Some doctors told him he'd live with the disease forever, but Khaled Alsheebani never stopped believing treatments would ...

A Falmouth teen, Jayden Wilsey, is the first to receive gene therapy to treat sickle cell at Boston Children's Hospital.

New gene therapy improves the life of 18-year-old with Sickle Cell Disease which affects 5,000 New Jersey residents living ...

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 ...

Two Italian women in their twenties on Tuesday received pioneering gene therapy for rare hereditary blood diseases in Pavia ...

The new gene therapies for sickle cell disease—including the gene-editing ... wants to go even further. "The editing therapy is a wonderful achievement, but only a small fraction of the people ...

“Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited.” Sickle ...

BEAM-101, a genetically modified cell therapy for sickle cell, is designed to turn on HbF to counteract mutated adult ...

Kiem is deputy director of the Translational Science and Therapeutics Division and holds the Stephanus Family Endowed Chair for Cell and Gene Therapy. Read on for his insights on the potential of gene ...

He started gene therapy on May 16 ... Symptoms brought on by sickle cell anemia have changed for Jayden over time, said Wilsey. In the last three years, Jayden has experience debilitating chronic ...

things like sickle cell disease, et cetera. And this approach of being able to have a gene editor given directly to someone may really bring down the cost and the complexity of gene therapy ...