Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their ...

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Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...

Dravet syndrome and other developmental epileptic encephalopathies are rare but devastating conditions that cause a host of ...

Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...

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

Dravet syndrome and other developmental epileptic encephalopathies are rare but devastating conditions that cause a host of symptoms in children, including seizures, intellectual disability, and even ...

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

Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...

Regenxbio's gene therapy faces market challenges as Goldman Sachs lowers its rating and price target, citing competition and ...

Cell and gene therapies can be life-changing for patients with certain conditions, but the process of receiving them poses barriers for patients and caregivers that require multistakeholder solutions, ...

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