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Jan 15, 2025 · 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 …
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 …
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 …
Stem cell transplant may be feasible for young SCD patients
2 days ago · 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 …
Gates Foundation to invest in Tessera SCD gene-editing treatment
Jan 9, 2025 · “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, …
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 …
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 …
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 …
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 …
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 …