When Vijay Sankaran was an MD-PhD student at Harvard Medical School in the mid-2000s, one of his first clinical encounters ...

Vertex Pharma's £1.65 million ($2.05 million) gene-editing therapy for sickle cell disease (SCD), Casgevy, has been given the green light for use by the NHS in England under a managed access ...

ROCHESTER, N.Y. – Researchers at Golisano Children’s Hospital have received a multi-million-dollar grant to lead a ...

“Gene therapy for hemophilia A with the use of lentiviral vector-transduced autologous HSCs resulted in stable factor VIII ...

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

“Cell and gene therapy manufacturing, supply chains and delivery are all very complex,” Anita Ralli, chair of the Collective and associate director of government affairs at Gilead ...

Recent headlines in the U.K. lauded the approval of a new sickle cell cure using gene-editing technology. Columnist Mary ...

All patients with sickle cell disease who underwent imaging were invited to have concurrent neuropsychometric testing done. A self-selected subset of 98 patients with sickle cell disease received ...

4, 2025 — Research has shown that a potential new targeted therapy ... of gene regulation during blastoid formation and ... New Drug Tested to Reduce Side Effect of 'Half-Matched' Stem Cell ...

Scientists said yesterday that they may be able to use gene therapy to tackle some types of cancer in humans. The U.S. researchers made their claim after they were able to prevent cancer in mice ...

Ensuring that cell and ... a new Cell and Gene Therapy (CGT) Access Model that the federal Centers for Medicare & Medicaid Services (CMS) Innovation Center is launching. The initial phase of the ...

Perhaps the biggest news in the genomics world in the last couple of years was the approval of the first CRISPR-based gene therapy, which was designed to treat sickle cell disease and beta-thalassemia ...