In-person event 8-11th April

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.

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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.

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

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Alessio Strano, PhD, is a postdoctoral scientist in the Platt Laboratory for Biological Engineering of ETH Zürich, Switzerland.

His current research focuses on developing CRISPR- and AAV-based technologies to perform functional genomic screens directly in vivo and applying them to study physiological and disease processes in the mouse brain, with a particular interest in aging and neurodegeneration.

Alessio obtained his PhD in 2020 from the University of Cambridge, UK, where he characterised the developmental mechanisms underlying patterning variation in pluripotent stem cell-derived neural differentiations, and collaborated on multiple studies using these systems to investigate Down syndrome, Alzheimer’s disease, and tauopathies.

Dr. Alvin Luk, Co-founder and CEO of HuidaGene Therapeutics, a clinical-stage company that is developing a broad pipeline of RNA- and DNA-editing therapeutics. He has more than 30 years of global drug development experience, specialising in rare diseases. He has held executive roles at multiple companies including Spark Therapeutics and Biogen, contributing to the approval of LUXTURNA®, the first AAV gene therapy. Dr. Luk has made over 250 regulatory submissions globally and served on the US-FDA Rare Disease Clinical Design Committee. He has contributed to 21 approved products and co-authored more than 100 scientific papers in prestigious journals. Dr. Luk holds an MBA from Harvard and a Ph.D. in Neuroscience from UC San Francisco Medical School.

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Dr. Annarita Miccio directs the Laboratory of Chromatin and Gene Regulation at the Imagine Institute in Paris. Her research focuses on understanding the transcriptional control haematopoiesis and new therapies for β-haemoglobinopathies. During her PhD, she created a lentiviral vector used in a β-thalassemia trial. As a post-doc and assistant professor, she specialised in erythroid development gene regulation and gene therapy for haematopoietic disorders. Since becoming Lab Director in 2014, she has advanced stem cell research and β-haemoglobinopathy treatments, optimising lentiviral vectors for sickle cell disease trials and developing CRISPR/Cas9 strategies. Dr. Miccio has authored over 50 publications, 15 patents, and received several awards.

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

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Antoine Decrulle PhD is Vice President of Research at Eligo Bioscience.

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Cecilia Jimenez-Mallebrera, based at Barcelona's Sant Joan de Déu Pediatric Hospital, specialises in translational research in neuromuscular diseases. Her work integrates advanced therapies and biomarker discovery for paediatric neuromuscular conditions. Following a Biology degree from the University of Navarra and a PhD in Genetics from University College London, studying the utrophin gene, she completed postdoctoral work at the Dubowitz Neuromuscular Centre in London, focusing on congenital muscular dystrophies.

Since establishing her research group in 2010, she has pioneered CRISPR/Cas9 applications for muscular dystrophies and implemented serum GDF-15 as a biomarker for mitochondrial diseases. Her research group participates in various clinical trials for Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, Pompe´s Disease and other muscle diseases and contributes to several research networks such as the EU ERDERA consortium and the COST Action on Gene Editing, GenE-HumDi, as well as the Spanish Biomedical Research Network on Rare Diseases, CIBERER.

Her research is supported through the European Reference Network for Neuromuscular Diseases, Spain's National Reference Centers, and various research consortia including the EU ERDERA and CIBERER networks.

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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.

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Daniel Siegwart is the W. Ray Wallace Distinguished Chair in Molecular Oncology Research and Professor at the University of Texas Southwestern Medical Center. His research programme is focused on developing advanced lipid- and polymer-based systems with precise control over structure and responsiveness for applications in drug delivery, imaging, genetic diseases, and cancer. He co-founded ReCode Therapeutics in order to translate UT Southwestern developed nanoparticles for delivery of genetic medicines. ReCode is a biopharmaceutical company developing precision medicines for pulmonary diseases with significant unmet medical need including primary ciliary dyskinesia and cystic fibrosis.

