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The CRISPR revolution: Changing life
The CRISPR revolution: Changing life will bring together experts from industry, academia and government to discuss the current state-of-the art in the use of the CRISPR/Cas9 system to understand biological pathways, to create cellular and animal models of disease, to develop improved agricultural crops and farm animals and to create new human therapies to correct somatic gene defects that lead to disease.
Following rapid advances in the research laboratory the field is beginning to witness the first commercial applications of CRISPR/Cas9. This meeting will address the technical, regulatory and ethical challenges associated with the commercialisation of CRISPR modified crop and animal species, and with the development of novel gene therapies created through the use of genome editing technologies.
See the programme below.
Attending this event
This open event is intended for participants from industry, academia and government who have an interest in genome editing.
Contact the Industry team for more information.
About the conference series
The conference is part of the Royal Society's Transforming our future conferences in the life sciences, supported by AstraZeneca. This conference series was launched to address the major scientific and technical challenges of the next decade and beyond. Each conference will focus on one topic and will seek to cover key issues, including:
- The current state of the key industry sectors involved
- The position of the UK and how it can benefit from the technology
- The future direction of research
- The challenges faced in turning research into commercial success
- The skills base needed to deliver major economic scientific advances
- The wider social and economic impacts
The conferences are a key component of the Society’s five-year Science, Industry and Translation initiative which demonstrates our commitment to reintegrate science and industry at the Society and to promote science and its value by connecting academia, industry and government.
Organisers
Schedule
Venki Ramakrishnan, President, The Royal Society
Venki Ramakrishnan, President, The Royal SocietyVenki Ramakrishnan has a long-standing interest in ribosome structure and function. In 2000, his laboratory determined the atomic structure of the 30S ribosomal subunit and its complexes with ligands and antibiotics. This work has led to insights into how the ribosome “reads” the genetic code, as well as into various aspects of antibiotic function. In the last few years, Ramakrishan’s lab has determined the high-resolution structures of functional complexes of the entire ribosome at various stages along the translational pathway, which has led to insights into its role in protein synthesis during decoding, peptidyl transfer, translocation and termination. More recently his laboratory has been applying cryoelectron microscopy to study eukaryotic and mitochondrial translation. Since 1999, he has been on the scientific staff of the MRC Laboratory of Molecular Biology in Cambridge and he is currently President of the Royal Society. |
Gene editing with CRISPR technology is transforming biology. Understanding the underlying chemical mechanisms of RNA-guided DNA and RNA cleavage provides a foundation for both conceptual advances and technology development. Professor Doudna will discuss how bacterial CRISPR adaptive immune systems inspire creation of powerful genome engineering tools, enabling advances in both fundamental biology and applications in medicine. She will also discuss the ethical challenges of some of these applications. Professor Jennifer Doudna ForMemRS, Professor and HHMI Investigator, UC Berkeley
Professor Jennifer Doudna ForMemRS, Professor and HHMI Investigator, UC BerkeleyAs an internationally renowned professor of Chemistry and Molecular and Cell Biology at U.C. Berkeley, Doudna and her colleagues rocked the research world in 2012 by describing a simple way of editing the DNA of any organism using an RNA-guided protein found in bacteria. This technology, called CRISPR-Cas9, has opened the floodgates of possibility for human and non-human applications of gene editing, including assisting researchers in the fight against HIV, sickle cell disease and muscular dystrophy. Doudna is an Investigator with the Howard Hughes Medical Institute and a member of the National Academy of Sciences, the National Academy of Medicine, the National Academy of Inventors and the American Academy of Arts and Sciences. She is also a Foreign Member of the Royal Society. |
Chair
Steve Rees, VP Discovery Biology, Discovery Sciences, AstraZeneca
Steve Rees, VP Discovery Biology, Discovery Sciences, AstraZeneca
In March 2017 Steve was appointed as Vice-President of the Discovery Biology department at AstraZeneca with global accountability for protein and cellular reagent generation and assay development, functional genomics and chemical biology. Prior to this Steve led the Screening Sciences and Sample Management department and successfully implemented strategies for hit identification, compound profiling, sample management and open innovation. Steve has led multiple international collaborations and has authored >60 scientific papers. Steve is currently Chair of the European Laboratory Research and Innovation group (ELRIG), has served as Chair of the SLAS Europe Council, and is a member of the Scientific Advisory Board for Axol Biosciences, LifeArc and the Centre for Membrane Protein and Receptor research at the Universities of Nottingham and Birmingham.
