News

The investigational gene editing therapy GPH101 will be the first potentially curative treatment for sickle cell disease (SCD) to be tested in a Phase 1/2 clinical trial. The U.S. Food and Drug Administration (FDA) has cleared GPH101 for clinical testing. With this decision, the therapy’s developer, Graphite Bio,…

BCH-BB694 — an experimental gene therapy targeting the BCL11A gene — safely increased the levels of fetal hemoglobin and prevented disease-associated complications in six people with severe sickle cell disease (SCD), according to interim data from a Phase 1 clinical trial. These findings further support the feasibility and…

Six months of treatment with oral IMR-687 safely and effectively reduced the number of vaso-occlusive crises (VOCs) and other disease-associated pain crises in adults with sickle cell disease (SCD), updated data from a Phase 2a clinical trial show. Most benefits were observed when patients were given the highest doses,…

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…

FT-4202, a potential disease-modifying oral treatment for sickle cell disease (SCD), is well-tolerated in people with SCD and induces improvements in several red blood cell parameters, early clinical trial data indicate. The results observed in this initial group of patients support the efficacy of Forma Therapeutics‘ investigational medication, as…

Sickle Cell Disease News brought you daily coverage of key findings, treatment developments, clinical trials, and other important events related to sickle cell disease (SCD) throughout 2020, a year marked by the COVID-19 pandemic. As a…

To identify at-risk service members, the U.S. Army has begun testing recruits for the sickle cell trait (SCT), and expects all to be screened within a year. The testing began last month and is aimed at giving the Army a snapshot of how SCT has affected its ranks, and…

CTX001, an experimental gene-editing cell therapy, safely and effectively increased the levels of fetal hemoglobin and prevented vaso-occlusive crises (VOCs) in three people with severe sickle cell disease (SCD), according to updated data from the CLIMB-SCD-121 Phase 1/2 trial. CTX001 is being jointly developed by CRISPR…

Emmaus Life Sciences has launched a program that will allow eligible sickle cell disease patients in the U.S. to access its Endari (L-glutamine) medication at low cost or free of charge. Patients who currently lack insurance coverage or financial means to afford treatment may reach out to Endari’s…

Treatment with Oxbryta (voxelotor) may be a feasible alternative to blood transfusions for sickle cell disease (SCD) patients who are experiencing respiratory distress and pneumonia related to COVID-19, a case report suggests. These findings are particularly important during the ongoing pandemic in the U.S., in which rising blood…