Steve Bryson, PhD,  science writer—

A new oral treatment, mitapivat, for sickle cell disease (SCD) significantly boosted hemoglobin levels in patients over a year, achieving one of the primary goals of a Phase 3 clinical trial. In the RISE UP trial (NCT05031780), the medication helped patients 16 and older experience a key increase…

Researchers have identified a new method to reactivate fetal hemoglobin, without the need for gene-editing therapy, that has the potential to treat more people with sickle cell disease (SCD) at a lower cost and with fewer risks, as reported in a new preclinical study. While investigating the underlying mechanism…

The first sickle cell disease (SCD) patient has been enrolled in a Phase 1b clinical trial evaluating iadademstat, Oryzon Genomics‘ investigational oral therapy designed to boost fetal hemoglobin (HbF). “SCD represents a major unmet medical need,” Carlos Buesa, PhD, Oryzon’s CEO, said in a company press release.

A Phase 1b clinical trial has exceeded the enrollment target for the group of adults with sickle cell disease (SCD) who will receive the highest dose of Fulcrum Therapeutics‘ experimental oral therapy pociredir. Previously reported data on the 12 mg dose group of the ongoing Phase 1b…

The European Medicines Agency (EMA) has confirmed the continuing suspension of marketing authorization for Pfizer’s sickle cell disease (SCD) treatment Oxbryta (voxelotor) — withdrawn from the market last year due to safety concerns — following a committee’s recommendation that “concluded that the benefit-risk balance … is no longer favourable.” The EMA’s…

Changes in the gut microbiome, the group of bacteria and other microbes living in the digestive tract, may drive chronic pain in people with sickle cell disease (SCD), a new study reveals. In a study using mice and human tissues, a team of U.S. researchers investigated how such changes,…

Researchers have used an existing noninvasive optical tool that leverages the way light interacts with tissue to detect disease in the small blood vessels in the brain, according to a study. This neurological complication of sickle cell disease (SCD) is linked to cognitive impairment. Used in a variety of…

Pfizer’s investigational therapy inclacumab failed to outperform a placebo in reducing the rate of vaso-occlusive crises — episodes marked by painful inflammatory responses — in people with sickle cell disease (SCD), according to new data from the Phase 3 THRIVE-131 study. “We recognize this news is disappointing…

The Centers for Medicare & Medicaid Services (CMS) has announced that 33 states, along with the District of Columbia and Puerto Rico, have agreed to participate in the Cell and Gene Therapy (CGT) Access Model, which is intended to boost access to cell and gene therapies for people with…

Acute chest syndrome (ACS), a form of lung injury associated with sickle cell disease (SCD), is driven by the abnormal activation of a part of the immune system known as the complement cascade, a study found. The researchers found elevated complement activation in SCD patients, especially in those…