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Thursday 13 October



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

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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

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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




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


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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

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Lunchtime sponsor


A lightbulb moment for gene editing

Chair: Lesley Eschinger, MaxCyte


Speaker: Elena Stoyanova, Touchlight Genetics

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Lunchtime sponsor


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

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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

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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

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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

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Break and Poster session II ( Posters with an EVEN number)

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The Molecular Mingle

National Museum of Scotland. More Information >

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