News

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

World’s first patient starts sickle cell gene therapy

20 May 2024

Kendric Cromer, a 12-year-old boy in Washington, last week became the first person in the world with sickle cell disease to begin a commercially approved gene therapy that may cure the condition, and change his whole life.

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Bluebird Bio Announces First Outcomes-Based Agreement with Medicaid for Sickle Cell Disease Gene Therapy

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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Study Highlights Sickle Cell Disease Prevalence in Socially Disadvantaged Areas

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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FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease

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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World’s first CRISPR medicine approved in UK for sickle cell, beta thalassemia

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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New treatment for sickle cell disease could soon be approved by the U.S. FDA: Dr. Peter Lin

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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Vertex and CRISPR Therapeutics Announce Authorization of the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), by the United Kingdom MHRA for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

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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A new cure for sickle cell disease may be coming. FDA advisers will review it next week.

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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NIH Researchers Work to Preserve Fertility for People Undergoing Gene Therapy

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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South Side Resident Takes Part in Rare Sickle Cell Disease Clinical Trial Through UChicago Medicine

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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FDA lifts its clinical hold on oral therapy FTX-6058 for SCD

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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Sickle Cell 101 Launches Summer Sickle Cell Trait Awareness and Hydration Campaign with DPJ Football Camp Partnership

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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Gene Therapy Trial Provides Hope for Sickle Cell Disease Patients

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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Tool To Diagnose and Monitor Sickle Cell Disease Receives U.S. Patent

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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Trailing other sickle cell drug makers, Editas still sees opportunity for its CRISPR therapy

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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Gene Editing Race Between Editas and Vertex, CRISPR Heats Up

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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Graphite’s hopes for sickle cell ‘cure’ blunted after first patient dosed experiences serious event

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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Sickle Cell Warrior Program from NMDP/Be The Match Helps Eliminate Barriers to Equitable Care

20 Sep 2022

The free program helps individuals become more care-prepared through information, resources, connections, counseling and support.

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Patients With Sickle Cell Disease Less Likely to Receive Specialized Care

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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With rare acquisition, Novo Nordisk makes $1B bet on sickle cell disease drug

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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A Proclamation on National Sickle Cell Awareness Month, 2022

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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Imara Announces Results of Interim Analyses of Tovinontrine (IMR-687) Phase 2b Clinical Trials in Sickle Cell Disease and Beta-Thalassemia

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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African health ministers launch drive to curb sickle cell disease toll

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

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

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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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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