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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 ...
FDA approves Casgevy, 1st gene-editing therapy to treat sickle cell
Dec 8, 2023 · 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 ...
BEAM-101 gene-editing stem cell therapy shows promise in SCD
6 days ago · by Lindsey Shapiro, PhD November 7, 2024. In a Phase 1/2 clinical trial, the gene-editing stem cell therapy BEAM-101 worked as intended, and showed early signs of efficacy in people with severe sickle cell disease (SCD), according to new early data. Developer Beam Therapeutics will present the findings at the upcoming American Society of ...
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 ...
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 ...
Oral AND017 granted orphan drug status for SCD
Oct 31, 2024 · by Margarida Maia, PhD October 31, 2024. The U.S. Food and Drug Administration has granted orphan drug status to Kind Pharmaceuticals’ investigational oral small molecule AND017 to treat sickle cell disease (SCD). This designation is granted to potential treatments for rare diseases, or those affecting fewer than 200,000 people in the U.S.
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 ...
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 ...
Small molecule shows promise in lab models for treating sickle cell
Aug 6, 2024 · Small molecule SR-18292 increased fetal hemoglobin in human blood cells. Sickle cell disease is caused by mutations that lead to the production of an abnormal form of hemoglobin — the protein that red blood cells use to carry oxygen throughout the body. The abnormal hemoglobin tends to clump up inside red blood cells, deforming them into the sickle-like shape that gives the disease its name ...