
PROGRAMME
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Education Session
08:00-09:00
Registration
09:00-10:30
Parallel 1a: Gene delivery platform
Chair: Virginia Haurigot, Spark Therapeutics
09:00 Virginia Haurigot, Spark Therapeutics
INV01 AAV vectors, from basics to challenges
09:25 Xavier Anguela, Ask Bio ⓘ
INV02 Non-viral gene transfer for rare diseases
09:50 Pedro Berraondo, Program of Immunology and Immunotherapy, Cima Universidad de Navarra ⓘ
INV03 Advances in mRNA-based therapeutics
10:15 Round Table
Room: Descubrimiento
09:00-10:30
Parallel 1b: Cellular Therapy
Chair: Javier Garcia Castro, Instituto de Salud Carlos III, Madrid
09:00 Pablo Menendez, Josep Carreras Leukaemia Research Institute, Barcelona ⓘ
INV04 CAR-based adoptive T-cell immunotherapies
09:25 Aida Platero-Luengo, Instituto de Biomedicina de Sevilla (IBiS), Hospital Universitario Virgen del Rocío/CSIC ⓘ
INV05 Crossing new frontiers in regenerative medicine: the reprogramming technology in cell therapy and aging
09:50 Javier Garcia Castro, Instituto de Salud Carlos III, Madrid ⓘ
INV06 The state of the art in Cellular Therapy
10:15 Round Table
Room: Ibiza
10:30-11:00
Break
11:00-12:30
Parallel 2a: Gene editing tools
Chair: Paula Rio, CIEMAT, Madrid
11:00 Javier Molina Estevez, GENYO, Granada ⓘ
INV07 Gene Editing 101: Cleaving DNA to correct mutations
11:25 Laura Ugalde, CIEMAT, Madrid
INV08 Base and prime editing strategies to correct mutations in human hematopoietic stem cells
11:50 Sonia Guedan, IDIBAPS, Barcelona ⓘ
INV09 Genome-editing technologies in CAR-T cell therapy for cancer
12:15 Round Table
Room: Descubrimiento
11:00-12:30
Parallel 2b: Disease modelling
Chair: Angel Raya, IDIBELL, Barcelona
11:00 Ander Izeta, Biodonostia, San Sebastian ⓘ
INV10 Disease modelling by single-cell RNA sequencing (scRNAseq)
11:25 Angel Raya, IDIBELL, Barcelona ⓘ
INV11 Dissecting human disease complexity with iPSC-based models
11:50 Pilar Martin Duque, Universidad de Zaragoza
INV12 Imaging techniques for in vivo detection of advanced therapies in disease models
12:15 Round Table
Room: Ibiza
Monday 30 May
12:30-13:30
Lunch and Biennial Congress Registration
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13:30-14:45
Plenary 1: Inaugural session
Chair: Gloria Gonzalez Aseguinolaza, University of Navarra, Pamplona, JuanJo Toledo-Aral, Universidad de Sevilla
13:30 Gloria Gonzalez Aseguinolaza, University of Navarra, Pamplona ⓘ
SETGYC since 2019: Society update
13:45 Mariano Esteban, CNB-CSIC, Cantoblanco, Madrid
INV13 Lessons and challenges in the development of the vaccine MVA-CoV2-S
Room: Descubrimiento
14:45-16:15
Parallel 3a: Gene delivery platforms
Chair: Karim Benabdellah, GENYO, Granada
14:45 Els Verhoeyen, ENS Lyon, Université de Nice ⓘ
INV15 Novel lentiviral pseudotypes for T and NK cell gene therapy and ‘nanoblades’ for efficient gene editing in hematopoietic gene therapy target cells and organoids
15:15 Pedro Berraondo, Program of Immunology and Immunotherapy, Cima Universidad de Navarra ⓘ
INV16 Messenger RNA in cancer immunotherapy
15:45 Pilar Martin Duque, Universidad de Zaragoza
OR01 Use and isolation of maternal milk exosomes: New vector for genetic hNIS therapy and diagnostic in oncology.
