AveXis is one step closer to developing a potential gene therapy for SOD1 ALS. The gene therapy company, based in Cleveland, Ohio, announced this month it has obtained the rights to develop treatments for ALS using REGENXBIO’s gene therapy delivery vehicle. The emerging vector, derived from adeno-associated virus 9 (AAV9), is being increasingly utilized to deliver potential therapies into the CNS for neurological diseases.
The strategy is one of a growing number of potential gene therapies for SOD1 ALS that aims to reduce levels of misfolded SOD1 in the CNS and in the muscles by silencing the expression of the SOD1 gene (see May 2017 conference news). The approach is being developed by a research team led by Nationwide Children’s Hospital’s Brian Kaspar in Ohio, who is also AveXis’ chief scientific officer and scientific founder (see December 2015 conference news; Thomsen et al., 2014; Foust et al., 2013).
The delivery vehicle, known as NAV AAV9, forms the basis of AveXis’ experimental gene therapy for the motor neuron disease spinal muscular atrophy (SMA). The strategy, known as AVXS-101, is currently being evaluated at the phase 1 stage at Nationwide Children’s Hospital as a treatment for Type 1 SMA, the most severe form of the disease. The approach builds on previous studies in 2009 led by Institute of Myology’s Martine Barkats in France and Brian Kaspar in the United States, which found that AAV9 could cross the blood-brain barrier and therefore, could be used to potentially treat motor neuron diseases (see December 2008 news; Duque et al., 2009; Foust et al., 2009).
Meanwhile, Martine Barkats, in collaboration with Maria Grazia Biferi, in France is using a different approach in hopes to treat SOD1 ALS (see May 2017 conference news). The strategy, which uses a related gene therapy delivery vehicle known as AAV10, also aims to reduce motor neuron toxicity by silencing the expression of the SOD1 gene. The strategy is currently being optimized and is at the preclinical stage. The researchers are now developing a similar strategy in hopes to treat C9orf72 ALS, the most common form of the disease.
To learn more about gene therapy and its potential for ALS, check out our recent news feature: A New Potential Gene Therapy Delivers A Key Milestone.
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