AVXS – AveXis
About: a clinical-stage gene therapy company, engages in developing and commercializing treatments for patients suffering from rare and life-threatening neurological genetic diseases.
- The 1st ever gene therapy study for the treatment of SMA Type 1
- Designed to address the monogenetic root cause of SMA by replacing the lost or defective primary SMN1 gene with a fully functioning human SMN gene
- Accepted into the European medicine’s agency prime program that enhance support for the development of medicines which is similar to FDA.
SMA Type 1:
- Is a devastating orphan disease that results in motor neuron loss and progressive muscle weakness
- One in 10,000 live births
- SMA is the most common genetic cause of infant deaths
- Rapidly progressive and devastating
- The average lifespan of a child with SMA Type I is only 8 months of age
Phase 1 result:
- one-time infusion of AVXS-101 appears to be generally tolerated with a favorable safety profile
- indicate a potential for clinically transformative effect on the event free survival
- rapid and increase in motor function
- achievement of motor milestone never seen in the history of this disease.
Plans for 2017: To participate in a chemistry manufacturing and control Type B meeting with the FDA in May.