16:00 Carlos Blanco-Benitez, LentiStem Biotech. GENYO. Centre for Genomics and Oncological Research: Pfizer / University of Granada / Andalusian Regional Government.PTS Granada
OR02 Lent-On-Plus: An all-in-one transactivator-free doxycycline inducible LVs ready for clinical applications.
Room: Descubrimiento
14:45-16:15
Parallel 3b: Pluripotent stem cells & organoids
Chair: Ricardo Pardal, Instituto de Biomedicina de Sevilla (IBiS), Hospital Universitario Virgen del Rocío/CSIC/
14:45 Nuria Montserrat, Institute for Bioengineering of Catalonia (IBEC)
INV17 Human pluripotent stem cells derived organoids: challenges and opportunities to study human development and disease
15:15 Isabel Fariñas, Departamento de Biología Celular, Biología Funcional y Antropología Física, Universidad de Valencia ⓘ
INV18 The impact of neural stem cell heterogeneity on niche dynamics
15:45 Beatriz Fernandez-Muñoz, Red Andaluza de Diseño y Traslación de Terapias Avanzadas, Sevilla
OR03 Using organoids to evaluate safety and potency of cell therapies
16:00 Ainhoa Irastorza-Lorenzo, Biodonostia Institute, San Sebastian
OR04 Development of biomimetic tympanic membrane substitutes for the treatment of chronic perforations
Room: Ibiza
16:15-17:15
Break and Poster session 1
17:15-18:45
Parallel 4a: Oncolytic viruses
Chair: Ramon Alemany, IDIBELL, Barcelona
17:15 Ramon Alemany, IDIBELL, Barcelona ⓘ
INV19 Cancer Virotherapy with Oncolytic Adenoviruses
17:45 Manuel Ramirez, Hospital Infantil Universitario Niño Jesús, Universidad Autónoma de Madrid
INV20 An update on the Celyvir translational research program
18:15 Alvaro Morales-Molina, Instituto de Salud Carlos III, Madrid
OR05 Using silent cells improves tumor homing and efficacy of of cell therapy for cancer
18:30 Cristian Smerdou, CIMA, Pamplona
OR06 Expression of Galectin-3 inhibitors from a self-replicating RNA vector as treatment for pediatric osteosarcoma
Room: Descubrimiento
17:15-18:45
Parallel 4b: Cell and gene therapy of metabolic diseases
Chair: Fatima Bosch, UAB Barcelona
17:15 Fatima Bosch, UAB Barcelona ⓘ
INV21 Gene therapy for maturity-onset diabetes of the young type 3 (MODY3)
17:45 Maria Ester Bernardo, SR Tiget, Milan
INV22 Update on the clinical trial results of HSC-gene therapy for mucopolysaccharidosis type 1 Hurler
18:15 Maria Garcia-Bravo, Division of Cell Technology, Biomedical Innovation Unit, CIEMAT, CIBERER, IIS-FJD, UAM, Madrid
OR07 Hyperoxaluric phenotype improvement after in vivo lentiviral vector gene therapy in a preclinical model of Primary Hyperoxaluria Type 1
18:30 Araceli Aguilar-González, GENyO- Centro de Genomica e Investigacion Oncologica: Pfizer / Universidad de Granada / Junta de Andalucia
OR08 Isogenic GAA-KO murine muscle cell lines mimicking severe Pompe mutations as preclinical models for screening of potential gene therapy strategies
Room: Ibiza
18:45-20:15
Plenary 2: Regulatory & Manufacturing
Chair: Juan Ruiz, Forge Therapeutics
18:45 Marcos Timon, AEMPS
INV23 Regulatory considerations for ATMP development
19:15 Cesar Trigueros, Viralgen
INV24 AAV serotype associated impurities under a production platform.
19:45 Julio Delgado, IDIBAPS, Barcelona ⓘ
INV25 Development of academic CART-cell products in Europe: is it possible?
Room: Descubrimiento
21:00
Gala Dinner
Meet in the hotel lobby between 20:30-20:45 to depart to las Setas by autobus. View venue map if you plan to meet us there.
