It’s been a banner year for the Gates Institute, which celebrated its launch in May 2023, consolidating the Gates Biomanufacturing Facility with the administrative team of the former Gates Center for Regenerative Medicine as well as the regulatory expertise of the Clinical Investigation and Regulatory Sciences (CELLS) team under one umbrella. The year has seen the launch of a new chimeric antigen receptor (CAR) T-cell clinical trial and continued enrollment of three existing CAR T-cell trials. The Gates Institute newsroom detailed these activities, as well as other areas of cell and gene therapy research in the works.
Read on for our top stories from our inaugural year, ranked by order of clicks.
5. Behind the Scenes of a CAR T-Cell Trial
Numerous experts work behind the scenes to carry out CAR T-cell trials. One such role is that of clinical research coordinator (CRC). We met with Jake Anna, MS, a CRC dedicated to lymphoma research in the hematology clinical trials unit in the Division of Hematology at CU School of Medicine. When a lymphoma patient is enrolled in a trial at UCHealth University of Colorado Hospital, Anna helps set up the trial protocol and serves as a liaison between the clinical and sponsor side of patient care. The job feeds his passions – meeting with patients in the post-treatment phase and immersing himself in the science of cell and gene therapy research.
4. Cracking the Code for Once 'Incurable' Diseases
Husband-and-wife research team Ganna Bilousova, PhD, and Igor Kogut, PhD, in collaboration with Dermatology Professor Dennis Roop, PhD, recently patented two complementary technologies to develop stem-cell created skin grafts, which could translate to cures for those with devastating skin diseases. With funding from the Epidermolysis Bullosa iPS Cell Consortium to manufacture their stem-cell created skin grafts, they’ve begun moving their technologies into the Gates Biomanufacturing Facility (GBF) to launch the production of their modified mRNA protocol.
Their next step is to move the combined gene editing and reprogramming to the GBF, which will produce clinically relevant, genetically corrected, patient-specific induced pluripotent stem cells (iPSCs), ready for differentiation.
Once the cells are differentiated into skin organoids, they are ready to use on patients for clinical trials.
3. Trial Launched to Test CAR T-Cell Therapy in Dogs Diagnosed With Solid Tumors
Dogs are like humans in many ways, sharing similar physiology as well as biological needs. Our four-legged friends are also vulnerable to some of the same diseases that we face, making the intersection of human and animal medicine an intriguing subject for study.
One new area of exploration is the use of chimeric antigen receptor (CAR) T-cell therapy, which utilizes the body’s immune system and modified white blood cells to fight cancer. Gates Institute member Steven Dow, DVM, PhD, a veterinary internist and cancer researcher at the Flint Animal Cancer Center at Colorado State University, said what excites clinicians – including veterinarians -- about the therapy is that it’s a living drug.
2. New CAR T-Cell Trial Enrolling Patients at CU Anschutz
Enrollment has begun for a new phase 1 study of chimeric antigen receptor (CAR) T-cell therapy at UCHealth University of Colorado Hospital for adults with B-cell acute lymphoblastic leukemia (B-ALL). The trial is the first launched through the newly formed Gates Institute, and it will be the fourth for which the Gates Biomanufacturing Facility produces the CAR T product for research at CU Anschutz Medical Campus.
1. Student With Rare Genetic Condition Searches for a Cure at Gates Institute
Few college seniors can say they are working in a world-renowned lab to develop therapies for a genetic disorder that plagues 1 in 5,000 people worldwide.
But Jeevan Mann is not the typical college senior.
As an intern at the Gates Institute, he’s spending his summer working with Ganna Bilousova, PhD, and a team of researchers in the lab to develop novel therapeutics for Ehlers-Danlos Syndrome (EDS)—a disease he was diagnosed with as a teenager.
April 2, 2024