Dr. Andrés Felipe Leal is a cell biologist with rich experience in drug development for leukemias, infectious diseases, and inherited disorders of metabolism. He obtained a Bachelor of Science from the Universidad de Santander (2009-2013), a Master of Science in Biochemistry from Universidad Nacional de Colombia (2015-2017), and a Ph.D. in Biological Sciences from Pontificia Universidad Javeriana (2019-2023). During his Ph.D., he demonstrated, for the first time, the potential of a CRISPR/nCas9-based genome editing strategy for treating mucopolysaccharidosis IVA using in vitro and in vivo approaches. He also worked on GM2 gangliosidoses and mucopolysaccharidosis IIIB. Since 2021, Dr. Andrés Felipe Leal has worked at Nemours Children’s Health, where he is now a Postdoctoral Fellow. Dr. Andrés F. Leal's research currently focuses on cell biology and genome editing for MPS IVA using in vitro, in vivo, and ex vivo approaches in collaboration with researchers from Colombia, India, Japan, Poland, Spain, and Türkiye.

Dr. Carlos Javier Almeciga-Diaz is a Full Professor and Director of the Institute for the Study of Inborn Errors of Metabolism at Pontificia Universidad Javeriana. He obtained a Bachelor of Pharmacy from Universidad Nacional de Colombia (1996-2001) and a Doctor of Biological Sciences from Pontificia Universidad Javeriana (2004-2009). He worked at the Pontificia Universidad Javeriana since 2009. He was a Visiting Research Scholar at the National Center for Advancing Translational Sciences (NCATS) (2017-2018). During his PhD, he carried out the first in vivo tests of gene therapy for Morquio A using adeno-associated viral vectors. As a professor and researcher at Pontificia Universidad Javeriana, he continued working in gene therapy for Morquio A using lentiviral vectors. Currently, he is exploring the use of CRISPR/Cas9 as a gene therapy tool for several inborn errors of metabolism. His research lines are: 1) Cellular and molecular bases of inborn Errors of Metabolism, 2) Diagnosis of Inborn Errors of Metabolism, and 3) Biotechnological Tools in Inborn Errors of Metabolism.

Prof. Dr. Shunji Tomatsu is the Director of Skeletal Dysplasia Center Pediatric Orthopedic Surgery, at Nemours Children's Health. He is a Professor at Thomas Jefferson University and St. Louis University and an Adjunct Professor at Shimane University and Gifu University. He is also an Affiliated Scientist at the University of Delaware. He has authored and co-authored over 250 peer-reviewed publications since 1997 (h-index; 51 in Scopus; 61 in Google Scholar). He significantly contributed to developing and publishing various therapeutic approaches targeting ERT, HSCT, SRT, and gene therapies (AAV, CRISPR Cas9, and lentiviral vectors) on MPS mouse models and established glycosaminoglycans assay system by ELISA and LC-MS/MS. As a PI or co-investigator, he has been awarded many NIH grants. Currently, he is conducting 1R01HD102545-01A1; Non-invasive functional assessment and pathogenesis of Morquio A (PI), 1R01HD104814-01A1; Enhancement of Newborn Screening Diagnostic Paradigms to Improve the Efficacy of Treatment for Krabbe Disease, Pompe Disease, and Mucopolysaccharidosis Type 1 (site PI), and 4R44HD102242-02; Identification of glycosaminoglycans for newborn screening and therapeutic monitoring of mucopolysaccharidoses (Co-I). They have published around 180 manuscripts related to Morquio A since 1991. Major studies on Morquio A have been performed by his group and his collaborators for the past 30 years, involving both basic and clinical research.