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Tuesday 31 May
08:00-09:00
Registration
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09:00-09:30
Plenary 3: Safety
Chair: Juan Bueren, CIEMAT, Madrid
09:00 Zoltan Ivics, Paul Ehrlich Institute, Langen ⓘ
INV26 Non-viral engineering of therapeutic cells with the Sleeping Beauty transposon system
Room: Descubrimiento
09:30-10:00
Advances therapies move to patients
Chair: Gloria Gonzalez-Aseguinolaza, University of Navarra, Pamplona
09:30 Juan Bueren, CIEMAT, Madrid ⓘ; Cristian Smerdou, CIMA Universidad de Navarra, Pamplona
INV27 Towards the implementation of gene therapy in the clinical practice
Room: Descubrimiento
10:00-11:30
Parallel 5a: CAR-T cells
Chair: José Antonio Pérez-Simón, Institute of Biomedicine of Sevilla
10:00 Antonio Perez-Martinez, Hospital Universitario La Paz, Madrid ⓘ
INV28 Cell therapy based on the Chimeric receptor NKG2D
10:30 Valentin Ortiz, Hospital Clínic de Barcelona
INV29 CART19-BE-01: A European academic trial on the administration of ARI-0001 cells in patients with acute lympoblastic leukemia and other CD19+ lympoproliferative disorders
11:00 Angel Martin Mallo, Cima Universidad de Navarra. IdiSNA.
OR09 Effect of the disease stage over the phenotype and functionality of CAR-T cells in multiple myeloma patients
11:15 Diego Sanchez-Martínez, Josep Carreras Leukemia Research Institute
OR10 Efficient treatment of cortical T cell acute lymphoblastic leukemia with T lymphocytes secreting anti-AntigenA T cell engagers
Room: Descubrimiento
10:00-11:30
Parallel 5b: Cell and gene therapy of Neural diseases
Chair: Juan José Toledo-Aral, Instituto de Biomedicina de Sevilla (IBiS), Hospital Universitario Virgen del Rocío/CSIC/ Universidad de Sevilla
10:00 Benedikt Berninger, Institute of Psychiatry, Psychology & Neuroscience Centre for Developmental Neurobiology & MRC Centre for Neurodevelopmental Disorders, King's College London ⓘ
INV30 Advancing in vivo glia-to-neuron reprogramming as an innovative approach for brain repair: promises, disappointments, and breakthroughs
10:30 Juan Ruiz, Forge Therapeutics
INV31 The RESKUE Trial - a Phase 1/2 Clinical Trial for Patients with Krabbe Disease
11:00 Sergi Verdés, Institut de Neurociències (INc), Universitat Autònoma de Barcelona (UAB)
OR11 Gene therapy for ALS by specifically overexpressing a pleiotropic chronokine, secreted α-Klotho (s-KL), in skeletal muscles
11:15 Nieves Iria Domínguez-García, Instituto de Biomedicina de Sevilla (IBiS)
OR12 Pathophysiological alterations of regulatory T cells in Parkinson's disease and generation of aSyn-CAR-Tregs as a novel therapeutic approach.
Room: Ibiza
11:30-12:30
Coffee Break and Poster Session 2
12:30-14:00
Parallel 6a: Cell and gene therapy of blood disorders
Chair: Jose Carlos Segovia, CIEMAT, Madrid
12:30 Jose Carlos Segovia, CIEMAT, Madrid ⓘ
INV32 Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency
13:00 Simón Méndez-Ferrer, Wellcome-MRC Cambridge Stem Cell Institute, Cambridge, UK
INV33 Targeting the stem cell niche in the myeloid malignancies
13:30 Gonzalo Martinez-Navajas, University of Granada
OR13 Correction of haematopoietic stem cells from Bernard-Soulier syndrome patients and disease models by gene therapy.
