Thursday 13 October
07:30-08:30
Registration
08:30-10:45
Pentland / Sidlaw
Parallel 4a
Gene and epigenetic editing II
Chairs: Francisco Martin, Genyo, Granada / Paula Rio, Ciemat, Madrid
INV35: Claudio Mussolino, University of Freiburg
Strategies to improve precision and safety of human cells manipulation.
INV36: Raffaella Di Micco, SR-Tiget, Milan
Mechanistic insights to advance hematopoietic stem cell based gene therapies
OR43: Mariacarmela Alloca, Editas Medicine
A Mutation-Independent CRISPR/Cas9-based ‘Knockout and Replace’ Strategy to Treat Rhodopsin-Associated Autosomal Dominant Retinitis Pigmentosa
OR44: Alice Rovai, Hannover Medical School
In vivo adenine base editing corrects the HFE C282Y mutation and improves iron metabolism in hemochromatosis mice
OR45: Martino Cappellutti, SR Tiget, Milan
Durable silencing of Pcsk9 by in vivo hit-and-run epigenome editing.
OR46: Arianna Moiani, Cellectis Therapeutics
Non-viral DNA delivery associated to TALEN® gene editing leads to highly efficient correction of sickle cell mutation in long-term repopulating hematopoietic stem cells
P144: Paula Rio, Ciemat Madrid
Therapeutic Base and Prime Editing in Fanconi Anemia Hematopoietic Stem and Progenitor Cells
P427: Marco Luciani, SR Tiget, Milan
Epigenome-editing strategies to enhance oligodendroglial differentiation from human induced pluripotent stem cells
08:30-10:45
Fintry
Parallel 4b
CNS & sensory II
Chairs: Simon Waddington, UCL London / Robin Ali, Kings College London
INV33: Nathalie Cartier, AskBio, Paris
Pathway Gene therapy for Huntington disease : A Phase I/II Dose-Finding study
to Evaluate BV-101 Striatal Administration in Adults with Early Manifest
Hutington’s disease
INV34: Hinrich Staecker, University of Kansas Medical Center
Leveraging cochlear implantation to accelerate the translation of inner ear gene therapy:
OR38: Larissa Nassauer, Hannover Medical School
Development of novel gene therapeutic approaches for protection from drug-induced ototoxicity
OR39: Rita Milazzo, Altheia Science s.r.l
Clinical benefit of PD-L1 hematopoietic stem and progenitor cell gene therapy in an animal model of multiple sclerosis
OR40: Sophia Millington Ward, Trinity College Dublin
AAV-delivered NDI1 improves mitochondrial function and provides benefit in AMD models
OR41: Younbok Lee, Kings College London
Intra-thalamic delivery of AVB.PGRN rescues pathology in Grn null mice and achieves widespread cortical expression in a large animal model
OR42: Barbara Bettegazzi, University Vita Salute San Raffaele Milan
A combinatorial gene therapy for temporal lobe epilepsy based on NPY and one of its receptors
08:30-10:45
Lomond Suite
Parallel 4c
Advanced therapies with CAR-T cells
Chairs: Ulrike Koehl, Fraunhofer IZI, Leipzig / Alessia Potenza, Università Vita-Salute San Raffaele, Milan
INV31: Monica Casucci, SR-Tiget, Milan
Exploiting N-glycosylation blockade to boost CAR-T cell efficacy in solid tumours
INV32: Maria Themeli, Amsterdam University Medical Center
Multi-targeting and combinatorial costimulation to improve CAR T cell therapy.
OR33: Isabel Lane, Harvard Medical School
Genetically retargeting E3 ligases to enhance CAR T cell therapy
OR34: Anne Galy, Genethon, France
Immunotherapy treatment with FAP-specific CAR-T cells can reduce skeletal muscle fibrosis in a murine model of Duchenne muscular dystrophy
OR35: Pedro Luis Justicia Lirio, GENYO, Granada
Generation of first-in-class doxycycline-inducible IL-18 releasing CAR-T cells targeting solid tumors
OR36: Christos Georgiadis, UCL, London
CRISPR-coupled CAR engineering of universal donor T cells for paediatric B-ALL
OR37: Mateo Doglio, SR Tiget, Milan
CAR-Tregs for Systemic Lupus Erythematosus
10:45-11:15
Break
11:15-13:25
Pentland Suite
Plenary 4
Presidential symposium
In memoriam: Dr Manfred Schmidt
Chairs: Hildegard Büning, Hannover Medical School / Juan Bueren, Ciemat, Madrid
Hildegard Büning, Hannover Medical School
Presidential address
INV37: Eugenio Montini, SR-Tiget, Milan
New frontiers in genotoxicity testing and clonal tracking methodologies
Presentation of the
ESGCT Outstanding Achievement Award
Christof von Kalle, Charité Berlin
Manfred Schmidt, In Memoriam
Young Investigator Award
INV39: Eliana Ruggiero, SR-Tiget, Milan
The TCR Therapy Revival: Gene-Edited WT1-Specific T Cells Treat Leukemia and Solid Tumours
Young Investigator Award
INV40: Raul Torres, CNIO Madrid
Genome engineering for cancer applications
Award ceremony for 2021 awards
13.25-15:30
Lunch
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13:45-14:45
Pentland
Lunchtime syposium
Enhanced support to academic developers of ATMPs
Patrick Celis, EMA
Academic ATMP development support pilot at the European Medicines Agency
Alessandro Aiuti, SR Tiget, Milan
The view of an academic developer
13:45-14:45
Fintry
Lunchtime sponsor
symposium
A lightbulb moment for gene editing
Chair: Lesley Eschinger, MaxCyte
Speaker: Elena Stoyanova, Touchlight Genetics
13:45-14:45
Sidlaw
Lunchtime sponsor
symposium
Unleashing the Allogeneic Potential: Applying Separation Technologies in Large Scale Manufacture to Achieve Off-the-Shelf Products in Advanced Therapies
Chair: Noushin Dianat, External Collaborations Manager, Sartorius
Guest Speaker: Lior Raviv, Vice-President & CTO, Pluri
Speaker: Michal Szelwicki, Product Specialist, Sartorius
15:30-17:30
Pentland /Sidlaw
Parallel 5a
AAV next generation vectors
Chairs: Jude Samulski, AskBio / Terry Flotte, University of Massachusetts Medical School
INV47: Leszek Lisowski, CMRI, University of Sydney
Designer therapeutics: pushing the limits of
AAV capsid engineering.
