Sickle Cell Disease News Home | Sickle Cell Disease News
Nov 1, 2024 · Editas’ new gene-editing strategy for SCD shows promise in mice. Editas Medicine has announced proof-of-concept data for a new gene-editing approach for sickle cell disease (SCD) that does not involve a stem cell transplant. The strategy instead makes use of targeted lipid nanoparticles, or LNPs, which are tiny vesicles made of fatty molecules.
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 other sickle cell ...
Vaso-Occlusive Crisis - Sickle Cell Disease News
Jan 4, 2022 · A vaso-occlusive crisis, or VOC, occurs when sickled red blood cells block blood flow to the point that tissues become deprived of oxygen. This in turn sets in motion an inflammatory response as the body tries to rectify the problem. The result is substantial pain, which can affect any part of the body, but most commonly occurs in the back ...
Bluebird set for Lyfgenia’s launch after ‘remarkable’ approvals
Dec 21, 2023 · Bluebird Bio’s Lyfgenia (lovotibeglogene autotemcel) — formerly lovo-cel — was cleared for SCD patients, ages 12 and older, with a history of vaso-occlusive events (VOEs). It was approved along with Casgevy (exagamglogene autotemcel), or exa-cel, from Vertex Pharmaceuticals and CRISPR Therapeutics. “To have two transformative therapies approved for this community, after nearly a ...
Gene Therapy – Sickle Cell Disease News
Four trials (NCT02186418, NCT03282656, NCT02247843, NCT02140554) are testing the efficacy and safety of gene therapy to replace the mutated HBB gene with a healthy HBB gene.These Phase 2 trials are recruiting both children and adults in the United States and Jamaica. Three trials (NCT02193191, NCT02989701, NCT03226691) are investigating the use of Mozobil (plerixafor) in patients with sickle ...
Top 10 Sickle Cell Disease Stories of 2022
Dec 29, 2022 · Here we’ve compiled a list of the 10 most-read articles published on our site in 2022, with a brief description of each. No. 10 — “ Trial of Cell Therapy to Raise HbF Levels Showing Potential, But Sanofi Leaving ”. SAR445136 is a gene-edited cell therapy designed to boost the production of fetal hemoglobin (HbF) in a patient’s blood ...
Study to compare SCD treatments for symptoms vs. a transplant
1 day ago · 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 ...
New regimen finds bone marrow transplant an SCD 'curative option'
Apr 30, 2024 · An allogeneic stem cell transplant uses donor stem cells to replace a patient’s blood-forming stem cells in the bone marrow. The transplanted cells then start to make new, healthy red blood cells. Allogeneic stem cell transplants offer a cure for SCD, but their use can be limited by difficulties in finding matched donors and the aggressiveness of myeloablative conditioning regimens.
Sickle Cell’s Prognosis Taught Me to Live with Purpose
Jan 29, 2020 · There are people with sickle cell living well past the average life expectancy. The oldest person currently living with sickle cell, Asiata Onikoyi-Laguda, is 94.I understand that this is rare, but I know that to get to that point, I must do what I …
SCD gene therapy lowers ratio of red blood cells with sickle shape
Oct 19, 2023 · The number of red blood cells that produce the abnormal hemoglobin S (HbS), which characterizes sickle cell disease (SCD), is reduced by the experimental gene therapy BCH-BB694, as is their tendency to take a sickle-like shape. That’s according to single-cell data from seven patients in an ongoing Phase 1 clinical trial (NCT03282656), whose ...