The Stanford IMA unveils new Cell and Gene Therapies Lab

Therapies that leverage cell and gene engineering, like CAR-T cell therapy and CRISPR-based medicines, have the potential to transform medicine, but specialized expertise, collaboration between scientists and clinicians, and creativity are all necessary to realize their full potential. The Stanford Innovative Medicines Accelerator (IMA) recently launched its new Cell and Gene Therapies Laboratory to help bring potential new medicines closer to patients. The facility, located in the Alexandria Center for Life Science at Stanford Research Park, will allow the IMA to expand and strengthen its work developing new kinds of medicines.

“With the opening of this space, more Stanford projects will have the potential to move further on the path from discovery to medicine,” said Chaitan Khosla, director of the IMA.

Born from a partnership between Sarafan ChEM-H and Stanford Medicine, the IMA is a key part of Stanford's Vision to advance solutions to the world's most critical problems. There are a lot of innovative, transformative discoveries made at Stanford that are never developed into medicines, partly because of a systemic problem: these projects are not advanced enough to be taken seriously by companies equipped to make medicines, but they are advanced enough that expertise beyond what is traditionally found in academia is needed to get them closer to becoming medicines.  

To bring medicine-making expertise to Stanford, the IMA has recruited world-class scientists from industry. These leaders help find projects best suited to IMA support, identify the key milestones on the path from idea to medicine, and connect Stanford scientists with the resources needed to meet each goal.  In 2023, Fabrizia Urbinati joined the team as Director of Cell and Gene Therapies. Before joining the IMA, Dr. Urbinati held a leadership position within the early discovery team at Astellas Gene Therapy (formerly Audentes) that developed gene therapy products for neuromuscular disorders. Prior to that, she worked at PACT Pharma, Cincinnati Children’s Hospital, UCLA, and Children’s Hospital of Los Angeles.

Urbinati and her team have supported seven projects, with two they are actively collaborating in the lab. Until now, they have leveraged partnerships inside and outside Stanford to advance these projects. With their new lab space, they will be able to expand their capabilities.

Gene therapies work by introducing new genetic material into a cell, but to be successful, that genetic material needs to be wrapped in a coating that helps it get into the cell intact. Since viruses are naturally adept at inserting their genetic material into cells, one of the most common strategies involves using engineered versions of a small virus known as adeno-associated virus (AAVs).

Through world-class industry expertise and technology, the new lab will allow for  the fast, cost-efficient, and high-quality design and production of AAV vectors to fast track projects in the IMA pipeline. The team not only adheres to best-in-class practices, but also aims to develop new viral vectors that might exceed current capabilities.

“Our AAV production method is using the industry standard process for purification,” said Urbinati. “But we are also putting an emphasis on using AI tools to design vectors that are more effective and safer.”

The lab will also allow them to develop  organoids and other three-dimensional models that mimic different disease states. These systems will help the team test how effective their cell and gene therapies are before anything goes into human patients. 

The cell and gene therapies team is currently collaborating  with Stanford researchers on seven projects in areas like cancer, chronic pain, and cardiac diseases. The goal, according to Urbinati, will  eventually be to support a variety of cell and gene therapies modalities independent of the disease area. 

During the lab opening celebration, Khosla noted that the science is already coming to life, but that this event ushers in a new era for the IMA. “This team has already enabled some amazing advances here at Stanford,” he said. “I’m excited to see the new kinds of medicine that will emerge because of this exceptional resource at the IMA.”

Khosla is a professor of chemical engineering and of chemistry and professor by courtesy of biochemistry. He is also the founding director and an institute scholar of Sarafan ChEM-H and a member of Stanford Bio-X, Maternal & Child Health Research Institute (MCHRI), Stanford Medicine Children’s Health Center for IBD and Celiac Disease, Stanford Cancer Institute, and Wu Tsai Neurosciences Institute.