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Monday 19 June

Biennial Congress Registration



Session 1: Inaugural session

Chairs: Gloria Gonzalez Aseguinolaza, University of Navarra, Pamplona, Ander Izeta, Biodonostia, San Sebastian


09:00    Gloria Gonzalez Aseguinolaza, University of Navarra, Pamplona

Opening and welcome

09:15    Ignacio MeleroUniversity of Navarra, Pamplona

INV01   Intratumoral and local cancer Immunotherapy: back to the future

Room: Sala Ciudadela 


Session 2: Gene editing

Chair:    Javier Molina, Juanro Rodríguez Madoz

10:00    Toni Cathomen, University of Freiburg

INV02   What can we learn from meaningful assessments of on- and off-target effects in genome editing?

10:30   Natalia Artigas, Integra Therapeutics S.L.

OR01   Characterization and deployment in vivo of FiCAT genome writer

10:45   Araceli Aguilar-González, GENyO/ Universidad de Granada / Junta de Andalucia

OR02   CRISPNA, a new tool for genome editing and diagnosis

11:00   Karim BenabdellahGENyO/ Universidad de Granada / Junta de Andalucia

OR03   Breaking Barriers and Building Bridges: The Impact of COST Action GenE-Humdi in Advancing Genome Editing for Human Disease Treatment

11:15  Laura TorellaFIMA, Universidad de Navarra, Pamplona

P049   AAV-SaCas9 Nickase targeting Hao1 is a safe and efficient therapy for Primary hyperoxaluria type 1 with a low AAV integration rate

11:20  Martin Bilbao Arribas, CIMA, Universidad de Navarra, Pamplona

P050   Optimizing analysis pipelines for the detection of adeno-associated virus (AAV) integration into CRISPR-induced DNA breaks

11:25  Sara Veiga, University of Santiago de Compostela

P054   Generation of brain organoids and zebrafish models to determine and reverse ASD-related phenotypes

Room: Sala Ciudadela 


Coffee Break 



Session 3a: Gene & cell therapy of metabolic diseases

Chair:    Nerea Zabaleta

12:00    Gloria Gonzalez-Aseguinolaza, FIMA, Universidad de Navarra, Pamplona

INV03    Gene therapy for liver metabolic disorders

12:30    Nicholas Weber, Vivet-Therapeutics

OR04    Effective Readministration of AAV Gene Therapy Prevents Tumorigenesis in Infant PFIC3 Mice, a Mouse Model Prone to Hepatocellular Carcinoma

12:45   Sergio Muñoz, CBATEG - Universitat Autònoma de Barcelona

OR05   Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV Gene Therapy

13:00   Juan Ruiz, Forge Biologics Inc

OR06   FBX–101 Clinical program for Patients with Krabbe Disease – Updates on the RESKUE & REKLAIM Phase I/II Gene Therapy Clinical Trials  

13:15   Oihana Murillo, ​CIMA, Universidad de Navarra, Pamplona

P042    Therapeutic efficacy of the combination of VTX-801 and D-Penicillamine for Wilson’s

13:20  Daniel JericoCIMA, Universidad de Navarra, Pamplona

P060    mRNA-based therapy in a new model of variegate porphyria in rabbits

13:25  Angie MolinaCIMA, Universidad de Navarra, Pamplona

P044   Improvement of progressive familiar intrahepatic cholestasis type 2 (PFIC2) via AAV-mediated hepatic BSEP expression in mice

Room: Sala Ciudadela 


Session 3b: Oncolytic viruses & cancer immunotherapy

Chair:    Alvaro Morales, Cristian Smerdou

12:00    Marta Alonso, University of Navarra, Pamplona

INV04   Virotherapy for the Treatment of Pediatric Brain Tumors

12:30   Maria Tristàn-Manzano, Lentistem

OR07   Lentiviral vectors for inducible, transactivator-free advanced therapy medicinal products: application to CAR-T cells

12:45   Marc Otero, IDIBAPS, Barcelona

OR08   KPC pancreatic cancer cells established from a murine pancreatic tumor support human adenovirus replication in vitro and in vivo

13:00   Ana Judith Perisé BarriosUniversidad Alfonso X el Sabio, Villanueva de la Cañada

OR09    Canine high-grade gliomas treated with the oncolytic adenovirus ICOCAV15: a preclinical model

13:15  Ana del Cañizo Lázaro, Universitat de Barcelona

P083    Improving poxvirus-mediated antitumor immune responses by deleting viral cGAMP-specific nuclease

13:20  Pau Fiol, IDIBELL-ICO, Barcelona

P081   Oncolytic adenovirus coding for MICA enhances immune responses against tumors

13:25  Patricia García Rodríguez, Institut de Salud Carlos III

P086    Oncolytic virus and CAR-T cells combination therapy to treat solid tumors.

