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5 days ago · Orna, Vertex partner on gene-editing therapies for SCD, TDT. Orna Therapeutics is partnering with Vertex Pharmaceuticals to develop gene-editing therapies for people with sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).
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.
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.
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 ...
New program to aid people living with sickle cell disease in...
3 days ago · Texas Children’s Global HOPE and Baylor College of Medicine Global Health, collaborating with the independent charity Bristol Myers Squibb Foundation, have announced a pioneering program to combat sickle cell disease (SCD) in Sub-Saharan Africa. Launching first in Tanzania and Uganda, this program ...
Blood transfusion for sickle cell disease
Aug 16, 2024 · SCD is a disorder that impacts red blood cells, which are primarily responsible for carrying oxygen throughout the body. SCD is caused by genetic mutations that lead to the production of an abnormal form of hemoglobin — the protein that red blood cells use to carry oxygen — with red blood cells taking on the sickle-like shape that characterizes the disease.
38 and counting: Thriving with sickle cell disease
Oct 16, 2024 · Sickle cell challenges intensify with age. I’ve faced painful crises, chronic fatigue syndrome, cardiomegaly, depression, avascular necrosis, infections, bone issues, physical disability, and other complications.However, I’ve developed a strong sense of resilience and a never-give-up attitude. Each challenge became a chance to …
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 ...
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.
About Us – Sickle Cell Disease News
Sickle Cell Disease News is a digital platform intended to provide the sickle cell disease (SCD) community with the most recent news and information on SCD, as well as first-hand community perspectives from our patient and caregiver columnists.. All articles on Sickle Cell Disease News are original content produced by in-house writers and editors. . …