The latest in features and updates that are relevant to sickle cell research, from publications around the world.
11 Mar 2024
Bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio") today announced it has signed its first Medicaid outcomes-based agreement for LYFGENIA™ (lovotibeglogene autotemcel, also known as lovo-cel) with the state of Michigan.
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12 Jan 2024
The study was presented at the annual meeting of the American Society of Hematology, underscoring the intricate needs of children with SCD and their families. It suggests that public health initiatives should be directed towards areas with higher social vulnerability to improve access to care.
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08 Dec 2023
Today, the U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older.
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17 Nov 2023
Health authorities in the U.K. on Thursday approved a new kind of medicine that uses CRISPR gene editing to treat the blood diseases sickle cell and beta thalassemia, marking it the first time a drug built with the Nobel Prize-winning technology has won regulatory clearance anywhere in the world.
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17 Nov 2023
Doctor Peter Lin will tell us about a groundbreaking new treatment that uses gene-editing…with the potential to cure a serious blood disorder. And it could soon get the greenlight for use in the U-S.
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16 Nov 2023
The United Kingdom (U.K.) Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional marketing authorization for CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT)
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06 Nov 2023
If approved by the FDA, it would be the first gene therapy on the U.S. market based on CRISPR, the gene editing tool that won its inventors the Nobel Prize in 2020.
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20 Oct 2023
Researchers at the National Institutes of Health have created a novel gene therapy procedure that could preserve fertility in people with sickle cell disease and other genetic blood conditions.
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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.
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01 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.
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31 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.
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06 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.
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06 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.
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09 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.
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09 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.
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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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