Virginijus Šikšnys holds a chair of distinguished professor at Vilnius University and serves as chairman of the board of Life Science Center of Vilnius University. His groundbreaking research on CRISPR-Cas has had a major impact on the gene-editing field, paving the way for the development of novel tools for genome-editing applications.

Virginijus studied chemistry at Vilnius University and obtained his Ph.D. from Moscow State University before returning to Vilnius where he moved through different research ranks at the Institute of Applied Enzymology/ Institute of Biotechnology. In 1994 he stayed as a visiting scientist in the lab of Prof. Robert Huber at Max Planck Institute of Biochemistry in Germany.

Virginijus is a member of Academia Europaea, EMBO, Lithuania Academy of Sciences and Norwegian Academy of Sciences and Letters. His work has been recently recognised with numerous awards and prizes including the Kavli Prize.

Lab webpage

Jimi Olaghere is a CASGEVY recipient who has developed a keen interest in the advancements of cell and gene therapies, since his transformative participation in the groundbreaking clinical trial.

Jimi is passionate about the positive impact that these therapies can have on patients in need and has become a staunch advocate for increased accessibility of gene therapies for sickle cell disease patients worldwide.

At CRISPRMED25, Jimi will share his story about living with sickle cell disease, before and after treatment with the recently approved CRISPR therapy CASGEVY.

Follow Jimi on LinkedIn

Dr. Alvin Luk is the Co-founder, CEO, and Head of Medical at HuidaGene Therapeutics.

Alvin brings 30+ years of global drug development experience, particularly in rare diseases. Alvin has held several executive roles at biotech/biopharma companies, including Shanghai Henlius, Spark Therapeutics (acquired by Roche), Biogen and others. Spark Therapeutics’ LUXTURNA^® was approved by the U.S. FDA in 2017 as the first AAV gene therapy when he served as the Head of Clinical Research and Operations.

Alvin has a reputation for navigating the regulatory landscape resulting in over 250 cumulative total regulatory submissions globally. Between 2006-2009, he also served as an industrial member of the US-FDA Rare Disease Clinical Design Committee, focusing on trial design and shortening drug development timelines. Today he has participated in the development of 21 approved products.

He is the co-author of more than 100 book chapters and scientific papers in highly-regarded journals, including the New England Journal of Medicine, Nature Medicine, Nature, Cell, and Science.

Dr. Luk holds an MBA from Harvard, received his Ph.D. in Neuroscience, and is certified in clinical research from the University of California-San Francisco Medical School.

Company website

Dr. Annarita Miccio is the Director of the Laboratory of Chromatin and Gene Regulation during Development at the Imagine Institute (Paris Cite’ University, INSERM Unit UMR1163) in Paris. Her primary research focuses on the transcriptional control of haematopoiesis and developing therapies for β-haemoglobinopathies.

During her PhD, she created a lentiviral vector used in an early β-thalassemia clinical trial. As a post-doc and assistant professor, she specialised in gene regulation during erythroid development and gene therapy for haematopoietic disorders. Appointed Lab Director in 2014, Dr. Miccio continued her research on transcriptional regulation in stem cells, advancing therapies for β-haemoglobinopathies. She optimized lentiviral vectors for a sickle cell disease clinical trial and developed CRISPR/Cas9 strategies.

Dr. Miccio has authored over 50 publications and 15 patents, coordinated numerous projects, and received several awards, including two Sanofi Innovation awards and the ASGCT Outstanding New Investigator Award.

LinkedIn Google Scholar Webpage

Dr. Madison is Chief Scientific Officer, Gene Therapy at Poseida Therapeutics, after joining the Company in 2019 and most recently serving as Vice President, Genetic Engineering.

At Poseida, he has played a leading role in advancing allogeneic CAR-T programs and developing key platforms, including the Cas-CLOVER nuclease and site-specific super piggyBac for applications in cell and gene therapies. Prior to Poseida,

Dr. Madison was a tenure-track Assistant Professor of Medicine and a National Institutes of Health (NIH)-funded investigator at Washington University in St. Louis, Missouri, and before that was a Postdoctoral Fellow in genetics at the University of Pennsylvania. Earlier in his career, he led R&D efforts at Transposagen Biopharmaceuticals as the Vice President of Research.

Dr. Madison holds a B.A. in Biology from Washington University and a Ph.D. in Cell and Molecular Biology from the University of Michigan.

LinkedIn Company Webpage

Dr. Emma Wang is the Chief Technology Officer at YolTech Therapeutics. Her focus is on advancing the non-viral delivery platform to support in vivo gene therapy development through research in novel lipid discovery, product development, process development, drug product characterisation as well as GMP manufacture. Prior to joining YolTech, Emma held research positions of growing responsibilities at Providence Therapeutics, Beam Therapeutics and Tessera Therapeutics. Emma holds a Ph.D. in Chemistry as a member of Prof. Katharina Landfester's group from Max Planck Institute for Polymer Research in Germany, a M.Eng and B.Eng in Chemical Engineering from McGill University in Canada.

