AveXis, Inc., a drug development company currently using gene therapy to address patients’ unmet needs suffering with orphan diseases such as Spinal Muscular Atrophy (SMA), has recently announced the completion of a $10 million financing set that was led by Deerfield Management and Roche Venture Fund. As part of the terms of the funding, Jonathan Leff from Deerfield and Carole Nuechterlein from Roche Venture Fund are joining the Board of Directors of AveXis.
The funding obtained from this financing round will be used to advance the development of chariSMA — AveXis’ gene therapy system — and to support the ongoing Phase I clinical trial in SMA Type 1 infants. In September 2013, the Research Institute at Nationwide Children’s Hospital received the IND approval and Fast Track designation. It is now open the recruitment and enrollment of candidates for the trial (NCT02122952).
SMA is an autosomal-recessive genetic disorder that is characterized by progressive muscle weakness, since there is decreased motor neuron loss. The disease is caused by a genetic mutation in the SMN1 gene, causing a critical defect. This gene encodes for SMN, a crucial protein for motor neurons to survive. SMA is the leading cause of genetic death among infants in the world today.
John A. Carbona, CEO of AveXis said: “We are delighted to welcome Deerfield and Roche as investors, and Jonathan and Carole to our Board, at an exciting time for AveXis. We have initiated the first-ever clinical trial with a gene replacement therapy for SMA, and are now in a position to further advance the product in development as rapidly as possible.”
Mr. Jonathan S. Leff, a Deerfield Management partner with proven experience as a director of both private and public biotechnology companies who also serves on the Spinal Muscular Atrophy Foundation Board, added: “We believe the chariSMATM program holds great promise for patients and families suffering with SMA. The initiation of clinical trials with the first gene replacement therapy for SMA is a significant accomplishment, and we are pleased to have the opportunity to help AveXis accelerate the development of treatments for SMA and other devastating genetic disorders.”
For its part, the Roche Venture Fund, which is the Venture Capital arm of leading biotechnology Roche, brings its proven drug development experience and reputation to the work that AveXis is doing to advance chariSMA. Ms. Nuechterlein has served as the head of Roche Venture Fund for the past 13 years, and previously worked at SangStat where she served as General Counsel. In addition to her role at the RVF, Ms. Nuechterlein also currently serves on the board of Lysosomal Therapeutics Inc. and led RVF’s investments in Alios, Ambit, BioFire Diagnostics, Conatus, Envoy, Nereus, and Pharmasset.