Dr. David Rawlings , Director, Center for Immunity and Immunotherapies, Seattle Children’s Research Institute

As the annual meeting of the American Society of Gene & Cell Therapy (ASGCT) approaches, I am looking forward to joining the cell and gene therapy research community to share findings and to find new avenues of collaboration. 

I’m proud to share that 17 of my Seattle Children’s immunology research colleagues, representing my lab, as well as the labs of Drs. Richard James , Carol Miao , and Bruce Torbett , are also attending this year’s conference. Of those, 10 Seattle Children’s researchers are sharing study results ; four are making oral abstract presentations and six will share poster presentations.

Over the last decade, many Seattle Children’s researchers within the Center for Immunity and Immunotherapies have been chosen by the ASGCT organizers to present our cutting-edge work. The competition for such presentation opportunities is fierce, and our continued success highlights the leadership role of our center in this field.

This year, our researchers’ abstracts highlight rapid progress in expanding novel cell therapy platforms invented at Seattle Children’s Research Institute, including in-vivo application of gene therapy in hemophilia, B cell therapeutics for immune and genetic diseases, and engineered regulatory T cells to restore immune tolerance.

Amazingly, there are now more than 500 single gene disorders that lead to immunodeficiency and/or autoimmunity in pediatric and adult patients, making this patient population likely the most complex within any pediatric subspecialty. 

Our clinical and research programs have been global leaders in identifying and treating these rare diseases. Seattle Children’s oversees a genetic diagnostic platform that allows for simultaneous screening of more than 700 immune-related genes. This, along with other specialized testing, permits us to rapidly determine the genetic basis of immune disease in most patients referred to Seattle Children’s.

Importantly, each of these genetic diseases requires a disease-specific approach that ranges from antiviral or antibacterial therapies, antibody replacement, targeted biologics, stem cell transplant, or gene therapy. As part of this care, the Seattle Children’s Research Institute’s Program for Cell and Gene Therapy, in collaboration with the Seattle Children’s stem cell transplant teams, coordinates multiple protocols for stem cell transplantation and operates Phase I/II lentiviral gene therapy clinical trials for these non‐malignant immunologic disorders.

We continue to expand our reach: Seattle Children’s is developing new Phase 1/2 clinical trials using lentiviral-based gene replacement strategies for two primary immunodeficiency disorders and our team has treated four infant patients in a first-in-human clinical trial for X-linked SCID .

In parallel, Seattle Children’s is a worldwide leader in the development and application of gene-editing technologies. This work has lead new cell therapeutic platforms designed to treat genetic and autoimmune diseases, including our novel technologies to make engineered regulatory T cells and engineered B cells. By editing primary B cells to generate drug-secreting B cells and gene editing hematopoietic stem cells, we’re moving closer developing therapies for genetic blood disorders. These significant achievements have led to several biotech partnerships to accelerate the expansion of novel technology and sequence data.

The commitment, curiosity, and perseverance of my Children’s colleagues inspires me, as does our shared goal of working toward a day when kids won’t need lifelong treatments. Instead, they’ll get a single infusion of cells that addresses a disease’s cause and, ideally, cures it forever. It’s team science at its best, and I look forward to meeting more of my fellow researchers and celebrating our common purposes at #ASGCT2024.