Why Cell and Gene Therapy?

Traditional approaches to treating patients using medicines and surgery have improved the lives of people throughout the world but there are many diseases, perhaps even most of them, for which these traditional approaches modulate the course of the disease but do not provide a cure. This shortcoming is particularly apparent for treating monogenic diseases, diseases caused by mutations in single genes that a person is born with. It is estimated that there are greater than 6,000 such diseases affecting over 350 million people worldwide.

Cells and genetically engineered cells have fundamentally different properties than medicines and surgery—they are “living drugs” that can heal and replace damaged tissues or diseased organs. These properties mean that they have the potential to provide curative therapies to a range of diseases that currently have no cure.

Translational Medicine

Translating medical discoveries into treatments available to patients is, for many reasons, a very arduous task, sometimes dubbed “The Translational Valley of Death.” What the CDCM aims to do is bridge the divide between discovery in the laboratory and translation to patients in clinical trials. It does so by creating a seamless workflow between multiple groups within Stanford University working on discovery, manufacturing, industry alliances and clinical trials in the field of cell and gene therapies. Gene Therapies allow for correcting the typographical errors in the genome that cause diseases. Either cells are modified ex vivo in a laboratory or they are modified in vivo using vectors to carry the genes into the body. Cell and gene therapies can also enhance the regeneration of diseased tissues by replacing damaged tissue with healthy cells or engineered cells that either directly replace the diseased tissue, enhance the recovery of the remaining healthy cells, or provide factors that reverse the damage in the diseased cells.

State of the Art CGT Technologies

Cell and Gene Therapies encompass a range of technologies that are starting to be used successfully in patients. The CDCM will focus on four core platforms, each covering a range of Therapeutic Areas:

The CDCM has already attracted more than two dozen programs covering each of the four platforms and several therapies are already in clinical stage while almost half of the others are expected to pass the critical regulatory steps of pre-IND or IND (Investigational New Drug approval) with the US FDA in 2018/19. Within two years, nearly all of them will be in clinical stage, demonstrating efficacy and safety in actual patients. Meanwhile, the CDCM expects to attract new Principal Investigators developing innovative CGT, expanding our capacity to other incurable diseases.