Health systems learn why cell and gene therapy success depends on workflows, staffing, and finances—not just science—and how to build them.

Despite innovative new research, many cell and gene therapies do not make it all the way to the patients. Researchers and ...

For patients with rare and devastating neurological disorders, treatment options can seem few and far between. However, gene therapy research, led by Dr. Russell Lonser and Dr. Brad Elder, is hoping ...

The UAE has launched two gene therapy projects targeting the root causes of autism and rare brain disorders, marking a ...

Henry Ford Health has treated the first Michigan patient outside clinical trials with Roctavian, the newly approved gene ...

A cell-specific gene therapy has been shown to silence pain encoding neurons in mice, offering a potential alternative to ...

Researchers engineered lipid nanoparticles to deliver a full CFTR gene into human airway cells, restoring near-normal ...

Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells.

UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene ...

What Is Waskyra, and Why Does It Matter? Waskyra (etuvetidigene autotemcel) is a new gene therapy approved to treat Wiskott-Aldrich syndrome (WAS), a rare inherited condition that affects the immune ...

FDA RMAT boosts inhaled gene therapy KB707, delivering IL‑2/IL‑12 to lung tumors; early trial shows responses with manageable ...