Novel Therapies: Cell and Gene Therapies

"What truly novel therapies in the earliest stages of discovery and development do you believe will have a significant impact in the future?"

Cell and gene therapies represent the leading edge of medicine and have the potential to impact many patients’ lives. The advent of CRISPR technology catapulted these emerging therapies into the world, with the first FDA IND approval for clinical trials occurring very recently. This targeted gene-editing technology also enabled the next generation of CAR-T therapy development, which has been shown to drastically improve the quality of life for patients with several different types of lymphoma. By harnessing the potential of genetic engineering, these cutting-edge therapies can be used for tumor-targeting cancer therapies, neurodegenerative disorders, orphan diseases, and so much more.