Member Type: Emerging Biotech/Pharmaceutical Companies

AveXis is passionately committed to moving gene therapies into the clinical setting for patients and families devastated by rare and life threatening neurological genetic diseases. AveXis is advancing cutting-edge science in order to treat rare and life-threatening genetic diseases — starting with a clinical-stage, proprietary gene therapy candidate, AVXS-101, for spinal muscular atrophy type 1 (SMA-1). This will be followed by expansion in to SMA-2 and building a pipeline of gene therapy treatments beyond SMA.

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