Sickle Cell Disease News Home | Sickle Cell Disease News
Dec 13, 2024 · 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.
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 ...
Gates Foundation to invest in Tessera SCD gene-editing treatment
1 day ago · “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.
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.
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.
Most sickle cell patients VOE-free after Lyfgenia gene therapy
Dec 12, 2023 · That’s according to new data from Lyfgenia’s developer, Bluebird Bio, at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition.Also known as lovo-cel, Lyfgenia was last week approved by the U.S. Food and Drug Administration (FDA) to treat SCD patients ages 12 and older with a history of vase-occlusive events (VOEs). “Years of long-term follow-up continue to suggest ...
Important things you should know about sickle cell disease
2 days ago · 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.. …
FDA clears study of potentially less toxic SCD conditioning regimen
Jul 30, 2024 · In SCD, mutations in the HBB gene result in the production of a faulty version of hemoglobin, the protein in red blood cells that carries oxygen through the body. Faulty hemoglobin tends to form clumps, forcing red blood cells to adopt a sickle-like shape. The misshaped red blood cells are more prone to die prematurely and can obstruct blood vessels, causing patients to develop anemia and ...
Low-intensity stem cell transplants may benefit SCD patients: Study
Sep 3, 2024 · “By using a low-intensity blood stem cell transplant for sickle cell disease, we may be able to stop the cycle of lung injury [seen in SCD patients] and prevent continued damage,” Parker Ruhl, MD, an associate research physician and pulmonologist at the NIH, and the study’s first author, said in an agency press release.. These findings may prove informative for people with SCD who may be ...
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.