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3 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.
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 ...
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 ...
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.
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 ...
Study to compare SCD treatments for symptoms vs. a transplant
Nov 12, 2024 · Particularly, the study by scientists with Golisano Children’s Hospital will compare sickle cell treatments that help to control disease symptoms, such as blood transfusions and hydroxyurea, with those aiming for a cure, like bone marrow transplantation.. The project aims to recruit nearly 500 children and young adults with SCD, who either underwent a transplant or are using disease ...
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 ...
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. . …
I’m tired of watching sickle cell patients have preventable deaths
Nov 22, 2024 · Here in the U.K., I’ve seen two recent reports about sickle cell patients dying from negligence. Both of their deaths were preventable. As MyLondon reported in September, Dave Onawelo presented to the emergency department in December 2023 and told staff he was experiencing a sickle cell crisis.According to a coroner’s report, he was deemed not acutely unwell after an initial assessment and ...