AveXis

AveXis is a biotechnology company that develops gene therapies to treat rare, genetic neurological disorders. Their approach involves delivering a healthy copy of a faulty gene directly into a patient's cells using a virus vector, aiming to replace or repair the defective gene's function. This technique offers potential for long-term or even curative treatment by addressing the root cause of genetic diseases. AveXis focuses on conditions like spinal muscular atrophy and other serious neurological conditions, seeking innovative solutions to improve patients' quality of life.