A Brief History of Advanced Therapeutics
August 02, 2024
Early biologics and ATMPs have undergone a transformative journey marked by significant milestones and challenges. Despite hurdles such as product costs, market fluctuations, and safety issues, ATMP developers are figuring out how to overcome these challenges with greater success.
In 1972, Theodore Friedmann and Richard Roblin authored a paper titled "Gene therapy for human genetic disease?" in Science where they referenced Stanfield Rogers' 1970 proposition, suggesting that healthy DNA could potentially substitute defective DNA in individuals affected by genetic disorders.
In the article, Friedmann and Roblin discuss the potential of gene therapy to improve certain genetic diseases in the future. The authors argue that research into gene therapy modalities should continue and expand. However, they also cautioned against applications of gene therapy in humans for several reasons involving their limited understanding of how genes interacted within human cells, the lack of understanding for how specific genetic defects caused diseases, and the uncertainty regarding the short-term and long-term side effects of gene therapy. They suggested the need for clear, ethical, and scientific guidelines to ensure gene therapy was used only when it's beneficial and not prematurely applied. Additionally, the paper referred to studies demonstrating the future possibility of genetically modifying mammalian cells, which would overcome a major technical hurdle of gene therapy.
It wasn't until 45 years after the publication of this paper that the FDA approved the first gene therapy product called voretigene neparvovec-rzyl (Luxturna) from Spark Therapeutics for the treatment of a rare inherited form of vision loss. The approval of Luxturna in 2017 marked a pivotal moment, highlighting the immense potential of gene therapy to address rare diseases and revolutionize healthcare.
As the ATMP industry continues to evolve, there has beena shift toward a new normal where therapies unlock substantial value for patients, society, and the healthcare system. With continued advancements and regulatory frameworks, cell and gene therapies hold promise for addressing unmet medical needs and shaping the future of medicine.
ATMP development indeed has revolutionized modern medicine. Interested in learning more? Download our latest whitepaper, “Enabling Acceleration & Risk Mitigation in ATMP & Beyond”.