News

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 first patient has been enrolled in a Phase 1 clinical trial evaluating the safety and tolerability of Rigel Pharmaceuticals’ fostamatinib — a medication approved in the U.S. for treating an autoimmune condition — in people with sickle cell disease (SCD). The Phase 1 study (NCT05904093) is…

People with sickle cell disease (SCD) have brains that appear to be more than a decade older than their actual age. That’s according to a recent study that also found that patients with older-looking brains had poorer cognitive function. “Our study explains how a chronic illness … can cause…

Immvention Therapeutix has partnered with Novo Nordisk to develop oral treatments for people with sickle cell disease (SCD) and other chronic conditions. The U.S.-based Immvention called the deal “a strategic collaboration and license agreement” in a company press release. “We are excited to partner with Novo…

A hematopoietic stem cell transplant (HCT) may be a feasible treatment option for adolescents and adults with severe sickle cell disease (SCD), according to a U.S. study. Two-year survival rates were similar among patients who had an available donor for HCT and among those who did not, although they were…

Fulcrum Therapeutics is planning to report, by midyear, new data from the ongoing PIONEER Phase 1b clinical trial that’s testing its oral therapy candidate pociredir (FTX-6058) in people with sickle cell disease (SCD), according to a new corporate presentation. That will include data from the study’s third-highest…

Texas Children’s Global HOPE and Baylor College of Medicine Global Health, collaborating with the independent charity Bristol Myers Squibb Foundation, have announced a pioneering program to combat sickle cell disease (SCD) in Sub-Saharan Africa. Launching first in Tanzania and Uganda, this program is intended to improve the survival and…

Orna Therapeutics is partnering with Vertex Pharmaceuticals to develop gene-editing therapies for people with sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). The three-year collaboration will leverage Orna’s proprietary lipid nanoparticle (LNP) delivery system. LNPs are tiny vesicles made up of fatty molecules that can be used…

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…