CLINICAL DEVELOPMENT PROGRAM

Broad Clinical Development Program Across All Types of SMA

Our Gene Therapy Products & Pipeline

Our Gene Therapy Products & Pipeline
Our Gene Therapy Products & Pipeline

The Goal of AVXS-101

AVXS-101 possesses four key elements that make it an optimal approach to the treatment of SMA.

This gene therapy is a well-suited approach for the treatment of SMA due to the monogenic nature of the disease—meaning it's caused by the deletion of, or mutations in, a single gene. We have designed it to be a one-time treatment designed to prevent further motor neuron loss and muscle degeneration caused by SMA through:

  • Delivery of a fully functional copy of a human SMN gene into target motor neuron cells
  • Production of sufficient levels of SMN protein required to improve motor neuron function
  • Rapid onset of effect in addition to sustained SMN protein expression