11:30 - 12:00 |
Opportunities and challenges of applying genome editing in farmed animals
The use of CRISPR-derived tools has greatly increased the efficiency with which specific genetic changes, from single base changes to targeted integration of novel sequences, can be made in farmed animals. The opportunities for using these technologies for genetic improvement in farmed animals, from fish to cattle, are now not constrained by technology but by uncertainty about the translation of the technology into animal production, due to regulatory uncertainty, questions of animal welfare and public acceptance. I will discuss how the technologies are being applied and current targets, for example disease resistance, and the issues of regulation and public perception. Professor Helen Sang, Personal Chair in Vertebrate Molecular Development, The Roslin Institute and R(D)SVS, University of Edinburgh
Professor Helen Sang, Personal Chair in Vertebrate Molecular Development, The Roslin Institute and R(D)SVS, University of EdinburghHelen Sang received a degree in Natural Sciences and PhD in genetics from Cambridge University. She continued developing a research career with fellowships held at Harvard and Edinburgh universities and was then appointed as Principal Investigator at The Roslin Institute, now part of the University of Edinburgh. Her main research focus at The Roslin Institute has been the development of technologies for genetic modification of the chicken, which are applied in basic biomedical research, biotechnology and investigating the potential for developing disease resistance in production chickens. Her research has been supported by the BBSRC, MRC, Wellcome Trust and industry. She is a Fellow of the Royal Society of Biology and the Royal Society of Edinburgh. |
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12:00 - 12:30 |
CRISPR crop opportunities
In this presentation I will look at how the CRISPR revolution is impacting our crop plants and at the opportunities this technology offers for development of improved agricultural crops. I will compare and contrast this new technology with conventional breeding methods and with genetic modification and I will use examples from our work in both Brassica and cereal crops to demonstrate the power of the technology. The latest CRISPR tools will be examined and the question of what impact these might have on future agricultural crops addressed. Professor Wendy Harwood, Senior Scientist, Crop Transformation Group, John Innes Centre
Professor Wendy Harwood, Senior Scientist, Crop Transformation Group, John Innes CentreWendy is a Senior Scientist at the John Innes Centre, Norwich. Her group works on the genetic modification and genome editing of crop plants including wheat, barley and Brassicas. The group uses genetic modification and CRISPR/Cas9 based genome editing as tools to better understand genes important in the development of improved agricultural crops. As well as focusing on developing improved technologies for crop engineering, the group has a particular interest in engineering drought tolerance in cereals. Wendy’s group is also responsible for the BRACT facility that is providing crop genetic modification and gene editing resources to the research community (www.bract.org). Wendy is active in public engagement and she is also an Honorary Lecturer at the University of East Anglia. |
12:30 - 13:00 |
Proportionate and adaptive governance of gene editing
CRISPR and related gene editing techniques could revolutionise our capability to meet human nutritional needs and desires, based at least partly on small and medium sized enterprises that create valuable new niche markets that are not of interest to the incumbent multinationals. However, delivery of this revolution will depend on how we decide to regulate these technologies. A new regulatory framework has been developed to support more proportionate and adaptive governance of innovative technologies with a focus on standards rather than legally based regulations, and considering the extent to which the innovation is disruptive of incumbent industry sector business models. Professor Joyce Tait CBE, Director, The Innogen Institute
Professor Joyce Tait CBE, Director, The Innogen InstituteJoyce Tait has an interdisciplinary background in natural and social sciences. Her research covers agrochemical, pharmaceutical and life science industry sectors, particularly: strategic planning for innovation; governance, risk management, regulation and standards; and stakeholder attitudes and influences. The focus has been particularly on genetic modification and synthetic biology, genetic databases, pharmaceuticals, regenerative medicine, stratified and translational medicine and cell therapies. Current projects include: ‘Proportionate and Adaptive Governance of Innovative Technologies’ with the British Standards Institution; BBSRC-funded Centre for Mammalian Synthetic Biology; EPSRC funded project ‘Implantable Microsystems for Personalised Anti-Cancer Therapy’; and ESRC-funded ‘Regenerative Medicine and its Development and Implementation’. Among current appointments are: UK Department for Business, Energy and Industrial Strategy (BEIS) Synthetic Biology Leadership Council (and Chair of its Governance Subgroup); Scientific Advisory Board, John Innes Centre; Board member, Industrial Biotechnology Innovation Centre; Board member, Roslin Foundation. |
Chair
Professor Peter Goodfellow FMedSci FRS
Professor Peter Goodfellow FMedSci FRS
Peter Goodfellow is a geneticist who tracked down the genetic essence of human ‘maleness’. In a high-profile scientific race to find the enigmatic ‘testis-determining factor’ (TDF; also known as sex-determining region Y (SRY) protein), Peter overturned others’ claims by correctly pinpointing the tiny SRY gene on the Y chromosome as the lynchpin of maleness in mammals.
