South Florida teen among first to receive gene therapy to treat sickle cell anemia

Major is one of the first pediatric patients in Florida to receive gene therapy for sickle cell disease since the FDA ...

South Florida teen hopeful after new gene therapy treatment for sickle cell anemia

Caden Major was born with sickle cell disease – an inherited blood disorder affecting red blood cells. Now at 19 years old, ...
Medscape

Gene Therapy for SCD, TDT Promising in Younger Kids, Too

In the big picture, matched sibling donor transplants remain the standard of care, Boelens said. But gene therapy is ...

Exa-cel gene therapy may off effective cure for beta-thalassemia and sickle cell disease in children younger than 12

Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an ...

Ayrmid Reports Promising New Real-World Data on Motixafortide for Stem Cell Mobilization in Sickle Cell Disease

Key Highlights Motixafortide effectively mobilized sufficient hematopoietic stem cells (HSCs) in patients with sickle cell disease enabling accelerated access to gene therapies 90% (9 of 10) of ...
Yahoo

Cape Cod teen to receive human gene therapy for sickle cell. He is one of the first.

Fifteen-year-old Jayden Wilsey of Falmouth is the first person at Boston Children's Hospital to receive human gene therapy for sickle cell disease, following approval of the therapy in 2024 by the U.S ...
The American Journal of Managed Care

Gene Therapy Meaningfully Improves Quality of Life in Severe Sickle Cell Disease

Exagamglogene autotemcel (exa-cel) significantly improved HRQOL in severe SCD patients, eliminating severe VOCs and hospitalizations in most cases. The CLIMB SCD-121 trial showed sustained ...

CT joins national push to open access to million-dollar sickle cell gene therapies through Medicaid

About 100,000 people in the U.S. have sickle cell disease, a condition that primarily affects people of color, the CDC says.
Medscape

NICE Backs CRISPR Gene Therapy for Severe Sickle Cell Disease

The National Institute for Health and Care Excellence (NICE) has issued final draft guidance recommending a CRISPR gene editing-based treatment for severe sickle cell disease (SCD). The regulator said ...

Study offers real-world data on commercial implementation of gene therapies for sickle cell disease, beta thalassemia

A study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers ...
Managed Healthcare Executive

Gene therapy in the real world: High priced, a long process and still relatively few patients | ASH 2025

The chief medical officer of Genetix Biotherapeutics, formerly known as Bluebird bio, discusses timelines and operational ...
WRBL

Columbus doctors turn to gene therapy for patients in continued fight against sickle cell disease

COLUMBUS, Ga. (WRBL) — September is Sickle Cell Awareness Month, and millions of people worldwide are living with the disease. But advances in treatment are being made. WRBL News 3 anchor Cameron ...