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4 days ago · Clinical trial data on pociredir as SCD therapy expected by midyear. Fulcrum Therapeutics is planning to report, by midyear, new data from the ongoing PIONEER Phase 1b clinical trial that’s testing its oral therapy candidate pociredir (FTX-6058) in people with sickle cell disease (SCD), according to a new corporate presentation.
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.
Prognosis of Sickle Cell Disease
Sickle cell anemia is an inherited blood disorder. It is caused by a mutation in the gene encoding for hemoglobin, the protein in red blood cells that binds to oxygen so it can be transported throughout the body. The abnormal hemoglobin causes disc-shaped red blood cells to deform into a crescent- or sickle-like shape, causing them to become easily trapped in …
News Archives - Sickle Cell Disease News
SCD patients free of VOEs after treatment with gene-editing therapy. Nearly all the patients with severe sickle cell disease (SCD) who were treated with renizgamglogene autogedtemcel (reni-cel) remained free of vaso-occlusive events (VOEs) for up to two years, new data from the Phase 1/2/3 RUBY clinical trial shows.
Stem cell transplant may be feasible for young SCD patients
5 days ago · A hematopoietic stem cell transplant (HCT) may be a feasible treatment option for adolescents and adults with severe sickle cell disease (SCD), according to a U.S. study. Two-year survival rates were similar among patients who had an available donor for HCT and among those who did not, although ...
SCD patients free of VOEs following gene-editing therapy reni-cel
Dec 19, 2024 · The treatment also increased total hemoglobin levels and was well tolerated, according to a company press release from Editas Medicine, the developer of the gene-editing therapy.. Data from 28 patients was presented at the Annual Meeting and Exposition of the American Society of Hematology (ASH) in a poster presentation titled, “Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine ...
Gates Foundation to invest in Tessera SCD gene-editing treatment
Jan 9, 2025 · “We look forward to advancing a genetic medicine that can potentially reshape the treatment landscape for sickle cell disease worldwide,” Michael Severino, MD, Tessera’s CEO, said in a company press release.. SCD is caused by mutations in a gene that provides instructions for making hemoglobin, the protein that red blood cells use to carry oxygen throughout the body.
Most patients free of vaso-occlusive crises for years after Casgevy
Dec 12, 2024 · Vertex Pharmaceuticals, which developed Casgevy alongside CRISPR Therapeutics, shared the findings at the 66 th American Society of Hematology (ASH) Annual Meeting and Exposition, held Dec. 7-10 in San Diego and virtually. The presentation was titled “ Durable Clinical Benefits with Exagamglogene Autotemcel for Severe Sickle Cell Disease.” Data specifically showed that, among 42 patients ...
Celebrating a milestone in gene therapy for sickle cell disease
Nov 18, 2024 · The extensive research, funding, and other resources required for gene therapy to be approved and for a patient to complete treatment can’t be understated.It also takes extreme courage and patience to be the first at anything in medicine. I commend Cromer and his family for taking this leap. Hopefully a long line of cured patients will follow.
Important things you should know about sickle cell disease
Jan 8, 2025 · Complications vary from person to person. While numerous potential complications are associated with sickle cell disease, it’s important to note that not every patient will experience the same issues. Just because one patient suffers a stroke, for example, doesn’t mean others will face the same fate, even if they have identical genotypes.. …