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Eric B. Kmiec, Ph.D., is Founder and Executive Director of ChristianaCare’s Gene Editing Institute Inc. (GEI), a translational research and education institute with a specific focus on enabling breakthrough technologies to reach underserved populations. Here, he directs a team of 25 people developing a novel CRISPR-based gene-editing approach for solid tumours through the FDA approval process.

Dr. Kmiec is an entrepreneur in genetic medicine, having founded and managed two biotechnology companies: Kimeragen, (now, Cibus) and CorriXR Therapeutics where he serves as Chief Scientific Officer and Chief Executive Officer.

He received his Ph.D. in Molecular Biology and Microbiology from the University of Florida School of Medicine and holds faculty appointments at the University of Delaware and the Wistar Institute. Dr. Kmiec has served as the Principal Investigator for research projects supported by the National Institute of Health, the National Science Foundation, the NIST-BIRD Foundation, and the American Cancer Society. He serves as the Section Editor for genome engineering of Gene and sits on numerous other editorial boards, has 174 peer-reviewed publications as primary or senior author, 18 issued patents, and has edited several books on gene therapy.

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Gerald Schwank, Associate Professor at the University of Zurich's Medical Faculty, specialises in translational genome editing. His team integrates 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 from the University of Zurich and completed postdoctoral work at the Hubrecht Institute, pioneering genome-editing in patient-derived organoids. He established his lab at ETH Zurich in 2015, developing CRISPR screening methods for cancer research and in vivo gene correction in the liver. His research is supported by rare disease organisations, the Swiss National Science Foundation, and the European Research Council.

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Grégoire completed his Ph.D. in Cell Biology and Physiopathology at the University of Bordeaux (France), where he focused on the genotoxicity of the CRISPR-Cas9 system. He then pursued his first postdoctoral experience, applying CRISPR-based diagnostic methods to monitor relapse in chronic myeloid leukaemia. In January 2021, Grégoire joined the Jacob Corn's lab at ETH Zurich as a postdoctoral researcher. His current research interests include both ex vivo and in vivo gene editing, with a continued focus on genomic stability.

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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.

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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.

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Jakob Nilsson is a group leader at the Danish Cancer Society using functional genomics to dissect cellular regulatory mechanisms. The group primarily uses base-editing screens to obtain high resolution information on protein function and couple this with biochemical approaches. Jakob Nilsson is furthermore establishing the open access infrastructure on optical pooled screening that will provide users with the possibility to couple genetic perturbations to visual readouts in the microscope, expanding on the phenotypic landscape that can be explored in functional genomics. Jakob obtained his PhD from University of Aarhus and following post-doctoral work he established his lab at University of Copenhagen in 2009 and has been at the Novo Nordisk Foundation Center for Protein Research since 2011.

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Dr. James Longden is Senior Director, Life Science at Evolvus where he leads the development of the life science knowledge base, a series of products that describe biological and chemical interactions (bioactivity) and chemical properties (ADME) for small molecules, protein degraders, conjugates and biological therapeutics. Prior to joining Evolvus, Dr. Longden worked in the pharmaceutical/biotech industry for over 20 years leading drug and target discovery projects with a focus on big data biology and the use of machine learning to predict the signalling network changes driving disease and how we can perturb them. He has a PhD in Biomedical Science from the University of Nottingham.

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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.

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Karim is a Senior Scientist at the GENyO. Pfizer-University of Granada-Junta de Andalucia Center for Genomics and Oncological Research in Granada, Spain.

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Kiana Aran is a biomedical entrepreneur and bioengineering professor at UCSD. She co-founded and was CSO of Cardeabio (acquired by Paragraf UK), 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. Kiana consults for the Gates Foundation and is a member of the National Academies' New Voices. Her achievements include the Clinical OMICs 10 under 40 Award, Athena Pinnacle Award, NSF Career award, Nature Research Award for Inspiring Women in Science (2021), Rutgers University's Distinguished Engineering Medal (2022), Inc. USA top 200 female founders (2023), and San Diego Engineering Council's Distinguished Engineer (2024). She was elected to the National Academy of Inventors as a senior member.

