Cell and Gene Therapy
Cell & Gene Therapy
We are reimagining medicine to power breakthrough innovation in cell and gene therapy
AveXis is now Novartis Gene Therapies
Novartis Gene Therapies is dedicated to developing and commercializing gene therapies for patients and families devastated by rare and life-threatening neurological genetic diseases.
What is cell and gene therapy?
Scientific discoveries such as vaccines and antibiotics changed the course of history. Now cell and gene therapies are creating a new turning point in medicine and how we treat disease.
Delivering on the promise of cell and gene therapy for patients
New advances in cell and gene therapies offer the potential to transform medicine. They create an inflection point in our ability to treat and even potentially cure many intractable illnesses.
Key focus areas
Currently, there are three key focus areas for development of transformative cell and gene therapies at Novartis: AAV-based therapies, CAR-T cell therapies and CRISPR-based technologies.
CAR-T Cell Therapy and Beyond
Novartis is pioneering the way in the class of cell and gene treatment. This includes setting the standard in patient and caregiver support, safety and efficacy, access, and partnerships with healthcare institutions, local health authorities, and academic institutions around the world.
Expanding CAR-T manufacturing in Europe
European Medicines Agency (EMA) recently approved two European Novartis sites for commercial CAR-T manufacturing.
A new era of medicine
Cell and gene therapies could help reduce or eliminate the need for treatments that need to be taken continuously, often for life.
Collaborating to explore new gene regulation therapies
Novartis and Sangamo Therapeutics plan to pursue potential treatments for neurodevelopmental diseases.