Member Spotlight: Axovant Gene Therapies


By: Kelly Garrity, Senior Director, Membership Services and Development

New NHC member Axovant operates with a sense of urgency to develop and deliver innovative gene therapies that transform the treatment of serious neurological and neuromuscular diseases. In this critical journey, Axovant is inspired by breakthrough science and driven to challenge the status quo on behalf of patients and their families. By harnessing the transformative power of gene therapies, it aims to fill their unmet medical needs with one-time therapies that deliver lifelong benefits.

Axovant’s team of world-class scientific, clinical and product development experts, and world-renowned partners are working together to develop gene therapies that harness the latest scientific advancements. They combine cutting-edge gene transfer techniques, innovative vector design, and tailored delivery methods to optimize the safety, efficacy and durability of the clinical effects of their gene therapies. Axovant continues to tailor these therapies to precisely address the needs of the diseases and patients it serves.

The company feels the urgency in pursuing better treatment options because it knows patients are waiting for them. To that end, it approaches gene therapy development by selecting the product candidates that have great potential for transformative impact. Its pipeline employs innovative gene therapies to address devastating neurological and neuromuscular diseases by targeting and altering the disease at a fundamental, biological level.

The current pipeline of gene therapy candidates targets rare diseases such as GM1 gangliosidosis, GM2 gangliosidosis (including Tay-Sachs disease and Sandhoff disease), oculopharyngeal muscular dystrophy (OPMD), as well as well-known neurological diseases such as Parkinson’s disease.

Axovant recognizes the responsibility of developing these gene therapies with patient needs at the forefront and are committed to working to understand the challenges and hopes. Axovant’s work begins and ends with the patients, families and caregivers, and NHC provides them the opportunity to work with other members to enhance patient-focused engagement within the health care system.

For information on becoming an NHC member, visit the membership section of our website.