Cell line development plays a pivotal role in the manufacturing of therapeutic proteins, vaccines and other biologics.

The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in ...

Previous research has shown that voretigene neparvovec administered subretinally early in childhood for RPE65-mediated ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three-to-six times each week.

The majority are in the very early preclinical stage of development, but applications for regulatory approval have been ...

Qure is planning soon to start enrolling patients in a second dose group as part of a clinical trial testing AMT-162 for SOD1 ...