Marisa Wexler, MS,  senior science writer—

A sickle cell disease (SCD) patient in Bahrain received the gene-editing therapy Casgevy (exagamglogene autotemcel), becoming the first patient outside the U.S. to be treated with the drug. Bahrain approved Casgevy in late 2023, becoming the first country in the Middle East to approve the therapy and the…

Children’s Hospital Los Angeles (CHLA) is now offering Casgevy (exagamglogene autotemcel), an approved gene-editing therapy for sickle cell disease (SCD). “This is a transformative time for Children’s Hospital Los Angeles and the pediatric community we serve,” Alan S. Wayne, MD, pediatrician-in-chief at CHLA, said in a press…

The Bill and Melinda Gates Foundation is investing up to $50 million in Tessera Therapeutics to support the development of a new gene-editing medicine for sickle cell disease (SCD), the company said. “We look forward to advancing a genetic medicine that can potentially reshape the treatment landscape for…

A newborn screening program in Catalonia improved outcomes for children with sickle cell disease (SCD), with patients being diagnosed earlier and having lower rates of major disease complications. “The data presented justify screening in countries where it has not yet been implemented, as it reduces patient morbidity and…

Most people with sickle cell disease (SCD) who received the gene-editing therapy Casgevy (exagamglogene autotemcel) in clinical trials have been free from vaso-occlusive crises (VOCs) over several years of follow-up, according to new data. Vertex Pharmaceuticals, which developed Casgevy alongside CRISPR Therapeutics, shared the findings…

The vast majority of people with sickle cell disease (SCD) who received the now-approved gene therapy Lyfgenia (lovotibeglogene autotemcel) in clinical trials have been free from vaso-occlusive events — simply called VOEs — in the years following treatment. That’s according to new data that Lyfgenia’s developer Bluebird…

Kanglin Biotechnology has raised $20 million in financing to advance the development of KL003, an experimental gene therapy for sickle cell disease (SCD) and beta thalassemia. The China-based company plans to launch a pivotal Phase 2 trial next year to test KL003 in people with beta thalassemia, a…

Two centers in California are enrolling nine people with severe sickle cell disease (SCD) for a clinical trial of a new gene editing therapy called CRISPR_SCD001. The Phase 1/2 trial (NCT04774536) is open to adolescents and adults, ages 12 through 35, who in the two years before enrollment…

The Clinton Health Access Initiative (CHAI) is leading a project that aims to expand access to diagnostic tests and treatments for sickle cell disease (SCD). The project is being funded by a three-year, $8 million grant from Open Philanthropy, an advised fund of the Silicon Valley Community Foundation, the…

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. They are used to deliver…