples-Jones (DPJ) continues to partner with Sickle Cell 101 in diverse capacities, including the NFL My Cause My Cleats Campaign to improve education, and research funding for sickle cell disease and sickle cell trait.
Learn more06 Jul 2023
The RUBY trial, conducted by Editas Medicine, explores an experimental gene therapy for sickle cell disease aiming to be a functional cure by modifying a patient’s own stem cells to correct the genetic defect, showing promising early results and improved quality of life for participants.
Learn more06 Jul 2023
Dr. Sarah Du, a researcher and associate professor at Florida Atlantic University, has recently been granted a patent by the United States Patent and Trademark Office. This groundbreaking invention offers patients a potentially enhanced approach to managing their disease.
Learn more09 Jun 2023
On Friday, Editas offered a preliminary update on the handful of patients who’ve received its gene-editing-based therapy for sickle cell, which is also being tested for another blood disorder, beta thalassemia.
Learn more09 Jun 2023
Editas Medicine unveiled early data from the RUBY and EdiTHAL trials, demonstrating the potential of its gene editor EDIT-301 in sickle cell disease and transfusion-dependent beta thalassemia.
Learn more06 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.
Learn more20 Sep 2022
The free program helps individuals become more care-prepared through information, resources, connections, counseling and support.
Learn more16 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.
Learn more07 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.
Learn more31 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.
Learn more23 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
Learn more23 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.
Learn more11 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.
Learn more08 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).
Learn more16 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.
Learn more08 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?)
Learn more07 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.
Learn more10 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.
Learn more10 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.
Learn more04 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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