Scientists have made tremendous progress over the past decade in understanding how to modulate genes and use them now to make transformative medicines. The first wave of these transformative medicines has recently launched or is on pace to reach patients soon. However, the field is still in its infancy.
Assembling the right technologies is key to opening up therapeutic opportunities ranging from rare diseases caused by a single genetic mutation to acquired diseases that affect millions.
In our work, three aspirations guide us:
Developing the ability to repair any cell in the body (“gene therapy”)
Developing the ability to replace any cell in the body (“cell therapy”)
Developing the technologies and processes to ensure access to our therapies