A new approach to a broken heart and a future where more hearts keep beating stronger and longer.

Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

Novel CRISPR-Cas9 treatments are proving effective in reversing disease for life. But ensuring their long-term safety will ...

The recent approval of Regeneron’s Otarmeni underscores the maturation of gene therapies across a range of diseases. Here, ...

A group of Queen’s researchers is hoping to use gene therapy and new grant money to cure a handful of rare, usually fatal ...

The Greater Philadelphia Business Coalition on Health webinar series provided an in-depth framework for the advantages of including cell and gene therapy coverage in employer health plans, as well as ...

Doctors have linked a boy's tumor to a gene therapy, but say the rare risks need to be weighed against the profound benefits ...

A planned first-in-human trial aims to treat GM1 gangliosidosis in utero, possibly laying the foundation for prenatal gene ...

Circio Holding has presented preclinical data at ASGCT demonstrating that its circVec circular RNA platform achieved up to 40 ...

Regenxbio’s gene therapy for Duchenne muscular dystrophy has smashed the primary endpoint of its pivotal trial, securing a ...

A breakthrough FDA-approved treatment is changing how genetic deafness is treated.