Room: Luneta


Poster session 1


Session 4a: Gene & cell therapy for haematological disorders

Chair:    Raul Torres, Susana Navarro

16:00    Paula Rio, CIEMAT, Madrid

INV05   Therapeutic Gene Editing in Hematopoetic Stem Cells: Meeting the Challenge in Fanconi Anemia

16:30    Maria Fernandez Garcia, Ciemat/CIBERER, Madrid

OR10    Improved efficacy of mesenchymal stromal cells stably expressing CXCR4 and IL-10 in a xenogeneic graft versus host disease mouse model

16:45    Manuel Palacios Pérez, Ciemat/CIBERER, Madrid

OR11    Development of a gene editing approach for the treatment of RPL5-deficient Diamond-Blackfan anemia patients

17:00    Beatriz OlallaCiemat/CIBERER, Madrid

OR12    Gene targeting of hematopoietic stem and progenitor cells by prime editing

17:15   Iris Ramos HernándezGENyO-Pfizer / Universidad de Granada / Junta de Andalucia 

P039    HSPCs targeted insertion in CX3CR1 enhances myeloid expression of integrated cassettes

17:20  Guillermo Güenechea Amurrio, CIEMAT/CIBERER, Madrid

P024   Lentiviral-mediated gene therapy restores telomere homeostasis in ex vivo assays on dyskeratosis congenita-like CD34+ cells

17:25  Irene Romayor, BioCruces/Hospital Universitario Cruces

P022   Disease-corrected hematopoietic progenitors from APDS2 syndrome derived iPS cells

Room: Sala Ciudadela 


Session 4b: Regenerative medicine

Chair:    Manu Mazo, Ander Izeta

16:00    Antonella Consiglio, Idibell, Barcelona


16:30    Leire Gardeazabal, Biodonostia Institute

OR13    Characterisation of cutaneous wound healing of aged Sox2EGFP mice

16:45    Julia López de Andrés,  University de Granada

OR14     Using patient-derived extracellular matrix for costumized tissue substitutes: Advancements in cellular and tissular therapies

17:00    Ainhoa Irastorza Lorenzo, Biodonostia Institute

OR15    Development of novel muscle bioink based on decellularised tissue and myogenic precursor cells for the treatment of stress urinary incontinence

17:15   Pilar Montero Calle, CIMA, Universidad de Navarra, Pamplona

P094   Composite human cardiac minitissues are a representative 3D model of anthracycline cardiotoxicity

17:20   Paula BarlabéCIMA, Universidad de Navarra, Pamplona

P095    Generation of heart and vascular system from pluripotent stem cells by blastocyst complementation.

17:25  Juan Antonio Marchal, CIBM, Universidad de Granada, Granada"

P098   Bioinks based on decellularised extracellular matrix from cells for osteochondral grafts

Room: Luneta


Coffee Break 



Session 5: Keynote

Chair:     Juan Bueren


18:00    Felipe Prosper, Universidad de Navarra

INV07   Understanding mechanisms of response and resistance of CAR-T cells in hematological malignancies

Room: Sala Ciudadela 

Welcome reception



Tuesday 20 June


Session 6a: In vivo gene & cell therapy non viral

Chair:        Maria Jesus Garrido, Kepa Berraondo

08:30    Gemma Navarro, Curevac

INV08   Optimizing RNA therapeutics delivery

09:00    Eric Rovira Barreira, CIMA, Universidad de Navarra, Pamplona 

OR16    Identification of novel sequences to regulate gene expression in response to approved drugs for application in gene therapy.

09:15   Lydia Alvarez-Erviti, Centro de Investigación Biomédica de La Rioja (CIBIR)

OR17   Delivery of shRNA minicircles by extracelullar vesicles to halt Parkinson’s disease neurodegeneration

09:30   Noelia Silva Pilpich, CIMA, Universidad de Navarra, Pamplona 

OR18   Targeting the PD-1/PD-L1 pathway with nanobodies encoded in self-amplifying RNA vectors induce potent antitumor responses

09:45   Yaiza Jimenez Martinez, Universidad de Granada

P051     ldrB gene: elucidating mechanism of action of a suicide gene.

