Reaching New Horizons in
Genetic Therapies
Founded by pioneers in genetic research and experts in gene therapy development and commercialization.
AavantiBio is working to change the trajectory in the field of genetic medicine.
We are leveraging our deep expertise in the science of innovative gene transfer and gene editing technologies to build a sustainable platform approach that starts with Friedreich’s ataxia, or FA, a progressive genetic neuromuscular disease with high unmet medical need, with a vision to extend to a broader spectrum of genetic diseases. We are also building a scalable manufacturing process that will allow us to produce our gene therapy product candidates efficiently and with breakthrough yields.
Friedreich’s Ataxia:
A Progressive Genetic Neuromuscular Disease With High Unmet Medical Need
FA is a multisystem disease that affects muscle control and coordination with possible loss of vision and hearing and slurred speech
1, 2
The primary cause of death is cardiac complications
FA is a multisystem disease that affects muscle control and coordination with possible vision and hearing and slurred speech. It is degenerative, progressive, life-shortening and debilitating
1
1, 2
As we look toward the future, we are rapidly building a product pipeline to allow today’s unmet health needs to reach new horizons and move groundbreaking genetic therapies from the future to the present for each and every patient who suffers from devastating diseases, like FA and beyond.
A Competitive Pipeline
Our competitive pipeline focuses on utilizing AAV-mediated approaches, immunomodulation strategies, and dual routes of administration.
References:
1. National Organization for Rare Disorders. Friedreich’s Ataxia. Accessed November 4, 2020.
2. National Institute of Neurological Disorders and Stroke. Friedreich’s Ataxia Fact Sheet. Accessed December 14, 2020.