Education Session



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


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



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


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

Lunch and Biennial Congress Registration


Transparent Logo.png


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



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


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

Break and Poster session 1


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


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


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

Viralgen Programme.png

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. 


Transparent Logo.png

Tuesday 31 May




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


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


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


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

Coffee Break and Poster Session 2


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


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



Transparent Logo.png


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

Izasa Programme.png


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


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




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

Poster session 3 and closing drinks



Wednesday 1 June
Ex Vivo and In Vivo Gene Editing: Present and Future Clinical Applications



Plenary 6: New gene editing tools

Chairs: Raul Torres, Centro Nacional de Investigaciones Oncológicas, CNIO, MadridMarc 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 


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



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 



Plenary 9: Gene editing in cancer immunotherapy

Chair: Sonia Guedan, IDIBAPS, BarcelonaKarim 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



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