AveXis: A Clinical-stage Gene Therapy Biotech Company

MOMENTUM IN GENE THERAPY: AVXS-101

Our proprietary gene therapy candidate, AVXS-101, is in human clinical trials for the treatment of spinal muscular atrophy (SMA) Type 1. SMA is a severe neuromuscular disease caused by a genetic defect in the SMN1 (survival motor neuron 1) gene, leading to the loss of motor neurons and resulting in progressive muscle weakness and paralysis.

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The AVXS-101 Design

We’ve developed AVXS-101 to possess four key elements of an optimal gene therapy approach to SMA.

01

Recombinant AAV9 Capsid Shell is a non-replicating adeno-associated virus capsid used to deliver a functional copy of the human SMN gene across the blood-brain barrier to cells without modifying the patient’s existing DNA
02

Human SMN Transgene is a stable, functioning SMN gene that is introduced into the cell’s nucleus
03

scAAV ITR
(Self-complementary DNA Technology) is a human SMN transgene introduced as a self-complementary double- stranded molecule. The inclusion of this technology enables rapid onset of effect
04

Continuous Promoter activates the transgene and is designed to allow for continuous and sustained SMN expression

01 Recombinant AAV9 Capsid Shell is a non-replicating adeno-associated virus capsid used to deliver a functional copy of the human SMN gene across the blood-brain barrier to cells without modifying the patient’s existing DNA

02 Human SMN Transgene is a stable, functioning SMN gene that is introduced into the cell’s nucleus

03 scAAV ITR (Self-complementary DNA Technology) is a human SMN transgene introduced as a self-complementary double- stranded molecule. The inclusion of this technology enables rapid onset of effect

04 Continuous Promoter activates the transgene and is designed to allow for continuous and sustained SMN expression

GOING AFTER THE #1 GENETIC CAUSE OF INFANT MORTALITY

Our initial focus is on helping patients and families affected by spinal muscular atrophy (SMA) Type 1—a life-threatening neuromuscular disease—with our clinical-stage, proprietary gene therapy candidate, AVXS-101.

ADDITIONAL TYPES OF SMA

We are progressively moving into other SMA subtypes, and have future plans to develop AVXS-101 to treat additional SMA types.

AND OTHER RARE, LIFE-THREATENING NEUROLOGICAL GENETIC DISEASES

We have exclusive worldwide license agreements to develop and commercialize gene therapy using the AAV9 vector to treat two rare neurological monogenic disorders: Rett syndrome (RTT) and a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene.

Get the facts about SMA