Statement from Novartis Gene Therapies: OAV301 program for familial ALS caused by SOD1 mutation

lesturnerFoundation Blog, Research News

Please see summary below from Novartis Gene Therapies on the OAV301 program for familial ALS caused by the SOD1 mutation:

Novartis Gene Therapies (formerly AveXis) deeply appreciates the collaboration and support of the amyotrophic lateral sclerosis (ALS) community. Our ALS advocacy partners are strong allies who have deepened our understanding of this disease as we advance promising science with transformative potential for ALS. It is in this spirit that we would like to transparently share a very recent decision that impacts our OAV301 program for familial ALS caused by SOD1 mutation.

We believe in the promise of gene therapy to address patients with SOD1 mutation. However, at the present time, multiple factors have converged on a need to re-assess our portfolio priorities. In that context, we have made the difficult decision to stop development of OAV301, and to focus our resources on OAV201 for Rett syndrome and OAV401 for Friedreich’s ataxia, as well as on our growing pipeline of early undisclosed gene therapies. We continue to believe that our preclinical safety and efficacy data for our SOD1 gene therapy would translate well into clinical studies, and we are currently considering strategic options for this asset and are hopeful that we can find a partner willing to move the program forward. We pledge our full support to exploring all options to ensure the successful transition if an option is identified. This was a very difficult decision for us as we are relentlessly driven by our desire to seek life-changing treatments for patients and families living with rare genetic disease.

Although this news must come as a disappointment to the ALS community, Novartis’ commitment to the community continues. We have several active programs for other genetic and sporadic forms of ALS and we look forward to a continued and rich partnership with the ALS community.

We appreciate the partnership and support of many strong ALS advocates and patients as we continue to leverage the power of innovative science to advance solutions for this devastating disease.


The Novartis Gene Therapies Team