Peter’s famous work began with his clever use of cell and molecular biology techniques to narrow down a segment of the Y chromosome as the location for the elusive TDF-encoding gene. Subsequently he found the crucial SRY gene and, with others, engineered a female mouse to become male through SRY expression.
In 1995, Peter won the Louis–Jeantet Prize for this work. The following year he began a decade-long chapter of his career, working in industry. Joining SmithKline Beecham as Head of Discovery, he then became — following a merger — Senior Vice-President of Discovery Research in the newly formed GlaxoSmithKline. Peter is now mostly retired.
14:10 - 14:40 |
Gene editing immunity
Experience from bone marrow transplantation has created a deep understanding of human immunity, and uncovered powerful immune mediated effects against infections and certain types of cancer. T-lymphocytes express a diverse repertoire of antigen-specific T cell receptors (TCRs) that recognise antigenic peptides in the context of specific human leukocyte antigen (HLA) and genetic engineering is being used to circumvent HLA-restriction and augment efficacy while avoiding non-specific, graft-versus-host effects. Recent advances in gene-editing technologies including TALE nucleases and CRISPR/Cas reagents has allowed the production of ‘universal’ anti-leukaemic cells. The cells can be collected, engineered in a specialist laboratory and cryopreserved until required. Early experience suggests these strategies may offer potent therapies for hard-to-treat malignancies and clinical trials are underway to test the approaches in the transplant arena. Professor Waseem Qasim, Professor of Cell and Gene Therapy, UCL Great Ormond Street Institute of Child Health
Professor Waseem Qasim, Professor of Cell and Gene Therapy, UCL Great Ormond Street Institute of Child HealthWaseem Qasim is Professor of Cell and Gene therapy at the Institute of Child Health, and a consultant immunologist at Great Ormond Street hospital, supported by the National Institute of Health Research. He trained in Immunology and Medicine, and then specialised in Paediatrics before PhD research at University College London. He is researching blood and marrow stem cell transplantation and the application of T cell therapies for cancer and infection. Waseem heads a research group developing gene based therapies, from basic laboratory development, through GMP manufacturing and into first-in-human trials. His group has a strong focus on lentiviral technology and gene-editing tools including TALENs and CRISPR/Cas reagents for novel therapeutic applications. |
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14:40 - 15:10 |
Development of a Subretinally-Delivered, CEP290-Specific CRISPR Medicine for the Treatment of Leber Congenital Amaurosis 10 (LCA10)
LCA10 is an early-onset, inherited retinal disease caused by mutations in the CEP290 gene. A reduction in CEP290 protein leads to a loss of vision due to the absence of outer segments in photoreceptors which renders these cells non-functional but viable. We aim to restore CEP290 expression by CRISPR editing and thereby photoreceptor function and vision. To correct the common IVS26 c.2991+1655 A>G mutation in the CEP290 gene, a panel of gRNAs with SaCas9 in human cells was screened. This screen identified a pair of gRNAs that efficiently cleaved intronic sequences flanking the mutation, resulting in the removal of the aberrant splice donor site created by the mutation. In fibroblasts derived from patients homozygous for the IVS26 mutation, editing led to significant reduction of mutant CEP290 mRNA and concomitant restoration of wild-type CEP290 mRNA and protein. The lead gRNAs had no detectable off-target cutting in multiple types of human cells as measured using both unbiased GUIDE-Seq and targeted sequencing methods. Productive CEP290 gene editing at expected therapeutic levels in neural retina was demonstrated in both human CEP290 IVS26 knock-in mice and cynomolgus macaques following subretinal delivery of a single AAV5 vector expressing both gRNAs and SaCas9 under the control of the photoreceptor-specific GRK1 promoter. Safety studies are underway to support development of the lead candidate. Editing of photoreceptors has the potential to address other inherited retinal diseases. Dr Charlie F Albright, CSO, Editas Medicine (Cambridge, MA)