Company website

Gerald Schwank is an Associate Professor at the Medical Faculty of the University of Zurich, specialized in translational genome editing. Since his appointment in 2019, his team has been integrating directed protein evolution and machine learning to further enhance the efficiency and precision of genome editing tools for therapeutic applications.

Gerald Schwank earned his PhD in Molecular Biology from the University of Zurich and completed postdoctoral training at the Hubrecht Institute, where he pioneered genome-editing strategies in patient-derived organoids.

In 2015, he established his own laboratory at ETH Zurich, where his team developed CRISPR screening methods to study cancer and approaches for in vivo gene correction in the liver. His ongoing research is supported by organizations focused on combating rare diseases, the Swiss National Science Foundation (SNSF), and the European Research Council (ERC).

LinkedIn Google Scholar Lab webpage

Professor Guillermo Montoya is Research Director and Group Leader at the Protein Structure and Function program at Novo Nordisk Foundation Center for Protein Research (CPR) at University of Copenhagen. His research aims to understand basic cellular mechanisms at the atomic level, and he firmly believes that the detailed unravelling of these mechanisms will be essential for future biomedical research.

The approach of the Montoya Group is to use advanced methodology, combining X-ray crystallography and cryo-electron microscopy with cell biology to study the structure and function of macromolecules involved in cell cycle progression, genome integrity and its manipulation.

LinkedIn ResearchGate Lab webpage

Jacob Corn is the Professor of Genome Biology at the ETH Zurich. His research aims to better understand and treat disease through next-generation genome-editing technologies.

Jacob’s career has bridged academia and industry, working in therapeutic areas that include infectious disease, neurobiology, and oncology. His research takes a multidisciplinary approach, combining cellular biochemistry, functional genomics, computational biology, bioengineering, and biophysics. Jacob’s graduate studies at the University of California, Berkeley explored how cells replicate and protect their genomes. His postdoctoral work at the University of Washington computationally designed protein inhibitors from scratch.

Jacob began his independent research career as a group leader at Genentech, where his lab discovered biological mechanisms for challenging therapeutic targets. Jacob then moved back to academia as the founding Scientific Director of the Innovative Genomics Institute and faculty at UC Berkeley.

In his spare time, Jacob enjoys trips in the mountains and rock climbing.

Webpage LinkedIn Publications

Jacob Giehm Mikkelsen is a Professor at the Department of Biomedicine, Aarhus University. Part of his current research focuses on providing a better understanding of virus-based protein delivery and cell-vehicle interactions to develop enveloped CRISPR/Cas delivery technologies further and bring these new tools closer to the clinic.

Jacob is a trained retrovirologist with >25 years of experience in gene transfer and virus-based gene delivery technologies with focus on gene therapy, genetic engineering, and more recently genome editing using CRISPR. Over the last 10 years, Jacob’s lab has fronted the development of lentivirus-derived nanoparticles for delivery of genome-modifying proteins and ribonucleoprotein complexes.

LinkedIn ORCID profile Lab webpage

Julian Grünewald is a physician-scientist, Emmy Noether group leader, and Assistant Professor of Gene Editing at the Technical University of Munich (TUM).

His lab at the Department of Cardiology and at the TUM Center for Organoid Systems is focused on engineering new CRISPR technologies for research and therapeutic applications in cardiovascular medicine.

After medical school and training as a resident of internal medicine, Julian trained as a postdoc in the laboratory of J. Keith Joung at Massachusetts General Hospital and Harvard Medical School. He started his lab at TUM in 2022.

LinkedIn Google Scholar Webpage

Kiana Aran is a biomedical entrepreneur and a bioengineering professor at the UCSD Jacobs School of Engineering as well as school of medicine. She was also the co-founder and CSO of Cardeabio (M&A, Paragraf UK) a biotechnology company mass-producing 2D electronics for healthcare applications.

She is also the co-founder of CRISPR QC, a company that develops analytical tools for CRISPR. She has served as a consultant for the gates foundation and has been recently elected as a member of new voices for the national academies of science, engineering and medicine. Her efforts have been recognized within the scientific community by the Clinical OMICs 10 under 40 Award, Athena Pinnacle Award in Life Sciences, NSF Career award, Nature Research Awards for Inspiring Women in Science: Scientific Achievement Category’s Overall Winner in 2021 and Distinguished Engineering Medal of Excellence from Rutgers University in 2022, Inc. USA top 200 female founders in 2023, Distinguished Engineer of the year San Diego Engineering Council in 2024 and was recently elected to national Academy of Inventors as senior member.

Google Scholar Webpage

Matthew Porteus MD, PhD is the Sutardja Chuk Professor of Definitive and Curative Medicine and a Professor in the Department of Pediatrics, Institute of Stem Cell Biology and Regenerative Medicine and Maternal-Child Health Research Institute at Stanford. His primary research focus is on developing genome editing as an approach to cure disease, particularly those of the blood (most notably sickle cell disease) but also of other organ systems as well.

He is the Director of the Stanford Center for Definitive and Curative Medicine and the co-Executive Director of the Stanford Cell and Gene Therapy GMP Facility called the Laboratory for Cell and Gene Medicine. He was recently elected to a leadership position for the ASGCT (2024-2027).

