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

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 ...

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 ...

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

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

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

Apertura Gene Therapy, a biotechnology company developing next-generation AAV capsids for delivering genetic medicines, today ...

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

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 ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College of Medicine and the UMass Chan Medical School have achieved ...

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) and Regenerative Medicine Advanced Therapy (RMAT) designation to SENTI-202 for the treatment of ...