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Study to compare SCD treatments for symptoms with a transplant. A hospital with the University of Rochester’s Medical Center has received a multi-million-dollar grant to conduct a study comparing the efficacy of some available treatments for sickle cell disease (SCD), helping families to make better-informed treatment decisions.
FDA approves Casgevy, 1st gene-editing therapy to treat sickle cell
2023年12月8日 · The U.S. Food and Drug Administration (FDA) has approved the gene-editing therapy exagamglogene autotemcel (exa-cel) — now known as Casgevy — for treating sickle cell disease (SCD).. The therapy from Vertex Pharmaceuticals and CRISPR Therapeutics is indicated for patients ages 12 and older who have recurrent vaso-occlusive crises (VOCs). With the decision, Casgevy has become the first cell ...
Vaso-Occlusive Crisis - Sickle Cell Disease News
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.
Lovo-cel, now Lyfgenia wins FDA approval for sickle cell disease
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 …
Most sickle cell patients VOE-free after Lyfgenia gene therapy
2023年12月12日 · 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
5 天之前 · 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.
Cyclosporin H boosts uptake of gene therapy in SCD newborn …
2 天之前 · Two gene therapies have been recently approved in the U.S. for SCD: Casgevy (exagamglogene autotemcel) and Lyfgenia (lovotibeglogene autotemcel). Both involve collecting a patient’s hematopoietic stem cells (HSCs), precursor cells that give rise to all types of blood cells, editing them to boost the production of a functional version of …
Study to compare SCD treatments for symptoms vs. a transplant
2024年11月12日 · 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 ...
SCD gene therapy lowers ratio of red blood cells with sickle shape
2023年10月19日 · By introducing these modified stem cells into the bone marrow, the therapy should promote the production of new red blood cells with high HbF levels, low HbS levels, and a reduced ability to sickle — diminishing SCD symptoms.. A research team led by scientists at Boston Children’s Hospital launched a pilot Phase 1 trial wherein a single infusion of BCH-BB694 was administered to 10 people ...
Top 10 Sickle Cell Disease Stories of 2022
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 ...