Inc, AveXis. A biotechnology company focused on the use of gene therapy to transform the lives of patients with severe genetic and orphan diseases, such as Spinal Muscular Atrophy (SMA), today announced the completion of a $10 million funding round led by Deerfield Management and Roche Venture Fund. In conjunction with this funding round, Jonathan Leff of Deerfield and Carole Nuechterlein of the Roche Venture Fund will join the Board of Directors of the Company.
The funding proceeds will be used to further develop the company’s gene therapy product, charismaTM, including additional support for the current Phase I clinical trial in SMA Type 1 infants. The National Children’s Hospital Research Institute received IND approval and Fast Track designation in September 2013. The trial is currently open for enrolment and recruitment of candidates (NCT02122952)
About spinal muscle atrophy
Spinal muscular atrophy (SMA) is 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 for SMN, a protein that is necessary for the survival of motor neurons. SMA kills more infants than any other genetic disease in the world today.
Based in Dallas, Texas, AveXis is a clinical-stage gene therapy platform company that establishes unique industry alliances to develop innovative treatments for people with unmet medical needs. Spinal muscular atrophy (SMA) is the company’s primary focus. For more information on AveXis, please visit