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The CRISPR revolution: Changing life

Event

Location

The Royal Society, London, 6-9 Carlton House Terrace, London, SW1Y 5AG

Overview

Image credit: AstraZeneca

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.

Event organisers

Select an organiser for more information

Schedule of talks

07 March

09:00-09:30

Registration

09:30-09:45

Welcome remarks

1 talk Show detail Hide detail

Venki Ramakrishnan, President, The Royal Society

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09:45-10:45

CRISPR systems: Nature’s toolkit for genome editing

1 talk Show detail Hide detail

Professor Jennifer Doudna ForMemRS, Professor and HHMI Investigator, UC Berkeley

Abstract

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.

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10:45-11:15

Coffee

11:15-13:00

Session 1: Agricultural science

3 talks Show detail Hide detail

Chairs

Steve Rees, VP Discovery Biology, Discovery Sciences, AstraZeneca

11:30-12:00 Opportunities and challenges of applying genome editing in farmed animals

Professor Helen Sang, Personal Chair in Vertebrate Molecular Development, The Roslin Institute and R(D)SVS, University of Edinburgh

Abstract

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.

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12:00-12:30 CRISPR crop opportunities

Professor Wendy Harwood, Senior Scientist, Crop Transformation Group, John Innes Centre

Abstract

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. 

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12:30-13:00 Proportionate and adaptive governance of gene editing

Professor Joyce Tait CBE, Director, The Innogen Institute

Abstract

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.

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13:00-14:00

Lunch

14:00-16:00

Session 2: Human medicine

4 talks Show detail Hide detail

Chairs

Professor Peter Goodfellow FMedSci FRS

14:10-14:40 Gene editing immunity

Professor Waseem Qasim, Professor of Cell and Gene Therapy, UCL Great Ormond Street Institute of Child Health

Abstract

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.

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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)

Dr Charlie F Albright, CSO, Editas Medicine (Cambridge, MA)

Abstract

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.   


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15:10-15:35 In Vivo applications of CRISPR

Dr Mohammad Bohlooly, Director, Translational Genomics, Discovery Sciences iMED, AstraZeneca

Abstract

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.

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15:35-16:00 CRISPR screens for oncology drug target discovery

Dr Mathew Garnett, Group Leader, Wellcome Trust Sanger Institute

Abstract

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.

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16:00-16:30

Tea

16:30-17:25

Panel discussion: Ethical considerations of genome editing

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Chairs

Baroness Helena Kennedy QC

Panellists (confirmed)

Dr Robin Lovell-Badge FMedSci FRS, Group Leader and Head of the Division of Stem Cell Biology and Developmental Genetics, The Francis Crick Institute
Dr Sarah Chan, Chancellor's Fellow, Usher Institute for Population Health Sciences and Informatics, University of Edinburgh
Dr Mark J Robertson, Director and Founder, Science Policy Compass

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The CRISPR revolution: Changing life The Royal Society, London 6-9 Carlton House Terrace London SW1Y 5AG UK