Novartis Gene Therapies

Biotechnology company specializing in gene therapies

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Novartis Gene Therapies is a biotechnology company that focuses on the development and commercialization of gene therapies. It is a subsidiary of Novartis, a global healthcare company based in Switzerland. Novartis Gene Therapies is dedicated to transforming the lives of patients with rare and life-threatening genetic diseases through innovative gene therapy solutions.

History

Novartis Gene Therapies was originally founded as AveXis, Inc. in 2010. The company was established to develop gene therapy treatments for rare neurological genetic disorders. In 2018, AveXis was acquired by Novartis for $8.7 billion, and it became a part of Novartis' broader strategy to expand its capabilities in advanced therapies.

Research and Development

Novartis Gene Therapies is at the forefront of gene therapy research, focusing on the development of treatments for rare genetic disorders. The company's flagship product is Zolgensma, a gene therapy for spinal muscular atrophy (SMA), a severe neuromuscular disease caused by a mutation in the SMN1 gene. Zolgensma is designed to deliver a functional copy of the SMN1 gene to motor neuron cells, thereby addressing the root cause of the disease.

The company employs advanced AAV vector technology to deliver therapeutic genes to target cells. This approach allows for precise targeting and long-lasting expression of the therapeutic gene, offering the potential for a one-time treatment.

Products

• Zolgensma (onasemnogene abeparvovec) is the first and only gene therapy approved for the treatment of spinal muscular atrophy in pediatric patients. It is administered as a one-time intravenous infusion and has shown significant improvements in motor function and survival in clinical trials.

Manufacturing and Facilities

Novartis Gene Therapies operates state-of-the-art manufacturing facilities to support the production of its gene therapy products. These facilities are equipped with advanced technologies to ensure the quality and scalability of gene therapy manufacturing. The company is committed to maintaining high standards of GMP compliance to ensure the safety and efficacy of its therapies.

Future Directions

Novartis Gene Therapies continues to explore new therapeutic areas and expand its pipeline of gene therapy candidates. The company is investing in research to develop treatments for other rare genetic disorders and is actively collaborating with academic institutions and industry partners to advance the field of gene therapy.

Related pages

• Gene therapy
• Spinal muscular atrophy
• Adeno-associated virus
• Novartis