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Jan 8, 2025 · Sickle cell disease can take a toll on a person’s mental health, too. Living with sickle cell disease comes with a heavy cost. Much of your time is spent seeking to understand your body, how the condition impacts you as a person, and then adjusting your lifestyle to …
Important things you should know about sickle cell disease
6 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.. …
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 ...
Gates Foundation to invest in Tessera SCD gene-editing treatment
6 days 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.
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 ...
Phase 3 RISE UP study of mitapivat for SCD completes enrollment
Oct 24, 2024 · Results from 79 patients who took part in the Phase 2 dose-finding study, announced late last year, indicated the two tested doses of mitapivat (50 or 100 mg taken twice daily) could boost hemoglobin levels compared with a placebo over the course of about three months.Both doses also led to reductions in the annualized rate of pain crises. In the Phase 3 study, participants will be randomly ...
Top 10 sickle cell disease stories of 2023
Jan 2, 2024 · 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 ...
Lovo-cel, now Lyfgenia wins FDA approval for sickle cell disease
Dec 8, 2023 · 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 …
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. . …
Top 10 Sickle Cell Disease Stories of 2022
Dec 29, 2022 · 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 ...