29th - 30th January 2025 | ExCeL, London
GENOME EDITING
Engineering Life: Gene Editing from Research To The Clinic
The Gene Editing Stage examines challenges in achieving precise gene edits and advancing gene and cell therapies, with talks on mitigating off-target effects and overcoming safety concerns in clinical applications.
DAY 1
On Day 1, The Genome Editing Stage will focus on advances in genome editing technologies and translating genome editing into the clinic.
[topic] ADVANCES IN GENOME EDITING TECHNOLOGIES
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Reproducibility & Reporting Guidelines for Genome Editing in Animal & Human Models
- How much do we know about the function of mammalian genes?
- Looking to the future of genome editing for animal models
- The LAGeR guidelines for Improving laboratory animal genetic reporting
- How it benefits and translate to human research
Speaker
Head of Unit, Genetic Engineering & Model Validation Department
Institut Clinique de la Souris
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Sponsored Talk Available
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Design of CRISPR Experiments & Cell Line Engineering
- Implementation of CRISPR techniques including creation of cell lines with CRISPR deletions
- Considerations for CRISPR knock-in experiments
- Guidelines to choosing the best CRISP delivery for cell engineering
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[break] Lunch
- Exhibition Floor: Wander around the vibrant exhibition floor to see the latest technologies and services on offer
- Food Options: Grab a sandwich at The Tree of Life Cafe or head on over to the Food Village for a range of hot food options including Indian, Jamaican and Japanese food
- Live Lounge Over Lunch: 15 mins technology focussed presentations
- Poster Zone: Browse the posters and reach out to potential new collaborators
- Talkaoke: The Flying Saucer of Chat, a fun and interactive pop-up show that offers you the opportunity to sit down with others and discuss any topic you like.
- Patient Perspectives: Talks and discussions on the crucial role of patients and patient families in the understanding and development of treatments for rare diseases
[topic] ADVANCES IN GENOME EDITING TECHNOLOGIES
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Removal of Antimicrobial Resistance Genes from Bacterial Strains and Communities Using CRISPR-Cas9
- Antimicrobial Resistance (AMR) is a major threat to human healthcare Often, AMR genes are carried on plasmids – bacterial mobile genetic elements.
- We engineer a broad host-range plasmid to encode CRISPR-Cas9 (pKJK5::csg). pKJK5::csg can be used to protect bacteria from uptake of AMR genes, or to resensitise them to antibiotics by removal of AMR plasmids.
- We further assess for which use-cases mobile CRISPR-Cas9 tools such as pKJK5::csg are most effective.
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Mobile CRISPR-Cas9 tools are a novel tool in the arsenal to combat the mounting tide of AMR.
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Quantifying Non-Specific Off-Target Activity By Base Editors
- Accurate editing quantification is essential for studying RNA editing, often limited by coverage and alignment problems.
- We discuss several common pitfalls and several strategies appropriate for coding and non-coding regions
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Methods developed and lessons learned from the endogenous editome may facilitate better site-directed editing design.
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Transposable Element Expression in Gene Expression during Early Mammalian Development
- How the genome controls transposable elements by epigenetic modifications in early development
- Using CELLO-seq to study locus-specific transposable element expression in single cells using long-read sequencing
- Functional tool such as CRISPRi to understand the role of locus-specific transposable element expression in development
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[break] Break
- Speed Networking: Fun and useful sessions where you’ll rapidly meet other Festival attendees to immediately expand your network
- Food Options: Grab a coffee and a cake at The Tree of Life Cafe or head on over to the Food Village for a range of hot food options including Indian, Jamaican and Thai food
- Exhibition Floor: Wander around the vibrant exhibition floor to see the latest technologies and services on offer
- Talkaoke: The Flying Saucer of Chat, a fun and interactive pop-up show that offers you the opportunity to sit down with others and discuss any topic you like
[topic] GENOME EDITING TRANSLATION INTO THE CLINIC
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Gene Therapy & Editing for Neurological Diseases
- Why we need gene therapy / gene editing for neurological diseases
- Current approaches available
- Innovative and novel genetic therapies for intractable neurological diseases using a variety of in vivo and ex vivo experimental techniques,
Speaker
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Mapping the Functional Impact of Non-Coding Regulatory Elements in Primary T Cells through Single-cell CRISPR Screens
- “Primary T cell crisprQTL” is a scalable, single-cell functional genomics approach for mapping regulatory elements to genes in primary human T cells.
