Teen Beats Deadly Leukemia With Life-Saving Gene-Edited Cell Therapy

A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell ...

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 ...

California boy, 3, receives first-ever gene therapy for his rare disease: ‘It’s just so exciting’

A California toddler is the first person in the world to receive gene therapy to treat his devastating disease.
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 ...

Novartis receives FDA approval for Itvisma(R), the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA)

ITVISMA is a prescription gene therapy used to treat adults and children 2 years of age and older with spinal muscular atrophy (SMA). ITVISMA is given as a one-time intrathecal injection. What is the ...
The Scientist

A Deep Dive into Viral Vectors for Gene Therapy

Viral vectors introduce genes and gene editing sequences into cells, and are a popular platform for gene therapy due to their safety and efficacy features. Adeno-associated viral (AAV) vectors in ...
FierceBiotech

Arbor sails into rocky gene editing waters with $74M for rare liver disease therapy

As other gene editing programs fold or get sold, Arbor Biotechnologies has secured $73.9 million to advance its lead liver-targeting gene therapy into human trials. The series C was led by Arch ...
Becker's Hospital Review

1st gene therapy delivered directly into brain approved

The FDA granted accelerated approved to the first gene therapy directly administered into the brain on Nov. 14. The therapy, Kebilidi, was approved to treat adults and children with aromatic L-amino ...
The Scientist

Moving Gene Editing Advances Into the Clinic

Gene therapy has long promised to provide a solution for genetic blood disorders, but delivering on that promise has proven complex due to challenges including difficulties delivering the editing ...