Cell and gene therapy represents groundbreaking advancements in modern medicine, offering transformative approaches to treating and potentially curing a wide range of diseases. These innovative therapies leverage the power of cellular and genetic engineering to address the root causes of illnesses, rather than merely managing symptoms.
How Cell & Gene Therapy Began
On September 15, 1990, a four-year-old girl received the first gene therapy for adenosine deaminase (ADA) deficiency, a rare immune disorder. A retroviral vector was used to insert functional copies of the ADA gene into her white blood cells, helping her immune system work better. Although this treatment wasn’t permanent and required follow-up therapies, it was a breakthrough in medicine. It showed that gene therapy could address genetic disorders at their source, bringing hope to patients and inspiring further research. This milestone paved the way for future advancements in gene therapy and genetic medicine.
Why is Cell and Gene Therapy Important?
These therapies offer hope for patients with conditions that were previously considered untreatable or incurable. By targeting the underlying mechanisms of disease, cell and gene therapy have the potential to deliver long-lasting, personalized solutions that improve quality of life and reduce healthcare costs over time.
Applications and Advancements
Chimeric Antigen Receptor (CAR)-T cell therapy has revolutionized the treatment of certain blood cancers.
Gene therapy is providing new options for conditions like spinal muscular atrophy and hemophilia.
Stem cell therapy is being explored for repairing heart tissue, treating neurodegenerative diseases, and more.
As research and technology continue to evolve, cell and gene therapy are poised to redefine the future of healthcare, offering hope and healing to millions worldwide.
The below Cell & Gene Overview contains important information including a comprehensive glossary of terms, advocacy groups and the most recent listing of approved, pipeline and uptake gene and cell therapies (as of June 2025).
2025 Cell & Gene Therapy Overview & Resources
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