The First Genome-Editing Trial

David Briner is a Senior Manager in Business Development at The McDonnell Genome Institute, a leading genomics research centre located in Washington University School of Medicine, St. Louis. With extensive experience in gene-editing technologies, including zinc finger nucleases, David now supports the institute's mission to serve the scientific community through federally funded consortia and multi-omics solutions for academia and industry.

David previously led a team of scientists at MilliporeSigma in the technology transfer and commercialisation of the zinc finger nuclease platform developed by Sangamo Biosciences (now Sangamo Therapeutics). In this role David worked with government-, academic-, and industry scientists to design gene-editing reagents and develop targeting strategies for thousands of gene targets, across a wide range of therapeutic applications and species.

Dana Carroll is Distinguished Professor Emeritus of Biochemistry at the University of Utah School of Medicine, where he has been on the faculty since 1975. He received his B.A. degree from Swarthmore College and a Ph.D. from the University of California, Berkeley. He did postdoctoral research with John Paul in Glasgow, Scotland, and Donald Brown in Baltimore, USA. He served as Co-chair and Chair of the Department of Biochemistry between 1985 and 2009. He is considered a pioneer in the development and applications of genome editing with programmable nucleases. His research group was the first to show that zinc-finger nucleases stimulate targeted mutagenesis and gene replacement in living cells and whole organisms.

Carroll’s research focused on making improvements and pursuing applications of genome editing technology, using all of the current platforms - ZFNs, TALENs, and CRISPR-Cas.

For his contributions, he received the Novitski Prize from the Genetics Society of America in 2012, was elected a Fellow of the American Association for the Advancement of Science in 2013, and was awarded the H.A. Sober Lectureship by the American Society for Biochemistry and Molecular Biology in 2014. He is a member of the American Academy of Arts and Sciences and the US National Academy of Sciences.

Webpage Profile of Dana Carroll (PNAS)

Edward Rebar is an RnD executive with more than thirty years of experience building and translating gene-editing technologies. He recently served as CTO of the cell therapy company Sana Biotechnology (2020-2024), helping to establish its pipeline, launch its IPO, and transition the company to clinical stage. His research included developing Cas12b for scalable editing of T cells and IPSCs for Sana’s oncology, autoimmunity and diabetes programs.

Prior to his time at Sana, Ed was with Sangamo Therapeutics (1998-2020) where he held a number of positions including CTO. His work focused on improving the activity, specificity and precision of the company’s ZFP technologies for research and clinical use. His team also developed the first TALENs for high-efficiency genome editing.

Ed has authored more than 60 publications on the development, characterisation and application of genome-targeting technologies. He earned his PhD in biophysics and structural biology from MIT.

Matthew Porteus is the Sutardja Chuk Professor of Definitive and Curative Medicine at Stanford University, focusing on genome editing to cure diseases, especially blood disorders. He directs the Stanford Center for Definitive and Curative Medicine and co-directs the Cell and Gene Therapy GMP Facility. He works as an attending physician on the Pediatric Hematopoietic Stem Cell Transplant service at Lucile Packard Children’s Hospital where he cares for children under going bone marrow transplantation for both malignant and non-malignant diseases. His goal is to combine his research and clinical interests to develop innovative curative therapies. He was recently elected to an ASGCT leadership position (2024-2027). He served on the 2017 National Academy Study Committee of Human Genome Editing and advises WADA and NIH on emerging technologies. He founded several biotech companies and advocates for global access to transformative medicines.

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Dr. Srinivasan Chandrasegaran, now Professor Emeritus at Johns Hopkins School of Public Health, completed his early education at Sainik School (Tamil Nadu), followed by a B.Sc. in Chemistry from Tagore Arts College (Pondicherry) and an M.Sc. from Presidency College (Chennai), both affiliated to Madras University. He obtained his PhD in Chemistry from Georgetown University, Washington D.C. in 1981 and completed post-doctoral research in Biophysics at Johns Hopkins University (JHU, 1981-1984), where he then served as a tenured Professor until retiring in 2021. He continues his affiliation with Johns Hopkins as a member of its Academy.

During his retirement, Professor Chandrasegaran teaches young scientists in India and serves as Science Advisor to the small biotech firm Pondicherry Biotech Private Limited, founded by his brother, S. Gunasegaran. His research team at JHU pioneered zinc finger nucleases (ZFNs) in 1996, demonstrated ZFN-mediated gene editing in frogs (2001, in collaboration with Dana Carroll's lab at University of Utah), achieved the first synthetic yeast chromosome synthesis (2014), and completed both synthetic chromosome IX and CRISPR correction of GBA mutation in human induced pluripotent stem cells (hiPSCs) for Gaucher disease treatment (2023). His expertise spans restriction-modification enzymes, designer nucleases, genome editing, synthetic biology, hiPSCs and gene therapy.

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