Dr Charlie F Albright, CSO, Editas Medicine (Cambridge, MA)Charlie joined Editas Medicine as Chief Scientific Officer in August 2016. He brings more than 25 years of life sciences industry and academic leadership experience, most recently serving as vice president of genetically defined diseases and genomics at Bristol-Myers Squibb (BMS). Over his career, Charlie has led discovery programs that advanced investigational medicines into clinical development in a wide range of therapeutic areas, including neurodegeneration, pain, psychiatry, oncology and inflammation. Prior to his position as vice president of genetically defined diseases and genomics, he held multiple scientific leadership roles in neuroscience biology at BMS. Previously, he held positions at Incyte Corporation and DuPont Pharmaceuticals and was an assistant professor of biochemistry at Vanderbilt University. Charlie received a Bachelor of Science in Chemical Engineering and a Ph.D. in biology from the Massachusetts Institute of Technology (MIT). He was a postdoctoral fellow in the laboratory of Professor Robert Weinberg at the Whitehead Institute for Biomedical Research at MIT. |
15:10 - 15:35 |
In Vivo applications of CRISPR
Animal models with specific gene modification are essential tools for studying development and disease. Technologies that can delete insert or modify the gene sequences of organisms to understand and validate the function of specific genes are an essential part of drug discovery research. The development of the genome editing tool CRISPR/Cas9 has already greatly impacted basic research, drug discovery and translational research by enabling such gene edits in a rapid, precise and controlled manner. As genome editing is developed for therapeutic applications, in vivo validation of genome editing becomes an essential requirement to assess the efficiency and safety of the CRISPR/Cas9 technology. Genome editing using CRISPR/Cas9 technology represents a fast, multiplexable, efficient and simple technique to generate sophisticated gene manipulation that can be inducible, conditional or introduce new functions such as endogenous reporters in any mammalian species. Dr Mohammad Bohlooly, Director, Translational Genomics, Discovery Sciences iMED, AstraZeneca
Dr Mohammad Bohlooly, Director, Translational Genomics, Discovery Sciences iMED, AstraZenecaMohammad received his PhD degree in physiology from the University of Gothenburg (Sweden), where he generated transgenic mice and studied for metabolic, cardiovascular and CNS related phenotype. Mohammad joined AstraZeneca R&D Sweden in 2001 as senior research scientist to build an in vivo phenotyping platform for transgenic animals. Since joining AstraZeneca, he had various positions and responsibilities (e.g. lead for in vivo team, transgenic team, iPS and stem cell team at Discovery science). In early 2017 he was promoted to director to lead the Translational Genomic department at Discovery Sciences in Gothenberg. |
15:35 - 16:00 |
CRISPR screens for oncology drug target discovery
Cancer is caused by multiple molecular alterations that lead to changes in the cellular processes necessary for malignancy. Cancer medicines can exploit cellular rewiring in malignant cells to selectively target tumour-specific dependencies, while sparing healthy cells. The histological, genetic and epigenetic heterogeneity of cancer means that identifying these dependencies is challenging, and limits our ability to develop effective therapies. To identify new oncology drug targets, we are performing whole-genome CRISPR-Cas9 drop-out screens across a large number of genomically-annotated human cancer cell lines. I will discuss these datasets and methods we are developing to systematically link gene essentiality with the molecular features of cancer to guide the development of molecularly targeted oncology therapies. Dr Mathew Garnett, Group Leader, Wellcome Trust Sanger Institute
Dr Mathew Garnett, Group Leader, Wellcome Trust Sanger InstituteMathew Garnett, PhD leads the Translational Cancer Genomics laboratory and Genomics of Drug Sensitivity in Cancer Project at the Wellcome Sanger Institute, Cambridge UK. He aims to understand how genetic changes contribute to cancer and to identify molecular biomarkers that will improve the development of new cancer therapies using high-throughput chemical and genetic screens in cancer cell lines and organoids. Mathew is also a member of the scientific leadership team for Open Targets which aims to use genome-scale experiments and analysis to evaluate new therapeutic targets. After obtaining a BSc in Biochemistry (Hons.) at the University of British Columbia, Canada, Mathew completed his PhD with Professor Richard Marais at the Institute of Cancer Research (London, UK) on the characterisation of BRAF as a cancer gene. In 2005 Mathew moved to the laboratory of Professor Ashok Venkitaraman (Cambridge, UK) for his post-doctoral research, where he discovered a new regulator of cell division. Mathew joined the Sanger Institute in 2009. Contact: mg12@sanger.ac.uk. |