- This framework can facilitate the interrogation of immune disease GWAS hits.
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The combination of experimental and QTL-based techniques is likely to address the variant-to-function problem.
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Application & Clinical Translation of CRISPR/Cas9 Genetic Screens in Cancer Cells
- Cancer dependency maps to support target identification and drug discovery
- The discovery of Werner Syndrome helicase as a synthetic-lethal target in microsatellite unstable cancers
DAY 2
Day 2 on The Genome Editing Stage will focus on Personalised Cell & Gene Therapy.
[topic] PERSONALISED CELL & GENE THERAPY
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A New Age of Genomic Therapies for Hearing Loss?
- Genetic diagnostics for hearing loss have become widely available in the UK, with better informed etiologies for hearing loss
- Several companies are pursuing gene therapies for hearing loss, and have reported success around the world for OTOF mutations
- There is likely to be a coming paradigm shift in how we manage genetic hearing losses in the near to medium term future
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Discovery & Preclinical Development of Therapeutics to Treat Hearing loss
- Bridge the gap and overcome challenges when developing animal models for genetic and acquired hearing loss
- Uncover the lack in current in vivo models of the inner ear, unable to recapitulate the cochlear hair cells, supporting cells, spiral ganglion neurons, vestibular hair cells and stria vascularis cells
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Explore novel in vitro models, such as organoids and iPSCs for better replication of the human inner ear and how these models could pave the way towards a patient-centric approach
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Gene Therapy for Lung Diseases
- Development of novel gene and cell therapy-based treatments for lung diseases
- Preclinical and clinical research related to advanced therapies for rare lung diseases i.e. cystic fibrosis and anti-trypsin deficiency
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[break] Lunch
- Exhibition Floor: Wander around the vibrant exhibition floor to see the latest technologies and services on offer
- Food Options: Grab a sandwich at The Tree of Life Cafe or head on over to the Food Village for a range of hot food options including Indian, Jamaican and Japanese food
- Live Lounge Over Lunch: 15 mins technology focussed presentations
- Poster Zone: Browse the posters and reach out to potential new collaborators
- Talkaoke: The Flying Saucer of Chat, a fun and interactive pop-up show that offers you the opportunity to sit down with others and discuss any topic you like.
[topic] PERSONALISED CELL & GENE THERAPY
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Crack in the Creation, Prenatal Therapy for Congenital Blood Disorders
- Prenatal Therapy In utero Gene Therapy
- Prenatal Therapy for Sickle Cell Disease
Speaker
Clinical Senior Lecturer and Consultant in Maternal and Fetal Medicine
King's College London
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Personalised Engineered Cell Therapies for Cystic Fibrosis
- Gene editing therapies for cystic fibrosis using gene editing, gene delivery, stem cell biology and cystic fibrosis epithelial biology
- Optimal methods for delivery of CRISPR/Cas9 formulations
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Air-liquid interface models of the CF epithelium for identifying biological readouts and testing therapies
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Cell Therapies for Cartlige Repair
- Cell-based therapies for the repair of cartilage defects ‘Omic (transcriptomic, quantitative proteomics and metabolomics) and bioinformatics approaches to identify better performing cells and for patient stratification
- Novel allogeneic therapies for the treatment of cartilage defects
- Cell-based therapies for the repair of cartilage defects
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[break] Break
- Speed Networking: Fun and useful sessions where you’ll rapidly meet other Festival attendees to immediately expand your network
- Food Options: Grab a sandwich at The Tree of Life Cafe or head on over to the Food Village for a range of hot food options including Indian, Jamaican and Thai food
- Exhibition Floor: Wander around the vibrant exhibition floor to see the latest technologies and services on offer
- Talkaoke: The Flying Saucer of Chat, a fun and interactive pop-up show that offers you the opportunity to sit down with others and discuss any topic you like
[topic] PERSONALISED CELL & GENE THERAPY
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In Vivo Evaluation of Novel Synthetic Promoters for CNS Gene Therapy
- Sania’s approach: precision genetic neuromodulation
- Generation of large GRE libraries for screening in motor neurons
- GRE-Scan: parallel, high throughput, AAV-based screening of many GREs in specific cell types
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Genomic Safety for Clinical Development of CRISPR-Edited Allogeneic CAR-T
- Gene Editing Safety in Non-Clinical Development of CAR-Ts