WebFeb 16, 2024 · Bluebird suspends studies of sickle cell gene therapy following cancer diagnoses in two more treated patients. B luebird Bio said Tuesday that it has suspended … http://hs.www.nature.com.dr2am.wust.edu.cn/subjects/sickle-cell-disease/cgt
Bluebird responds to FDA on sickle cell gene therapy
Historically, sickle-cell disease claimed many lives in childhood. Advances in medical care mean that people who are affected can now survive to middle age, and a growing arsenal of drugs is helping these individuals to manage their pain and other symptoms effectively. But right now, the only option for long-term … See more Strategies based on restoring functional β-globin expression have generally relied on lentiviruses to deliver their therapeutic payload. These retroviruses can insert foreign genes into host genomes in a process known as … See more Lentiviral vectors have a strong track record in gene therapy, and Kohn notes that “hundreds of patients have gotten current-generation lentiviral vectors into their stem cells for … See more Even if there is a link to cancer risk, this will not be the end of the line for gene therapy in sickle-cell disease. For example, as Williams and Kohn reopen their respective lentivirus … See more Enthusiasm about gene therapy’s potential for treating sickle-cell disease has been tempered by recent safety concerns that have delayed multiple studies. In February, Bluebird Bio announced that it was suspending its trial … See more WebSep 14, 2024 · An experimental gene therapy she received last year successfully rid her of her sickle cell disease, eliminating her intense suffering and transforming her into a … cso from idg
UPDATE 1-Vertex/CRISPR
WebNov 30, 2024 · Background: Sickle cell disease encompasses a group of genetic disorders characterized by the presence of at least one hemoglobin S (Hb S) allele, and a second abnormal allele that could allow abnormal haemoglobin polymerisation leading to a symptomatic disorder. Autosomal recessive disorders (such as sickle cell disease) are … WebDec 10, 2024 · Sickle cell disease (SCD) has been well characterized for over 100 years, with the first clinical report published in 1910 describing it as the “first molecular disease.” 1 … Web1 day ago · ICER backs sickle cell gene therapies at one-off cost of $2m. The influential Institute for Clinical and Economic Review (ICER) in the US has issued draft guidance … eai mano brown