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 served on the 2017 National Academy Study Committee of Human Genome Editing and currently serves on the Scientific Advisory Board for WADA on Cell and Gene Doping and the NIH NexTRAC advisory committee evaluating the emergence of new technologies. He has been a scientific founder of CRISPR Tx and a founder of Graphite Bio and then Kamau Tx and serves on several SAB’s. He is a strong advocate for assuring that the next generation of transformative medicines reaches the global community in partnership with those communities.

LinkedIn Google Scholar Lab webpage

Raffaella is a Group Leader at SR-TIGET, Milan and Associate Professor of Pathology at IUSS, Pavia. Her research is focused on optimising gene therapies based on haematopoietic stem and progenitor cells.

LinkedIn ResearchGate Webpage

Dr. Suk See De Ravin is a Senior Research Physician and Chief of the Gene Therapy Development Unit at National Institute of Allergy and Infectious Diseases. Her research is focused towards translating better and safer gene therapy approaches for treatment of X-linked severe combined immunodeficiency and chronic granulomatous disease, applying similar approaches to treatment of broader spectrum of inborn errors of immunity.

She obtained her medical degree and paediatrics subspecialty training in Australia. Upon completion of her Ph.D. at Sydney Children’s Hospital/University of New South Wales, Australia, she continued her training at the Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, with Dr. Harry L. Malech.

ResearchGate Webpage

Dr. Peranteau is an attending surgeon in the Division of Pediatric General, Thoracic and Fetal Surgery and an associate professor of Surgery at CHOP. His research interest is in foetal development as it relates to the potential understanding of and foetal intervention for congenital anomalies. He has amassed significant experience evaluating in utero haematopoietic stem cell transplantation in the murine and canine model. These studies provide interesting information and address basic questions on the development of the foetal haematopoietic niche.

One of the main goals of Dr. Peranteau's research is a better understanding of the barriers to successful allogeneic in utero bone marrow transplantation. Allogeneic in utero bone marrow transplantation would allow for non-myeloablative unmatched transplants without the need for immunosuppression and thus holds significant clinical promise for the treatment of haemoglobinopathies, such as sickle cell disease.

LinkedIn Google Scholar CHOP webpage

Xavier Duportet PhD is Co-founder and CEO of Eligo Biosciences, France

LinkedIn Google Scholar

Alessia is a Group Leader and Associate Professor in Gene Therapy at University College London's Great Ormond Street Institute of Child Health.

Google Scholar ORCIID LinkedIn Lab Webpage

Anna is a Professor and Vice Director of the Department of Cellular, Computational and Integrative Biology at University of Trento, where she leads the Laboratory of Molecular Virology. She is also co-founder of Alia Therapeutics.

Google Scholar ORCIID LinkedIn Lab Webpage

Ayal is a Principal Investigator at The Mina and Everard Goodman Faculty of Life Sciences at Bar-Ilan University.

Google Scholar ORCIID LinkedIn Lab Webpage

Marcello is the Senior Director of Genome Engineering at AstraZeneca.

LinkedIn ResearchGate

Rasmus is a tenured Associate Professor at the Department of Biomedicine at Aarhus University. He is also the co-founder of UNIKUM Therapeutics.

Google Scholar ORCIID LinkedIn Lab Webpage

Life Edit Therapeutics, an ElevateBio portfolio company, is a gene editing company on a mission to rewrite the future by curing disease, making any edit, anywhere. Life Edit has built a world-class genome editing platform with one of the world’s largest and most diverse collections of novel RNA-guided nucleases (RGNs) and base editors. The company’s next-generation editing systems will propel the development of novel human therapeutics by enabling ex vivo engineering for cell therapies and regenerative medicines and in vivo delivery of gene therapies.

In addition to developing its own pipeline of cell and gene therapies, Life Edit Therapeutics will continue to strengthen its platform of genome-editing enzymes, provide gene editing expertise to strategic partners and ElevateBio’s portfolio companies, and form other third-party partnerships to discover and develop new therapies. Life Edit Therapeutics is based in Research Triangle Park, North Carolina.

Company website

ReCode Therapeutics is a clinical-stage genetic medicines company using precision delivery to power the next wave of mRNA and gene correction therapeutics. ReCode’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform enables highly precise and targeted delivery of genetic medicines directly to the organs, tissues, and cells implicated in disease, enabling improved efficacy and potency.

ReCode’s lead programmes include RCT1100 for the treatment of primary ciliary dyskinesia caused by pathogenic mutations in the DNAI1 gene, and RCT2100 for the treatment of the 10-13% of cystic fibrosis patients who have Class I or other mutations in the CFTR gene that do not respond to currently approved CFTR modulators. RCT1100 and RCT2100 are inhaled disease-modifying mRNA-based therapies formulated using the SORT LNP delivery platform.

Vivlion

IDT

Aldevron

BioSpring

Project Lead and Co-Founder at Ariya Bio.

Company website

Co-Founder and Chief Business Officer at QUiCKR Bio.

Company website

Co-CEO at Akribion Genomics.

Company website