Guest Voice: Moving, literally, in the right direction for sickle cell care. Richard Bayliss is 44 years old and has sickle cell disease with hemoglobin SS disease.

5 天之前 · With the addition of Casgevy, CHLA is now offering two gene therapies for sickle cell. The hospital has been offering Lyfgenia (lovotibeglogene autotemcel), a gene therapy that works by providing patients with a gene that encodes a sickle-resistant version of adult hemoglobin.. Both therapies were approved by the U.S. Food and Drug Administration (FDA) in late 2023 to treat people with SCD ...

2025年1月31日 · New treatments and therapies are being developed every day for people with sickle cell disease. I take hydroxycarbamide daily. The medicine was initially designed for cancer patients, but sickle cell patients have also noted improvements in well-being while taking it.

2022年1月4日 · 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.

2024年1月2日 · No. 9 – Bluebird provides FDA with manufacturing information on gene therapy. Lovo-cel (lovotibeglogene autotemcel) is a one-time gene therapy developed by Bluebird Bio and now approved and marketed as Lyfgenia in the U.S. It’s designed to insert a functional modified copy of a gene, which is defective in SCD, into patient-derived blood stem cells.. The modified stem cells are then ...

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. . …

2023年12月8日 · The U.S. Food and Drug Administration (FDA) has approved lovotibeglogene autotemcel, a gene therapy from Bluebird Bio known as lovo-cel, for treating sickle cell disease (SCD) patients ages 12 and older who have a history of vaso-occlusive events (VOEs), including painful vaso-occlusive crises (VOCs), acute chest syndrome, and …

2025年1月23日 · 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 ...

2022年12月29日 · CTX001 is a gene-editing therapy that aims to boost the production of HbF in blood cells. Its developers CRISPR Therapeutics and Vertex announced the launch of a Phase 3 trial called VX21-CTX001-151 (NCT05329649) to test the therapy in children ages 2 to 11 with severe SCD who either don’t respond to hydroxyurea or are intolerant to it. A similar trial, called VX21-CTX001-141 (NCT05356195 ...

2024年12月19日 · 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 ...