AveXis avexis.com


Public lists: Rare Diseases (230)

AveXis is a clinical-stage gene therapy company developing treatments for rare and life-threatening neurological genetic diseases. The company's initial product candidate, scAAV9.CB.SMN, is in an ongoing Phase 1 clinical trial for the treatment of spinal muscular atrophy (SMA) Type 1, an autosomal-recessive genetic disorder characterized by lower motor neuron loss and progressive muscle weakness. SMA is caused by a genetic defect in the SMN1 gene that codes SMN, a protein necessary for the survi...Show all

AveXis is a clinical-stage gene therapy company developing treatments for rare and life-threatening neurological genetic diseases. The company's initial product candidate, scAAV9.CB.SMN, is in an ongoing Phase 1 clinical trial for the treatment of sp...Show all

Company (Acquired)

Phone: 847-572-8280

Fax:

2275 Half Day Road
Suite 160
Deerfield, 60015
Illinois, United States

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See more competitors data Competitors
Total Funding Date of Last Funding
AveXis $170M Apr 9, 2018
Audentes Therapeutics $212.5M Jul 20, 2016

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