09:50   Natalia Hernández BellidoI3A, University of Zaragoza)

P064    Non-viral delivery of microRNA therapies for cardiac dysfunction using DNA nanostructures

09:55  Luis SendraInstituto de Investigación Sanitaria La Fe

P065   Hepatic hydrofection of naked hIL10 gene in pig to study its immunomodulatory effect in liver transplantation scenario

Room: Sala Ciudadela 


Session 6b: Gene editing II

Chair:   Laura Torella, Sandra Rodriguez

08:30   Eliana Ruggiero, HSR, Milan

INV09   Engineering T lymphocytes for the treatment of hematological and solid tumors

09:00   Irene Peña Gutierrez, Ciemat/CIBERER, Madrid 

OR19    Mimicking FA mutations in hematopoietic stem and progenitor cells by gene editing approaches 

09:15   Alex Bassons Bascunana, Ciemat/CIBERER, Madrid 

OR20   Highly efficient non-viral gene editing approaches applicable to all COL7A1 mutations known. 

09:30   Ignacio Perez de CastroInstituto de Salud Carlos III

OR21   Investigating the potential of different gene therapy strategies for treating LMNA-associated congenital muscular dystrophy: varied outcomes depending on the pathological contexts

09:45   Simonas Valiuska, University of Barcelona

P052    Combinatorial effect of Polypurine Reverse Hoogsteen against MYC and KRAS oncogenes in the human PC-3 cancer cell line

09:50   Jose Carlos Segovia-SanzCIEMAT/CIBERER, Madrid

P050    Developing new mouse gene editing tools to assess Pyruvate Kinase Deficiency stem cell gene editing in a mouse model of the disease

09:55  Kristina PavlovicGENyO-Pfizer / Universidad de Granada / Junta de Andalucia 

P025   Memory Universal CD19-CAR T cells: a new strategy against B-cell malignancies

Room: Luneta


Coffee Break



Session 7: Engineered cells for cancer immunotherapy I

Chair:   Javier Garcia-Castro, Francisco Martin 

10:30   Luis Alvarez-Vallina, H12O-CNIO Cancer Immunotherapy Clinical Research Unit Clinical Research Programme,

Hospital Universitario 12 de Octubre (H12O), Spanish National Cancer Research Centre (CNIO) 

INV10   Novel T cell-redirecting strategies for enhanced cancer immunotherapy: Dynamic targeting.

11:00    Diego Sanchez Martinez,  Josep Carreras Institute and CIBERINFEC, Barcelona

OR22    A novel fratricide-resistant CAR-T cell immunotherapy for T-ALL

11:15    Alba Rodriguez Garcia, IDIBAPS, Barcelona

OR23    Sensitivity of CAR-T cells to PD-1/PD-L1-mediated inhibition is modulated by CAR affinity

11:30    Pedro Luis Justicia Lirio, Lentistem

OR24    Development of first-in-class doxycycline-inducible IL-18 secreting CAR-T cells targeting aggressive tumors

11:45   Claudia Augusta Di TraniCIMA, Universidad de Navarra, Pamplona 

P082    Intra-tumoral delivery of mRNA encoding IL-12-fused to diabodies targeting CSF1R and PD-L1 exert potent anti-tumor efficacy while restraining systemic exposure to IL-12.

11:50  Catalina Barceló, IDIBAPS, Barcelona

P005    Competition of co-transduced CARs for cellular resources in dual-targeting CAR T cells

11:55  Paula Heredia VelázquezGENyO-Pfizer / Universidad de Granada / Junta de Andalucia 

P009   Improving exosome production and immunogenicity for more effective CAR T cell therapy

Room: Sala Ciudadela 


Session 8: The future of advanced therapies in Spain

Chair: Paula Rio and Ander Izeta


12:00    Raul Torres, CIEMAT/CIBERER, Madrid

INV11a   Desarrollo de Terapias Avanzadas en España

12:30    Nerea Zabaleta, Harvard Medical School /  Mass Eye and Ear

INV11b  Rational engineering of AAV libraries reveals capsid residues that determine tropism

