Gene and Cell Therapy Milestones

Indiana University School of Medicine and the Herman B Wells Center for Pediatric Research in collaboration with Riley Hospital for Children have celebrated more than three decades of groundbreaking milestones and transformative events in gene and cell therapy.

These pioneering contributions and events highlight the shared commitment to advancing health care through the power of genetic research and medicine.

• David Williams, MD, becomes the first director of the Herman B Wells Center for Pediatric Research and launches a gene therapy program.

  • Dr. Williams was recognized as the 2023 recipient of the American Society of Gene and Cell Therapy (ASGCT) Founders Award due to his decades of work at the forefront of stem cell research and clinical gene therapy. While at the Wells Center, Williams discovered the effects of fibronectin (RetroNectin). RetroNectin is now commonly used in laboratories to enhance the efficiency of gene therapy and cell therapy processes as it helps create a favorable environment for cell growth and gene transfer, ultimately improving the success of genetic engineering and cell-based therapies.

• Kenneth Cornetta, MD, and his co-investigators in the IU Bone Marrow Transplantation program initiated the first clinical use of gene transfer technology at Indiana University in order to understand the challenges of autologous transplant for acute myeloid leukemia. Over the next ten years, Drs. James Croop, Rafat Abonour, Kenneth Cornetta and former Wells Center directors Drs. David Williams and Mary Dinauer would go on to study gene therapy in the setting of autologous transplant for cancer and genetic disease.

• Between 1995 and 2009, the National Institutes of Health (NIH) funded IU's National Gene Vector Laboratory, making it only one of three institutions funded to support the manufacturing of novel gene therapies in support of human clinical trials. 

• IU School of Medicine receives an Institutional National Research Service Award (T32) from the National Heart Lung and Blood Institute to support the program, “Basic Sciences Studies on Gene Therapy of Blood Diseases.” This program has continuously been funded from 1999 to today and aims to train the next generation of scientists in gene regulation and therapy.

• The National Gene Vector Laboratory is replaced by the National Gene Vector Biorepository led by IU which is now the sole center nationally assisting investigators in meeting Food and Drug Administration (FDA) requirements associated with preclinical and clinical gene therapy.

• The Brown Center for Immunotherapy at IU School of Medicine was established in December 2016 with a $30 million gift from Indianapolis entrepreneur and IU School of Medicine alumnus Don Brown. The mission of the Brown Center is to develop new treatments and cure disease through the use of cell-based immunotherapies.

  • In 2021, Huda Salman, MD, PhD, MA, became director of the Brown Center to expand CAR-T cell therapy at IU School of Medicine.

• Roland Herzog, PhD, joins the Herman B Wells Center for Pediatric Research as the program leader for the new Gene and Cell Therapy research group. Dr. Herzog’s research interests are in gene therapy and immune tolerance for hemophilia and in AAV vectors.

• Riley Hospital for Children treats the first pediatric cancer patient in Indiana with chimeric antigen receptor (CAR) T-cell therapy for leukemia.

• Roland Herzog, PhD, receives a 5-year appointment as editor-in-chief of Molecular Therapy, the leading journal in the gene therapy field.

• Weidong Xiao, PhD, a leader in the development of adeno-associated viral (AAV) vectors, joins the Gene and Cell Therapy program at the Wells Center.

• A 4-month-old Riley Hospital for Children patient with Spinal Muscular Atrophy (SMA) receives Zolgensma, the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy.

• A multi-institute $12 million grant led by Roland Herzog, PhD, from the National Heart Lung and Blood Institute is received to fund studies that explore three major themes in a gene therapy approach that could lead to safer treatments for hemophilia.

• Moanaro Biswas, MS, PhD, receives the 2022 Career Development Award from the ASGCT for her research related to the engineering of regulatory T cells.