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.

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 ...
Morning Overview on MSNOpinion

How genetic engineering could reshape medicine and human life

Genetic engineering is moving from the lab bench into clinics, farms, and even family planning decisions, promising to change ...

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 ...
BioSpace

FDA Minutes Confirm Challenging Path Ahead for UniQure’s Huntington’s Gene Therapy

Investor optimism has waned as final minutes from uniQure’s pre-BLA meeting with the FDA convey that data from the company’s ...
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

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 ...

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

Ad hoc announcement pursuant to Art. 53 LR Itvisma (onasemnogene abeparvovec-brve) demonstrated improved motor function and stabilization in patients regardless of SMA treatment history in Phase III ...