"Regulatory flexibility must be tailored for cell and gene therapies," commented FDA Commissioner Marty Makary. "These are ...

The U.S. Food and Drug Administration (FDA) announced a new process called “Rare Disease Evidence Principles” (RDEP), under which eligible drugs and biologics for ultra-rare diseases caused by known ...

A large genetic screen has revealed how stem cells transform into brain cells, exposing hundreds of genes that make this ...

An international research team identified hundreds of genes essential for the development of brain cells, including one gene ...

With the FDA expecting to approve 10–20 Cell and Gene Therapies (CGTs) annually by 2025, it is evident that the field will continue to hold immense commercial and medical opportunities. To harness ...

The cell and gene therapy sector has never lacked attention, but all too often, the narrative swings from one extreme to ...

A genome-wide CRISPR study maps 331 genes essential for early brain development, identifying PEDS1 as a new ...

Gene therapies can correct diseases at their genetic core, but their success hinges on viral vector manufacturing. As gene therapy technology becomes more advanced, producing viral vectors becomes ...

The team of Professor Christof Niehrs at the Institute of Molecular Biology (IMB) in Mainz, Germany, has discovered that a DNA modification called 5-formylcytosine (5fC) functions as an activating ...