News

A first patient has been enrolled in BEACON, a Phase 1/2 clinical trial into the safety and efficacy of BEAM-101, Beam Therapeutics’ transplant-based gene-editing cell therapy for sickle cell disease (SCD). Recruitment of adults with severe SCD, ages 18–35, continues at Boston Children’s Hospital in Massachusetts and…

A Phase 1b clinical trial testing the safety and pharmacological properties of the investigational oral medication FTX-6058 in people with sickle cell disease (SCD) is expected to continue enrolling patients through 2023, according to Fulcrum Therapeutics, the therapy’s developer. “We believe we are well positioned, with a…

Recruitment is now underway in BEACON, a Phase 1/2 U.S. trial testing BEAM-101, a transplant-based gene-editing cell therapy for sickle cell disease (SCD). The interventional study, in which all participants will be given a single dose of the experimental treatment, is expected to enroll up to 15 adults with…

Researchers have developed a method to detect and measure large unintended DNA modifications created by CRISPR-Cas9 gene-editing methods currently being investigated as treatments for sickle cell disease (SCD). High levels of unintended gene modifications that occurred at selected target sites demonstrate the need for a more careful evaluation of…

The risk of death from end-stage kidney disease (ESKD) has generally declined in the U.S. over the last 20 years — but not as much for people with sickle cell disease (SCD). Patients with the genetic disorder still have a higher risk of mortality than those without SCD,…

Television host KiKi Shepard’s 4th Annual Celebrity Golf Classic, taking place Monday, Nov. 14, aims to raise awareness of sickle cell disease (SCD) — and funding for programs and services at the nonprofit she founded, called the KIS Foundation. The golfing event, organized by Shepard and select corporate sponsors,…

A gene editing platform developed by Cellectis called TALEN was able to restore healthy hemoglobin production in preclinical models of sickle cell disease (SCD). That’s according to data presented by Arianna Moiani, PhD, senior scientist and team leader of Innovation Gene Therapy at Cellectis, at the 29th Congress of…

PerkinElmer has donated to an initiative that would extend newborn screening for sickle cell disease (SCD) in Ghana. The goal, which is expected to be achieved by 2030, is to boost screening from its current 4% to 50% of all babies born in the West African country. The…

Activation of a protein critical for the body’s response to low oxygen leads to increased levels of fetal hemoglobin in adults — a finding that could offer new therapeutic avenues for people with sickle cell disease (SCD) or beta-thalassemia, a study says. In cell cultures of red blood cells…

Blood flow and oxygen extraction fraction — a measure of how well oxygen is used by tissues — in the brain was improved among children with sickle cell disease (SCD) who underwent a hematopoietic stem cell transplant (HSCT), a small study showed. According to researchers, these findings may…