Chair
Baroness Helena Kennedy QC
Baroness Helena Kennedy QC
Panellists (confirmed)
Dr Robin Lovell-Badge FMedSci FRS
Dr Robin Lovell-Badge FMedSci FRSRobin Lovell-Badge obtained his PhD at University College London in 1978 and was a postdoc in Cambridge, both with Martin Evans. After an EMBO fellowship in Paris he established his independent laboratory in 1982 at the MRC Mammalian Development Unit, UCL, directed by Anne McLaren. In 1988 he moved to the MRC National Institute for Medical Research, becoming Head of the Division of Stem Cell Biology and Developmental Genetics in 1993. The NIMR was incorporated into the Francis Crick Institute in April 2015. In 1990, his lab discovered Sry, the Y-linked sex determining gene and the first members of the Sox gene family. He has had long-standing interests in the biology of stem cells, in how genes work in the context of embryo development, and how decisions of cell fate are made. Major themes of his current work include sex determination, development of the nervous system and pituitary, and the biology of stem cells within these systems. He is also very active in both public engagement and policy work, notably around stem cells, genetics, human embryo and animal research, and in ways science is regulated and disseminated. He chairs the Royal Society’s Genetic Technologies Programme and has been involved in the human genome editing Summit meetings since their inception. He was elected a member of EMBO (1993), a Fellow of the Academy of Medical Sciences (1999), the Royal Society (2001), the Royal Society of Biology (2011), and the American Association for the Advancement of Science (AAAS) (2018). He has received the Louis Jeantet Prize for Medicine (1995), the Amory Prize (1996), the Feldberg Foundation Prize (2008), the Waddington Medal of the British Society for Developmental Biology (2010), the ISSCR Public Service Award (2021), and the Genetics Society Medal (2022). He was awarded a CBE in the 2018 New Year’s Honours List. He is an honorary professor at University College, London and at King’s College, London, and a Special Visiting Professor at the University of Hong Kong. Dr Sarah Chan, Chancellor's Fellow, Usher Institute for Population Health Sciences and Informatics, University of Edinburgh
Dr Sarah Chan, Chancellor's Fellow, Usher Institute for Population Health Sciences and Informatics, University of EdinburghSarah Chan is a Chancellor’s Fellow at the Usher Institute for Population Health Sciences and Informatics, and Deputy Director of the Mason Institute for Medicine, Life Sciences and Law, University of Edinburgh. Previously, from 2005 to 2015, she was a Research Fellow in Bioethics at the University of Manchester, first at the Centre for Social Ethics and Policy and from 2008 the Institute for Science Ethics and Innovation, where she was Deputy Director. Sarah’s previous research in interdisciplinary bioethics has focused on the ethics of new biomedical technologies, including stem cell and embryo research; reproductive medicine; synthetic biology; gene therapy and genetic modification; and human and animal enhancement. Her current work draws on these interests to explore the ethics of emerging modes of biomedicine at the interface of health care research, medical treatment and consumer medicine including population-level health and genetic data research; the use of biomaterials in both research and treatment; and access to experimental treatments and medical innovation. Dr Mark J Robertson, Director and Founder, Science Policy Compass
Dr Mark J Robertson, Director and Founder, Science Policy CompassMark is Founder & Director of Science Policy Compass Ltd. a new consultancy offering science policy support and advice on a range of bioethical topics. His current interest areas include: Biosector reputation; Access to human biological samples for research use including donor and patient consent and privacy; Precise genome editing /gene therapy ethics; Nagoya Protocol - access and benefit sharing. Until August 2016, Mark was Director in the Global Policy & Corporate Responsibility team in Corporate Affairs, AstraZeneca with 7 years dedicated science policy experience. As Chair of the AstraZeneca Bioethics Advisory Group, he was responsible for evolving the AstraZeneca Global Bioethics Policy and strategy. Educated at Southampton University, UK and University of Chicago, USA, Mark is a PhD pharmacologist (>50 peer-reviewed publications) and with 30 years Big Pharma experience mostly in novel medicine project delivery. |