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Lilian Lamech is a Senior Scientist at Life Edit Therapeutics, an ElevateBio company, where she leads development of genome-editing systems based on novel RNA-guide nucleases to drive the advancement of therapeutic programmes.

Prior to joining Life Edit, Lilian was a Senior Scientist at Chemeleon, a small Brooklyn-based startup, where she focused on developing novel colorimetric biosensors for point-of-care detection of cerebrospinal fluid leaks and other disease biomarkers. Lilian completed her postdoctoral research at Memorial Sloan Kettering Cancer Center, where she investigated lysosomal nutrient recycling and scavenging pathways critical for cancer cell survival. She earned her Ph.D. in Microbiology from the University of Texas at Austin, and her B.S. in Biochemistry from Denison University, Ohio

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Marcello is the Senior Director of Genome Engineering at AstraZeneca.

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Martin Pacesa is a postdoctoral researcher in the Laboratory of Protein Design and Immunoengineering at EPFL, Lausanne, Switzerland. His work spans computational and structural biology, with a focus on protein-nucleic acid interactions, genome editing, and deep learning-based protein design.
Martin earned his Ph.D. from the University of Zurich under the mentorship of Prof. Martin Jinek, where he explored the structural and biochemical mechanisms of CRISPR-Cas systems. His research revealed the conformational dynamics of Cas9 during target DNA binding, as well as the mechanistic and structural basis of Cas9 off-target activity and mismatch tolerance.

At EPFL in the group of Prof. Bruno Correia, Martin has expanded his research into computational protein design. He co-developed a method for the functional solubilisation of membrane proteins, a long standing problem in biochemistry. Additionally, he developed BindCraft, an open-source pipeline for accurate de novo binder design, without the need for extensive experimental screening or optimisation.

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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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Özcan Met PhD is Associate Professor and Head of Cell Therapy Unit CCIT-DK and DTU HealthTech, both in Denmark. As head of the cell therapy unit at CCIT-DK since 2010, Özcan directs all operational activities required for the manufacturing of cellular products intended for use in clinical trials. As Head of Cell Therapy, he has been responsible for the release of more than 200 cell therapy products in Phase I, II, and III immunotherapy clinical trials.
Özcan Met has more than 20 years of research experience in tailoring the immune system to combat cancer and is leading a team at the cutting edge in the development of novel cell-based technologies for patient treatment.

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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.

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

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Dr. Rob Wolthuis is Associate Professor at the Department of Human Genetics, Amsterdam UMC. He is the Head of the Section of Oncogenetics and co-manager of the Amsterdam UMC CRISPR Expertise Center. Wolthius' team is engaged in genome-wide screens in conjunction with targeted cancer drugs or relevant compounds. This research is critical in understanding genes underlying drug responses and reveal potential mechanisms of drug resistance. Obtained insights may also help to reveal disease prevention strategies for cancer predisposed individuals. They are also pioneering the development of other CRISPR-related applications, e.g. the use of Cas12 in genome diagnostics. Looking ahead, there is an ambitious vision to explore the integration of targeted gene editing within clinically relevant therapeutic genetics.

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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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Thorsten Stafforst is a Full Professor of Nucleic Acid Biochemistry at the Interfaculty Institute of Biochemistry at the University of Tuebingen, Germany. His research focuses on the engineering of RNA drugs and RNA-guided tools. He has a background in chemistry and bioengineering. He is a founding member of the Gene and RNA Therapy Center at the University Hospital in Tuebingen and co-founder of AIRNA (www.airna.com) where he serves as a director on the board.

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Dr. Traver Hart is an Associate Professor in the Department of Systems Biology at MD Anderson. He earned his PhD in Cell and Molecular Biology in 2008 at the University of Texas at Austin, studying functional genomics in yeast and mammalian cells.

