CLINICAL DEVELOPMENT PROGRAM FOR SMA

Broad Clinical Development Program Studying AVXS-101 Across All Types of SMA

Broad Clinical Development Program Studying AVXS-101 Across All Types of SMA
*Final design of REACH to be informed by STRONG

Our Gene Therapy Pipeline

Our Gene Therapy Pipeline
*The brand name ZOLGENSMA® (onasemnogene abeparvovec-xioi) has been provisionally approved by the FDA for the investigational product AVXS-101, but the product itself has not received marketing authorization or Biologics License Application approval from any regulatory authorities

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 muscle degeneration caused by SMA through:

  • Delivery of a fully functional copy of 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
 

4 Key Elements of AVXS-101

Discover the components that make AVXS-101 an optimal gene therapy approach.

01 Recombinant AAV (Capsid Shell)

A non-replicating adeno-associated virus (AAV) capsid is used to deliver a fully functional copy of human SMN gene to the patient's own cells without modifying the existing DNA of the patient.

Unlike many other capsids, the AAV9 capsid utilized in AVXS-101 crosses the blood-brain barrier—a tight protective barrier that regulates the passage of substances between the bloodstream and the brain—allowing the option of intravenous administration. AAV9 has been observed in preclinical studies to efficiently target motor neuron cells when delivered via either intrathecal or intravenous administration.

The DNA contained within the capsid shell is engineered to contain the other three critical elements of AVXS-101.