Living Therapies, also known as cellular therapies, are among our most sophisticated disease treatments developed to date.  The potential power of manipulating cells for therapeutic use had been recognized for decades, but until recently the technical challenges for generating these agents and then managing the clinical side-effects had severely limited their development and use. However, in the past 12 years, the field has exploded with scientific and technological advancements that have allowed scientists to more precisely design genetically-modified cells with improved safety and efficacy and have allowed doctors to better manage their clinical delivery with improved outcomes.  As a result, it is possible to correct the genetic defects in certain cell types and manipulate immune cells to attack and treat diseases such as some cancers and autoimmune diseases.  These early successes are helping drive the design of more sophisticated therapies for more challenging indications.

UCSF has long been at the forefront of cellular therapies.  The first chimeric antigen receptor T cells (CAR-T) were initially described by UCSF’s Art Weiss, MD, PhD, over 30 years ago.  Shinya Yamanaka, MD, PhD, of UCSF won the Nobel prize in 2012 for his groundbreaking work in reprogramming mature cells to become pluripotent stem cells.  These pluripotent stem cells, also called iPSC cells, are foundational technology driving novel cellular therapy development across a range of complex diseases.  More recently, our faculty have been leaders in modifying signaling circuits, improving methodologies, and harnessing the power of CRISPR/Cas9 technologies to correct or alter cells for disease treatment.  Wendell Lim, PhD, is developing “smarter CAR T-cells” by designing them to recognize multiple features of tumors and using that information to initiate more specific and effective anti-tumor responses. Alex Marson, MD, PhD, is developing the next generation CRISPR tools that are vastly improving our ability to modify and genetically map human T-cells.  

The vision of the Living Therapeutics Initiative (LTI) is to harness the vast scientific talent and clinical expertise at UCSF. The mission is to advance future promising cellular therapies from early discovery through proof-of-concept studies and into clinical trial evaluation to provide meaningful therapeutic solutions and inform future innovations.