INV48: Ken MacNamara, AskBio
PromPT: data-driven vector design for precise control of gene expression
OR55: Waldemar Schäfer, University Medical Center Hamburg
Nanobodies as versatile ligands to retarget AAV
OR56: Jennifer Marx, Hannover Medical School
Capsid-engineered adeno-associated virus (AAV) vector for neurotrophin gene therapy in inner ear disease
OR57: Guangping Gao, Umass Chan Medical School
Endogenous human SMN1 promoter-driven gene replacement improves the efficacy and safety of AAV9-mediated gene therapy for spinal muscular atrophy (SMA) in mice
OR58: Jacqueline Bogedein, LMU Munich
Mutagenesis of capsid surface residues for improvement of AAV vectors with retinal tropism
15:30-17:30
Fintry
Parallel 5b
Developments in
manufacturing and scale up
Chairs: Mustafa Munye, Complement Therapeutics / Beata Surmacz-Cordle, GSK
INV43: Brian Mullan, Yposkesi, Paris
Cell & Gene therapy manufacturing – current state and future developments
INV44: Ulriche Köhl, Fraunhofer IZI, Leipzig
Manufacturing and quality control of CAR effector cells.
INV45: Eduard Ayuso, Dinamiqs
Seamless AAV manufacturing from proof-of-concept to clinical applications
INV46: Sophie Mountcastle, MRC
New hubs and their capabilities
& Anji Miller, LifeArc
Supporting translation of gene therapies through manufacturing infrastructure and a new research fund for early-phase clinical trials
OR51: Panagiota Cristofi, University of Patras
Characterization and GMP manufacturing of epigenetically reprogrammed HLA-G expressing, T regulatory cells (iG-Tregs)
OR52: Fabrizio Benedicenti, SR Tiget, Milan
Sonication Linker Mediated-PCR (SLiM-PCR), an efficient method for quantitative retrieval of vector integration sites
OR53: Yuriko Makino, Tosoh Corp
Application of novel AAV purification and analysis column with Adeno-Associated Virus Receptor (AAVR)
OR54: Mohamed Ashrafali, Biomarin, Pharmaceutical
Vector genome loss and epigenetic modifications impact long-term transgene expression of AAV5 vectors produced in mammalian HEK293 and insect Sf cells
15:30-17:30
Lomond Suite
Parallel 5c
Blood diseases: Haematopoietic cell disorders
Chairs: Alessandro Aiuti, SR-Tiget, Milan / Jose Carlos Segovia-Sanz, Ciemat, Madrid
INV41: Juan Bueren, CIEMAT, Madrid
Preliminary Conclusions of the Phase I/II Gene therapy Trial in Patients with
Fanconi Anemia-A
INV42: Punam Malik, Cincinnati Children's Hospital
Gene therapy for hematopoietic disorders: Improving Engraftment of Genetically Modified Cells
OR47: Rafi Emmanuel, EmendoBio, Israel
EMD-101, an autologous, allele-specific gene-edited hematopoietic stem cell product, for treating ELANE-mediated severe congenital neutropenia
OR48: Panagiotis Antoniou, Institut Imagine, Paris
Sharpening the adenine and cytosine base editing outcome in hematopoietic stem cells
OR49: Oscar Quintana Bustamante, Ciemat, Madrid
Correction of Congenital Dyserythropoietic Anemia Type II using Lentiviral Gene Therapy
OR50: Samantha Scaramuzza, SR Tiget, Milan
Reconstitution of Humoral and Cellular Immunity in Thalassemic Patients Treated with HSC LV-mediated Gene Therapy Following Myeloablation
17:30-19:15
Break and Poster session II ( Posters with an EVEN number)
