News

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…

In a $5.4 billion deal, Pfizer fully acquired Global Blood Therapeutics (GBT), bolstering its decades-long standing in the field of rare blood disorders, particularly regarding sickle cell disease (SCD). With the newly completed acquisition — plans were announced in August — Pfizer gains GBT’s therapy portfolio and pipeline.

The Sickle Cell Disease Association of America (SCDAA), in collaboration with Forma Therapeutics, is launching a campaign to raise awareness and boost knowledge about sickle cell disease (SCD). The campaign, called “Believe it!”, will be promoted on television and radio channels, encouraging people to go through the website…

Through a collaboration with Emmaus Life Sciences, Kier “Junior” Spates — of the nationally syndicated radio program “The Steve Harvey Morning Show” — will share his experiences with sickle cell disease (SCD) and the Emmaus treatment Endari (L-glutamine). The radio show features host Harvey and a team that…

Vertex Pharmaceuticals and CRISPR Therapeutics are planning a November launch for a biologics license application (BLA) for their gene-editing therapy exagamglogene autotemcel — known as exa-cel — seeking its approval for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia. The U.S. Food and Drug Administration (FDA)…

Long-term treatment with Ferriprox (deferiprone) safely and effectively reduced iron load in children and adults with blood transfusion-induced iron overload due to sickle cell disease (SCD) or other anemias. That is according to three years of data from a one-year Phase 4 clinical trial, called FIRST (NCT02041299), and its…

Children with sickle cell disease (SCD) are at a high risk of developing severe symptoms after infection with the SARS-CoV-2 Omicron variant, one of the most recent variants of the virus that causes COVID-19, according to a new French study. In fact, more than 85% of children infected with…