Advanced therapies represent a paradigm shift, not only in how diseases can be treated, but in how medicines can be made. Unlike traditional therapeutics with established manufacturing processes, gene and other breakthrough therapies require innovative processes and specialized equipment and personnel.
As one of the first companies to bring these processes to life, Novartis Gene Therapies sees manufacturing as having a critical role to play in the future viability and success of gene therapies.
Innovative manufacturing as a business-critical step in gene therapy development.
Over the past decade, the first gene therapies have come to market, representing a scientific breakthrough with the potential to revolutionize the treatment of genetic diseases. But making these treatments available to patients who need them has also required breakthroughs in manufacturing.
In fact, when Novartis Gene Therapies began developing one of the first gene therapies to be approved by the U.S. Food and Drug Administration, these processes did not yet exist. With no model to follow, the company began from scratch, designing innovative but complex processes with 24/7 hands-on attention from specially trained technicians under highly specific conditions, using precision equipment and advanced facilities.
As companies have refined processes, one thing has become clear — investment in high-capacity manufacturing is critical for a gene therapy to be successful and reach patients.
Despite this, only a handful of companies have put in place the facilities necessary to adequately build out capacity — understandable, since investment can be difficult to make without a close-to-market product.
Since acquiring AveXis (now Novartis Gene Therapies), Novartis has invested significantly in gene therapy manufacturing to build out facilities to produce material for global supply. This investment in manufacturing — even for a small rare disease population — is critical to ensuring regulatory standards around the world are met in order to fulfill global supply chain needs.
As the field continues to grow and evolve, where does manufacturing go next and what does it mean for the future of gene therapy?
How changes in gene therapy manufacturing will advance the field.
First, companies will begin investing in manufacturing earlier in the development process. Ideally, companies should have commercial-grade processes and product ready at the time of clinical development. A big hurdle for gene therapy is how to use data from early-stage trials because it was generated with a pre-commercial product. Because of this, many companies have been denied a path to bioequivalence between early stage and late stage and an early focus on manufacturing to advance a product is now nearly non-negotiable.
Second, engineering advances will drive manufacturing innovation. The combination of science and engineering is unique in gene therapy, and in many ways, under-explored. Progress in engineering will not be cookie-cutter. It will require balancing dependencies — including disease, route of administration and scale. Those advances are what will ultimately allow companies to scale gene therapy in an industrial way, achieving a widely shared goal of bringing life-changing gene therapy to more patients with unmet medical needs worldwide.
The future of gene therapy manufacturing is bright.
Gene therapy is a breakthrough area of medicine and so are the manufacturing processes that bring them to life. As the field continues to evolve, the industry is learning and refining. Novartis Gene Therapies is optimistic that with innovative manufacturing working hand-in-hand with groundbreaking science, gene therapies will continue to drive transformation for patients and society into the future.
Novartis Gene Therapies is re-imagining medicine to transform the lives of people living with rare genetic diseases, and has played a leading role in the journey the field has taken to date. Visit the company’s website to learn more about how Novartis Gene Therapies is poised to leverage these trends into the future.
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