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…

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…

All 18 adults with severe sickle cell disease (SCD) treated with the gene-editing therapy renizgamglogene autogedtemcel — known as reni-cel — remained free of vaso-occlusive events, or VOEs, for up to nearly two years, according to new interim data from the Phase 1/2/3 RUBY clinical trial. The therapy, being…

The Bahrain Oncology Center now is among the first in the world to offer Casgevy (exagamglogene autotemcel), an approved gene-editing therapy, to people with sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT), a related blood disorder. That milestone, announced in a release by the country’s regulatory authority,…

The European Commission has granted conditional approval to the gene-editing therapy Casgevy (exagamglogene autotemcel) for patients ages 12 and older with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT), two blood disorders. Eligible patients include those with SCD who experience recurrent vaso-occlusive crises (VOCs) and…

Saudi Arabia’s regulatory authorities have approved Casgevy (exagamglogene autotemcel), a one-time gene-editing therapy, to treat sickle cell disease (SCD) in adults and adolescents, ages 12 and older. The approval by the Saudi Food and Drug Authority (SFDA), the country’s equivalent to the U.S. Food and Drug Administration, also…

Throughout 2023, Sickle Cell Disease News brought readers coverage of the latest clinical research and scientific breakthroughs related to sickle cell disease (SCD). Here are the year’s top 10 most-read articles, each with a brief description. We hope to remain a dependable resource for the SCD community in…

A European Medicines Agency committee has issued a positive opinion on the gene-editing therapy Casgevy (exagamglogene autotemcel) in treating sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The Committee for Medicinal Products for Human Use (CHMP) recommendation will be considered by the European Commission in making a final…

Health regulators in the Kingdom of Bahrain have approved the gene-editing therapy Casgevy (exagamglogene autotemcel) to treat people with sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). This decision makes Bahrain, a small island nation in the Persian Gulf, the second country to approve Casgevy. The therapy was…