Update on Novartis Gene Therapies Friedreich’s ataxia Program

Novartis Gene Therapies,  2022 . Novartis Gene Therapies had been developing a therapeutic approach for Friedreich’s ataxia. One candidate, OAV401, is an investigational AAV9-based gene therapy. The journey to bring new therapies from the lab to patients is complex and our team works with a sense of urgency, a need for flexibility and innovation, and an ability to accept challenges in a developing field of science. In this context, we have made the difficult decision to retire development of the OAV401 gene therapy program for Friedreich’s ataxia. However, we remain fully committed to innovative approaches, such as gene therapy, and are actively pursuing multiple pathways to harness this therapeutic platform. 

Unfortunately, the totality of the pre-clinical data for OAV401 does not support a path forward for this development candidate to human clinical trials. More specifically, data generated from a recent biodistribution study revealed that adequate gene expression was not achieved in the specific cells and tissues believed to be necessary for potential therapeutic benefit in patients with Friedreich’s ataxia. 

These data are complementary, not contradictory, to the research we have presented previously. The prior data demonstrated that OAV401 reached the brain. These more detailed findings, however, indicate that AAV9 does not distribute to the specific brain regions or cells that we believe would be necessary for therapeutic benefit in patients with Friedreich’s ataxia. 

We have made this data available to enhance the knowledge in the field of AAV9 gene therapy for neurologic conditions. The title of this paper is “Intrathecal sc-AAV9-CB-GFP: Systemic Distribution Predominates Following Single-Dose Administration in Cynomolgus Macaques,” and it is posted on bioRxiv https://www.biorxiv.org/content/10.1101/2021.11.28.470258v1 ahead of peer-reviewed publication, expected later in 2022. 

We recognize this update is especially disappointing to the Friedreich’s ataxia patient community. We highly value our collaboration with the community and recognize that this partnership has significantly advanced the scientific understanding of the potential impact gene therapy could have for patients and families and has contributed to the development of potential clinical programs. We will continue to partner with the Friedreich’s ataxia community as we explore therapeutic options for this and other diseases.