In a mouse study, blood stem cells from sickle cell disease (SCD) patients modified with CRISPR/Cas9 gene editing showed reduced long-term contribution compared with cells treated using other gene-based approaches. “While all methods showed therapeutic potential, base editing and [gene therapy] provided superior outcomes over CRISPR-Cas9-mediated editing in a…
News
Emmaus Life Sciences has entered into an agreement to sell the North American rights to Endari (oral L-glutamine), its oral treatment to prevent acute complications related to sickle cell disease (SCD), to Neoimmunetech. Under the agreement, Neoimmunetech will receive an exclusive license to all rights for marketing,…
Hydroxyurea, blood transfusions, and donor blood stem cell transplants can significantly lower the risk of stroke in children and adolescents with sickle cell anemia (SCA), the most common and severe form of sickle cell disease. That’s according to a systematic review and meta-analysis of 40 studies that also highlighted…
Big Nova has made a $3 million donation to support NMDP’s efforts to help more patients, including those with sickle cell disease (SCD), access blood stem cell transplants. The philanthropic gift helped NMDP exceed its $100 million fundraising goal, a milestone the organization said will support its…
A high dose of pociredir, Fulcrum Therapeutics’ investigational oral therapy for sickle cell disease (SCD), was associated with increased fetal hemoglobin (HbF) levels and reduced markers of red blood cell destruction in early trial data. Those preliminary findings come from a Phase 1b clinical trial called PIONEER…
Risto-cel, Beam Therapeutics‘ single-dose, gene-edited cell therapy candidate for people with severe sickle cell disease (SCD), continues to demonstrate sustained therapeutic benefits for as long as 20 months, or nearly two years. That’s according to new data covering 31 adults and adolescents with SCD who received risto-cel, formerly…
The gene-editing therapy Casgevy (exagamglogene autotemcel) safely and effectively prevents vaso-occlusive crises (VOCs) in children as young as 5 years old with severe sickle cell disease (SCD), according to preliminary data from a Phase 3 clinical trial. As of the latest follow-up, all children in the…
Vascarta‘s VAS‑101, a skin gel containing curcumin — a component of the spice turmeric — was found in laboratory research to partially restore the health of mitochondria, the powerhouses of cells, in the hearts of a sickle cell disease (SCD) mouse model. “Treatment with VAS‑101 for 21 days…
Having a low household- or neighborhood-level socioeconomic status is not significantly associated with a higher risk of stroke in children with sickle cell disease (SCD). These findings are based on a secondary analysis of data from the observational DISPLACE study (NCT03621826), which was originally designed to identify barriers…
As part of a program to support children with sickle cell disease (SCD) and cancers, supplemental insurance company Aflac has released a new children’s book that aims to illustrate how empathy and compassion can help people, especially children, who are facing health problems. The book, titled “Beyond Words,” is…
Recent Posts
- Adults with SCD face high risk of stroke in their 30s and 40s, study finds
- Sickle cell doctors face higher burnout than other specialists: Study
- Disease burden heavy for SCD patients in US, despite promise of gene therapy
- The conversation we avoid: Telling children they have sickle cell disease
- New study finds distinct molecular signatures in newborns with SCT
- Blood test may improve kidney damage detection in children with SCD
- Understanding the impact of leg ulcers in sickle cell disease
- Asthma seven times more likely in SCD children than in unaffected siblings
- Sickle cell drug at high dose eases anemia symptoms in severe SCD
- Researchers urge more talk on menstrual pain in sickle cell clinics