A small proof-of-concept clinical trial evaluating motixafortide for stem cell mobilization — regimens essential for gene therapies — in sickle cell disease (SCD) has dosed its first patient. Stem cell mobilization regimens currently available can cause serious side effects in SCD patients. This Phase 1 study (NCT05618301),…
Throughout 2023, Sickle Cell Disease News brought readers coverage of the latest clinical research and scientific breakthroughs related to sickle cell disease (SCD). Here are the year’s top 10 most-read articles, each with a brief description. We hope to remain a dependable resource for the SCD community in…
Rates of life-threatening infections by pneumococcal bacteria have significantly declined, by about 80%, among children with sickle cell disease (SCD) since the introduction of pneumococcal conjugate vaccines 20 years ago. That’s according to a study analyzing more than two decades of healthcare data from young SCD patients in metropolitan…
Treatment with the investigational gene-editing therapy OTQ923 led to the sustained production of fetal hemoglobin and prevented vaso-occlusive crises (VOCs) in three adults with severe sickle cell disease (SCD), according to data from a Phase 1 study. The treatment involved collecting red blood cell precursors, called hematopoietic stem…
Preventive red blood cell transfusions may protect against complications associated with pregnancy in women with sickle cell disease (SCD), a 13-year study has found. Findings indicate that the rates of premature births, as well as SCD complications, such as vaso-occlusive crises (VOCs) and acute chest syndrome (ACS), are…
Lobe Sciences has partnered with Pentec Health to distribute Altemia, an emulsified formulation of docosahexaenoic acid (DHA), a type of omega-3 fat, for sickle cell disease (SCD) patients in the U.S. and its territories. As part of the agreement, Pentec, which provides clinical nutrition and specialty care…
The investigational oral treatment FTX-6058 effectively raised the level of fetal hemoglobin in a sickle cell disease (SCD) patient to 24.9% in a clinical trial that’s now been placed on hold by regulators. According to the therapy’s developer, Fulcrum Therapeutics, these initial data had been obtained but not…
A Sickle Cell Carrier Status Awareness program has been launched to help identify people who carry genetic variants associated with sickle cell disease (SCD). Established through a collaboration between the Sickle Cell Foundation of Georgia (SCFG), the Morehouse School of Medicine, and the human genetic testing company 23andMe,…
Bluebird Bio has provided the U.S. Food and Drug Administration (FDA) with information regarding the commercial manufacturing of its experimental gene therapy for sickle cell disease (SCD) in preparation for requesting its approval. The company had expected to submit a biologics license application seeking the approval of…
Jasper Therapeutics’ investigational conditioning therapy briquilimab safely improved the success of stem cell transplants for three people with sickle cell disease (SCD), early Phase 1/2 trial data suggest. Stem cell transplant success was observed in all three patients treated with briquilimab, plus a less-aggressive non-myeloablative conditioning regimen,…
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