Novartis Gene Therapies, formerly AveXis, is an integral part of Novartis focused on developing and commercializing novel gene therapies for patients suffering from debilitating rare neurological genetic diseases. Their pioneering work includes Zolgensma®, a one-time treatment for spinal muscular atrophy (SMA). Novartis Gene Therapies leverages cutting-edge AAV-based technology and has significant manufacturing and research capabilities. In late 2023, Novartis announced the further integration of its gene therapy operations into its broader global BioPharma organization to enhance innovation, development, and delivery of these transformative treatments. While it operates as a key component of Novartis, its origins and specialized focus on gene replacement and AAV technologies remain central to its mission.
Key operational hub for gene therapy research, process development, and strategic operations within Novartis.
Advanced laboratories and facilities dedicated to AAV-based gene therapy research and development, supporting a pipeline of potential treatments.
As part of Novartis, the culture emphasizes innovation, collaboration, scientific rigor, and a strong patient-first orientation, particularly critical in the pioneering field of gene therapy.
Historically the headquarters of AveXis, this location was instrumental in the development of Zolgensma® and remains a center of excellence for Novartis in the gene therapy field, driving innovation and manufacturing expertise.
As part of Novartis, Novartis Gene Therapies's reach is global. Novartis has operations in approximately 140 countries worldwide. The gene therapy products, like Zolgensma, are approved in numerous countries across North America, Europe, Asia, and Latin America. The global presence supports clinical trials, regulatory affairs, manufacturing, and commercialization of gene therapies, aiming to bring these transformative treatments to patients globally. Manufacturing and R&D sites are strategically located in the US and Europe.
2275 Half Day Road, Suite 200
Bannockburn
Illinois
USA
Address: 1 T.W. Alexander Drive, Durham, NC 27703, USA
Serves as a primary gene therapy production and supply chain hub for the United States and global markets, with advanced capabilities in viral vector manufacturing.
Address: 4000 Nelson Rd, Longmont, CO 80503, USA
Contributes to the global supply of gene therapies, focusing on scalable and efficient manufacturing processes.
Address: 10675 John Jay Hopkins Dr, San Diego, CA 92121, USA (Novartis Institutes for BioMedical Research - NIBR)
Focuses on cutting-edge biomedical research and discovery, feeding into Novartis's global drug development pipeline, including innovative genetic medicines.
Address: 1950 USG Drive, Libertyville, IL 60048, USA
Supports the overall manufacturing capacity for Novartis's gene therapy portfolio.
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As of April 2025, Novartis Gene Therapies' leadership includes:
Novartis Gene Therapies has been backed by several prominent investors over the years, including:
Novartis has been integrating its Gene Therapies unit into the broader organization, leading to some leadership transitions and role alignments within the parent company. Key appointments at the Novartis corporate level also significantly impact the strategy and development of gene therapies.
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Novartis, including its Gene Therapies division, commonly uses a standard corporate email format. The most prevalent format is combining the employee's first name and last name, separated by a dot.
firstname.lastname@novartis.com
Format
john.doe@novartis.com
Example
90%
Success rate
Novartis Press Release • March 25, 2024
Novartis announced new data presentations at the 2024 American Academy of Neurology (AAN) Annual Meeting reinforcing the transformative and sustained benefit of Zolgensma (onasemnogene abeparvovec), its gene therapy for spinal muscular atrophy (SMA). The data highlighted age-appropriate development in children treated presymptomatically and long-term durability....more
Endpoints News • October 5, 2023
Novartis announced it is integrating its standalone gene therapy unit into its broader research and development organization. This move aims to streamline operations and accelerate the development of new gene therapies by leveraging the full scale and expertise of Novartis's global R&D engine....more
Novartis Press Release • July 18, 2023
In its Q2 2023 financial results, Novartis reported continued strong performance for Zolgensma, with sales of USD 319 million. The company emphasized its commitment to expanding access to the gene therapy globally and advancing its pipeline of gene therapy candidates....more
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