Gene therapy has been successfully used to treat a number of diseases, including immune deficiencies, hereditary blindness, ...

Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big ...

A first-of-its-kind inhalable gene therapy for lung cancer that genetically modifies people’s lung cells has been fast-tracked towards potential approval after promising clinical trial results. “Very ...

Two dose-limiting toxicities have been recorded in uniQure’s trial evaluating an investigational AAV gene therapy designed to treat the rare condition Fabry disease. In the phase 1/2 study, two ...

GM1 gangliosidosis, caused by biallelic variants in GLB1, results from deficiency of lysosomal β-galactosidase, which degrades GM1 ganglioside. This fatal neurodegenerative disease currently has no ...

A UC San Diego–led team has discovered that restoring a key cardiac protein called connexin‑43 in a mouse model can dramatically improve heart function and extend survival in several inherited forms ...

When Harith Rajagopalan considers the millions of patients who have taken a GLP-1 drug to treat diabetes or obesity, he sees a revolution that is failing to realize its promise. “We are literally ...

2025 was a rollercoaster year for the cell and gene therapy field. Stories such as the successful treatment of ‘Baby KJ’ using a bespoke gene editor developed through a public–private partnership, and ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...

Animation has always enjoyed a devoted following, but 2025’s releases revealed that its audience is bigger and broader than most of us thought. In a tough year for most live-action releases, one of ...

PHILADELPHIA (WPVI) -- A miracle baby who was treated with a pioneering gene-therapy at Children's Hospital of Philadelphia has reached a major milestone as he took his first steps. KJ Muldoon was the ...