13:45 Yari Gimenez-Martinez, CIEMAT, CIBERER, IIS-FJD, UAM, Madrid
OR14 Towards Lentiviral Gene Therapy for RPS19-Diamond Blackfan Anemia Patients
Room: Descubrimiento
12:30-14:00
Parallel 6b: Cell and gene therapy of skin, skeletal & cardiac muscles
Chair: Ander Izeta, Biodonostia, San Sebastian
12:30 Fernando Larcher, Ciemat Madrid
INV34 Correction of recessive dystrophic epidermolysis bullosa using genome editing strategies
13:00 Manuel Mazo, Universidad de Navarra, Pamplona ⓘ
INV35 Computational biomechanics, 3D printing and hiPSCs: towards the generation of a biological ventricular assist device
13:30 Leire Gardeazabal, Biodonostia Institute, San Sebastian
OR15 Alterations in extracellular matrix deposition by wound bed fibroblasts during cutaneous wound healing of aged Sox2EGFP mice
13:45 Angeles Mencia Rodriguez, CIEMAT, Madrid
OR16 Genome editing for genodermatoses therapy: correction of a Netherton syndrome patient´s cells carrying a deep intronic pathogenic variation.
Room: Ibiza
14:00-15:00
Lunch
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15:00-16:30
Plenary 4: Clinical Trials
Chair: Guillermo Guenechea Amurrio, CIEMAT, Madrid
15:00 Carlos Fernandez de Larrea, IDIBAPS, Barcelona ⓘ
INV36 CAR-T cells for the treatment of multiple myeloma
15:30 Juan Bueren, CIEMAT, Madrid ⓘ
INV37 Advances in Hematopoietic Stem Cell Gene Therapy
16:00 Federico Mingozzi, Spark Therapeutics ⓘ
INV38 In vivo gene therapy for hemophilia, progress and lessons learned
Room: Descubrimiento
16:30-18:00
Parallel 7a: Gene editing tools
Chair: Francisco Martin, Genyo, Granada
16:30 Karim Benabdellah, Genyo, Granada
INV39 Fueling Cancer Immunotherapy with Genome editing
17:00 Marc Guëll, Universitat Pompeu Fabra, Barcelona
INV40 Gene writing in human cells
17:30 Raul Torres Ruiz, Centro Nacional de Investigaciones Oncológicas, CNIO, Madrid
INV41 CRISPR targeting of fusion oncogenes for selective elimination of cancer cells
17:45 Mercedes Dessy Rodríguez, CIEMAT, Madrid
OR17 Correction of congenital dyserythropoietic anemia type II using lentiviral gene therapy
Room: Descubrimiento
16:30-18:00
Parallel 7b: Cell and gene cancer immunotherapy
Chair: JuanRo Rodriguez, CIMA Universidad de Navarra
16:30 Juan-Jose Lasarte, Cima Universidad de Navarra
INV42 Addressing the Tumor Microenvironment to enhance the efficacy of CART cell therapy in solid tumors
17:00 Alba Rodriguez, IDIBAPS, Barcelona ⓘ
INV43 Strategies to improve CAR-T cell therapy for solid tumors
17:30 Angel Fernandez-Ramirez, UNICA, Hospital Universitario 12 de Octubre, Madrid
OR18 Synapse topology and downmodulation events determine the functional outcome of anti-CD19 T cell-redirecting strategies
17:45 Berta Marzal, IDIBAPS, Barcelona
OR19 Preclinical development of low-affinity HER2-specific CAR-T cells for the treatment of HER2+ breast cancer
Room: Ibiza
18:00-18:30
Break
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18:30–19:30
Plenary 5: Awards and closing plenary
Chair: Gloria Gonzalez Aseguinolaza, University of Navarra, Pamplona
In memoriam of Juan Carlos Ramirez
Jose Lopez Barneo, Instituto de Biomedicina de Sevilla (IBiS), Hospital Universitario Virgen del Rocío/CSIC/Universidad de Sevilla
INV47 Acute Oxygen Sensing in Health and Disease
Room: Descubrimiento
20:00
Poster session 3 and closing drinks
