Sickle Cell Disease News Home | Sickle Cell Disease News
Nov 1, 2024 · BEAM-101 gene-editing stem cell therapy shows promise in SCD. In a Phase 1/2 clinical trial, the gene-editing stem cell therapy BEAM-101 worked as intended, and showed early signs of efficacy in people with severe sickle cell disease (SCD), according to new early data.
More stem cells for sickle cell gene therapy mobilized with...
11 hours ago · The proof-of-concept clinical trial (NCT05618301) is testing how safe and well tolerated motixafortide is in a small group of adults with SCD, and how well it works alone or with natalizumab to mobilize CD34-positive hematopoietic stem cells, which can give rise to red blood cells. “We are encouraged by the initial findings in this Phase 1 study showing that motixafortide is safe and well ...
Vaso-Occlusive Crisis - Sickle Cell Disease News
Jan 4, 2022 · Sickle cell disease (SCD) is an inherited disorder caused by a mutation in the gene encoding a subunit of hemoglobin, the protein that carries oxygen in red blood cells. Mutant hemoglobin — called sickle hemoglobin or hemoglobin S — tends to clump together in red blood cells, causing them to take on an irregular “sickle-like” shape that gives the disease its name.
Oral AND017 granted orphan drug status for SCD
Oct 31, 2024 · Better safety, efficacy. In preclinical studies, AND017 appeared to be safe and work as intended. For Gang Huang, PhD, a professor at the University of Texas Health San Antonio who was involved in those studies, AND017 “might not only provide a novel oral treatment with unique mechanism of action, but also an obvious better safety and efficacy profile.”
Study to compare SCD treatments for symptoms vs. a transplant
3 days ago · A bone marrow transplant, in which hematopoietic stem cells responsible for giving rise to new blood cells are replaced by those derived from a healthy donor, is an invasive treatment but one also seen as a possible cure of SCD.However, the donor must match the patient’s human leukocyte antigens, a set of proteins …
Gene Therapy – Sickle Cell Disease News
Four trials (NCT02186418, NCT03282656, NCT02247843, NCT02140554) are testing the efficacy and safety of gene therapy to replace the mutated HBB gene with a healthy HBB gene.These Phase 2 trials are recruiting both children and adults in the United States and Jamaica. Three trials (NCT02193191, NCT02989701, NCT03226691) are investigating the use of Mozobil (plerixafor) in patients with sickle ...
New regimen finds bone marrow transplant an SCD 'curative option'
Apr 30, 2024 · An allogeneic stem cell transplant uses donor stem cells to replace a patient’s blood-forming stem cells in the bone marrow. The transplanted cells then start to make new, healthy red blood cells. Allogeneic stem cell transplants offer a cure for SCD, but their use can be limited by difficulties in finding matched donors and the aggressiveness of myeloablative conditioning regimens.
AB Science gets European patent covering masitinib for SCD
Nov 5, 2024 · AB Science said it received a notice of allowance from the European Patent Office for a patent covering the use of the experimental oral therapy masitinib to treat sickle cell disease (SCD). The decision was based on preclinical data demonstrating that masitinib was able to prevent vaso-occlusive ...
Risk of blood clots more likely with sickle cell trait, but still low
Sep 19, 2024 · Still, the researchers said, the risk of these blood clots was low, occurring in just over 2% of people carrying SCT. The study, “ Ancestry-Independent Risk of Venous Thromboembolism in Individuals with Sickle Cell Trait vs. Factor V Leiden,” was published in Blood Advances. “I hope this study informs health professionals, particularly hematologists, that when thinking about risks ...
Infections are a silent menace for many with sickle cell disease
Mar 27, 2024 · Pain and anemia seem to be the loud hallmarks of sickle cell disease.Because many patients and caregivers prioritize addressing the challenges posed by these issues, they tend to overlook infections. But despite their quieter nature, infections pose a grave threat and stand as the leading cause of death among people living with sickle cell disease.