A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain ...

FDA accepts Rocket Pharmaceuticals' resubmission for Kresladi gene therapy in severe LAD-I, with a decision expected by March ...

Oncologists' experience with CGT has increased, with more patients treated and earlier lines of therapy being utilized.

The global cell and gene therapy market is undergoing rapid expansion, driven by the surge in regulatory approvals and continual advancements in therapeutic technologies. The market is anticipated to ...

In a small pivotal trial of an investigational gene therapy, most cases of congenital profound deafness from genetic variants ...

The move aims to regulate cell and gene therapies, and xenografts or animal-to-human transplants. Such therapies are ...

In a note to investors on Sunday, analysts at Leerink Partners called these findings “compelling,” adding that they indicate ...

The Big Pharma’s window to take up exclusive rights to Taysha’s lead asset, dubbed TSHA-102, has officially closed, according ...

AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced the publication in peer-reviewed journal Nature Medicine of 12-month ...

Despite announcing a broad pivot to siRNA earlier this year, Sarepta is following through with an investigational gene ...

Researchers announce breakthrough results for AMT-130, the first disease-modifying treatment for Huntington's disease, with ...