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Wednesday 1 June
Ex Vivo and In Vivo Gene Editing: Present and Future Clinical Applications
08:30-09:00
Registration
09:00-10:30
Plenary 6: New gene editing tools
Chairs: Raul Torres, Centro Nacional de Investigaciones Oncológicas, CNIO, Madrid; Marc Güell, Universitat Pompeu Fabra, Barcelona
09:00 Julian Grünewald, Technical University of Munich
INV48 Engineered CRISPR prime editors with compact, untethered reverse transcriptases
09:30 Pilar Puig-Serra, CNIO, Madrid ⓘ
INV49 Targeting and detection of cancer fusion oncogenes using CRISPR/Cas13
10:00 Guillermo Güenechea Amurrio, CIEMAT, CIBERER, Madrid
OR20 Editing of Telomerase RNA Component gene in hematopoietic stem cells: An alternative tool to treat bone marrow failure in patients with autosomal dominant dyskeratosis congenita
10:15 Isabel Ojeda-Perez, CIEMAT, CIBERER, Mardid
OR21 Studying Hematopoietic Stem Cell clonality in Gene Editing Strategies using DNA Barcoding
10:30-12:00
Plenary 7: Gene editing ex vivo
Chair: Francisco Martin, Paula Rio, CIEMAT, Madrid
10:30 Annarita Miccio, Imagine Institute, Paris
INV50 Genome editing approaches for beta-hemoglobinopathies
11:00 Alessia Cavazza, UCL, London ⓘ
INV51 Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome
11:30 Matthew Porteus, Stanford University ⓘ
INV52 Genome Editing of Hematopoietic Stem Cells
12:00-12:30
Break
12:30-13:45
Plenary 8: in vivo Gene editing applications
Chairs: Gloria Gonzalez Aseguinolaza, University of Navarra, Pamplona; Juan Bueren, CIEMAT, Madrid
12:30 Gloria Gonzalez Aseguinolaza, University of Navarra, Pamplona ⓘ
INV53 In vivo gene editing to treat Primary hyperoxaluria type I
13:00 Jordi Barquinero, Vall d'Hebron Research Institute, Barcelona ⓘ
INV54 In vivo gene editing for Mitochondrial NeuroGastroIntestinal Encephalomyopathy
13:30 Ignacio Perez de Castro, Instituto de Salud Carlos III, Madrid
OR22 CRISPR-based strategies revert the LMNAR249W induced phenotype both in cultured cells and in vivo
13:45-14:45
Lunch
14:45-16:30
Plenary 9: Gene editing in cancer immunotherapy
Chair: Sonia Guedan, IDIBAPS, Barcelona; Karim Benabdhellah, GENYO, Granada
14:45 Laurent Poirot, Cellectis ⓘ
INV55 Repurposing endogenous immune pathways to tailor and control chimeric antigen receptor T cell functionality
15:15 JuanRo Rodríguez, CIMA Universidad de Navarra
INV56 Genome editing for improved allogeneic CAR-T cells
15:45 Sandra Rodríguez Perales, Spanish National Cancer Centre (CNIO), Madrid ⓘ
INV57 CRISPR systems for cancer study, diagnosis and treatment
16:15 Noelia Maldonado Pérez, GENyO- Centro de Genomica e Investigacion Oncologica: Pfizer / Universidad de Granada / Junta de Andalucia
OR23 Feasibility of generating universal ARI-0001 T cells for the treatment of type B malignances
16:30-17:00
Break
17:00-18:15
Plenary 10: Safety studies in gene editing
Chair: Annarita Miccio, Imagine Institute, Paris
17:00 Giandominco Turchiano, University College London ⓘ
INV59 Comprehensive genome stability characterization in ex-vivo gene edited primary cells
17:30 Anastasia Conti, SR Tiget, Milan ⓘ
INV60 Mechanistic insights into the response of Hematopoietic Stem Cells to Gene Editing
18:00 Gloria Gonzalez-Aseguinolaza, University of Navarra, Ander Izeta, Biodonostia, San Sebastian
Closing Remarks
Room: Descubrimiento