Room: Sala Ciudadela 



Poster session 2



Session 9: Engineered cells for cancer immunotherapy II

Chair:   JuanJo Lasarte, Sonia Guedan

15:30   Cristina Puig-Saus, UCLA

INV12   Engineering a potent T cell response against solid tumors

16:00    Mireia Bachiller, IDIBAPS, Barcelona

OR25    ARI0003: Co-transduced CD19/BCMA dual-targeting CAR-T cells for the treatment of non-Hodgkin lymphoma

16:15    Paula Rodríguez Márquez, CIMA, Universidad de Navarra, Pamplona

OR26    Prolonged haematologic toxicity after BCMA CAR-T cell treatment: from clinical data to ex-vivo studies at a single center

16:30    Andrea Lopez, CIEMAT/CIBERER, Madrid

OR27    Trogocytosis in CAR-T cells against HNSCC cell lines

16:45   Angela CovoCIMA, Universidad de Navarra, Pamplona

P001    Semliki Forest virus-based vaccines encoding CAR antigens to foster the activation, expansion and antitumor efficacy of CAR-T cells. 

16:50  Lorea Jordana, CIMA, Universidad de Navarra, Pamplona

P006   Identification of molecular mechanisms governing CAR-T cell response in MM patients using single cell transcriptomics

16:55  Patricia Garcia RodriguezInstituto de Salud Carlos III, Madrid   

P011    CAR T therapy efficacy in hypoxic osteosarcoma spheroids.

Room: Sala Ciudadela 

Coffee Break



Session 10a: Manufacturing

Chair:   Maria Eugenia Fernandez, Begoña Diez

17:30   Gemma Fernández de Miguel, Pharmaceutical Inspection and Enforcement Department

INV13  Global Overview of Advanced Therapy Medicines (ATMPs) Manufacturing

18:00    Gloria Carmona, Junta de Andalucia

INV14   Quality Controls in Open and Closed Processes

18:30    Maria Camanyes Novellón, IDIBAPS, Barcelona

OR28    Quality control establishment for gene therapy medicinal products

18:45   Silvia Torrents Zapata, Banc de Sang i Teixits

P070    Optimized reagents for immunopotency assays on mesenchymal stromal cells for clinical use

18:50  Ana Carreras GonzálezViralgen Vector Core

P072   Addressing the Challenges of Capillary Electrophoresis for AAV Purity Characterization

18:55 Katherine Orange-Bromehead,TAAV Manufacturing Solutions  

P075 TAAV-manufactured enzymatic DNA: an efficient technology to overcome rAAV manufacturing challenges

Room: Sala Ciudadela 


Session 10b:  Gene & cell therapy for neurological disorders

Chair:    Carmen Unzu, Ruben Hernandez

17:30    Juan Jo Toledo Aral, University of Sevilla

INV016 Antiparkinsonian cell therapy: pros and cons of the coronavirus pandemic.

18:00    Sara Marcó CostaCBATEG - Universitat Autònoma de Barcelona

OR29     A new gene therapy approach to treat Niemann-Pick Type C2 disease

18:15    Anna Mateu BoschIDIBAPS, Barcelona

OR30    GCDH gene replacement therapy ameliorates glutaric aciduria type I phenotype in mice.

18:30   Sergi VerdésUniversitat Autònoma de Barcelona (UAB)

OR31    A novel gene therapy approach for ALS by overexpressing in muscles the pleiotropic chronokine α-Klotho

18:45  Eulàlia Segur-BailachIDIBAPS, Barcelona  

P038   Substrate reduction gene therapy rescues glutaric aciduria type I in mice.

18:50  Ruben Hernandez-AlcocebaCIMA, Universidad de Navarra, Pamplona

P026    Strategies to obtain preferential transgene expression in GABAergic neurons using adenoviral vectors

18:55  Joan Roig Soriano, Universitat Autònoma Barcelona

P028    AAV9-Mediated expression of secreted Klotho reduced several aging-associated phenotypes and increased longevity

Room: Luneta

Session 11: Closing keynote

Chair:     Gloria Gonzalez-Aseguinolaza, Ander Izeta


19:00    Juan Bueren, CIEMAT, Madrid

INV17    Gene Therapy in Fanconi Anemia and LAD-I diseases: Two models of stem and mature cell diseases

19:45 Awards

Room: Sala Ciudadela 

20:30 onwards
Networking evening


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