A central nervous system (CNS) tumor has prompted the FDA to place clinical holds on two Regenxbio gene therapies, including ...

A five-year-old patient dosed with RGX-111 has developed a brain tumour four years after being dosed with the gene therapy.

When Genenta Science listed on the Nasdaq at the tail end of 2021, the Milan-based biotech was squarely focused on its pipeline of cell-based gene therapies. | Four years after listing on the Nasdaq, ...

An AAV vector genome integration event associated with PLAG1 overexpression was identified in a preliminary analysis of the resected tumor.

It’s now possible to treat inherited blood diseases, such as sickle cell disease, with gene editing. Blood stem cells are extracted from the patient, modified, and infused back into their bone ...

Fractyl Health is developing a shot that would program the body to make more of the GLP-1 hormone naturally, a risky bet that it can provide a longer-lasting benefit than blockbuster weight-loss drugs ...

Fred Hutchinson researchers are testing a simple idea with surprisingly big potential: a special type of foam that makes gene therapy more efficient.

For those born with certain types of congenital deafness, the cochlear implant has been a positive and enabling technology.

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. A single one-time gene therapy could free ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.

Opportunities include the growing demand for scalable solutions to support the burgeoning cell and gene therapy pipeline, ...