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

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

Researchers developed an epigenetic editing approach to silence the PCSK9 gene, achieving long-term cholesterol reduction ...

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

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

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

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

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

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

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 five-year trial saw patients who received a single injection of Lumevoq sustaining clinical recovery in a rare condition ...