During his postdoc with Dr. Jason Moffat at the Donnelly Centre in Toronto, he helped develop CRISPR/Cas9 pooled library knockout technology for genetic screening of cancer cells and identifying context-specific essential genes that might be good tumour-specific drug targets.

He joined MD Anderson in 2016, where he runs a hybrid lab developing experimental and computational tools for finding cancer targets and understanding the hierarchical organisation of the cell. His latest work expands CRISPR capabilities to handle the combinatorial perturbations required for understanding synthetic lethality.

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Amanda Haas is a Business Development Manager at Agilent Technologies with more than 19 years of experience in life sciences and biotechnology. She has been with Agilent for 3 years, growing gene-editing therapeutic services, using market research, voice of the customer and collaborating with the development team. Amanda holds a B.S. degree in Chemistry from the University of Miamy. She previously worked at Horizon Discovery, Dharmacon, and Thermo Fisher Scientific.

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Ashley Jacobi currently leads the Genomic Medicine & CRISPR Application Development team at Integrated DNA Technologies (IDT). She received her Molecular Genetics & Biochemistry degree from Cornell College in 2006. Ashley has since spent the last 18 years holding several roles in the Research & Product Development teams at IDT. She has conducted research in RNAi and antisense oligo technologies, and more recently has been focused on studying CRISPR gene editing, which has included high-throughput screening of CRISPR-Cas guide RNAs, optimising the composition and delivery of synthetic RNA reagents complexed to recombinant CRISPR nucleases, and developing analysis solutions to understand the genetic outcomes following CRISPR gene editing. Ashley is also a certified practitioner in Customer Insight and leads external scientific collaborations at IDT.

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Iván Hernández is the CSO and co-founder of Countagen, a Stockholm-based startup developing innovative genotyping solutions that simplify and streamline gene editing validation. With 15 years of experience in academic research and industrial R&D, Iván specialises in assay and product development for nucleic acid analysis.

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Juliana Campo is a Field Application Scientist at GenScript, focusing on the company's RNA and CRISPR services. Juliana's research background includes stem cell biology and neuroimmunology.

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Aldevron

Philipp Kamm is a Project Lead at BioSpring GmbH, a world-leading CDMO for oligonucleotide and mRNA manufacturing. He has been with BioSpring for three years, establishing enzymatic manufacturing of nucleic acids including GMP-grade mRNA. Philipp holds a PhD in Chemistry jointly awarded by Queensland University of Technology and Karlsruhe Institute of Technology.

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Simon is the Co-Founder and CSO at Broken String Biosciences.

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Manuel is a LOEWE-FCI Professor at Goethe University Frankfurt. He is also the Chief Scientific Officer at Vivlion, and the head of the Frankfurt CRISPR Screening Center.

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Dr. Giedrius Gasiunas is the co-founder, board member, and CSO at Caszyme, a biotech company specialising in CRISPR-Cas gene-editing technology. In 2012, Dr. Gasiunas earned his PhD in Biochemistry from Vilnius University, where he studied under CRISPR pioneer Prof. Virginijus Siksnys. His research focused on the Cas9 protein, demonstrating its role as a programmable RNA-guided DNA endonuclease. In 2016, Dr. Gasiunas received the young scientist scholarship established by the Lithuanian Academy of Sciences, and in 2017, along with his colleagues, he was awarded the Lithuanian Science Prize.

Dr. Gasiunas is also a senior researcher and lecturer at Vilnius University Life Science Center, Institute of Biotechnology. With over 17 years of experience in the CRISPR-Cas field, Dr. Gasiunas has contributed to several Cas9-related patents and co-authored 33 scientific publications, which have been cited more than 4,000 times, resulting in an h-index of 21.

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Co-Founder and Chief Business Officer at QUiCKR Bio.

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Co-CEO at Akribion Therapeutics.

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CEO, Board Member and Co-Founder at CasZyme.

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CEO and Co-Founder at TaKenOti Bio

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