Monday 19 June
08:30-09:00
Biennial Congress Registration
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09:00-10:00
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
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09:15 Ignacio Melero, University of Navarra, Pamplona
INV01 Intratumoral and local cancer Immunotherapy: back to the future
Room: Sala Ciudadela
10:00-11:30
Session 2: Gene editing
Chair: Javier Molina, Juanro Rodríguez Madoz
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10:00 Toni Cathomen, University of Freiburg
INV02 What can we learn from meaningful assessments of on- and off-target effects in genome editing?
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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 Benabdellah, GENyO/ 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
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11:15 Laura Torella, FIMA, 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
11:30-12:00
Coffee Break
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12:00-13:30
Session 3a: Gene & cell therapy of metabolic diseases
Chair: Nerea Zabaleta
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12:00 Gloria Gonzalez-Aseguinolaza, FIMA, Universidad de Navarra, Pamplona
INV03 Gene therapy for liver metabolic disorders
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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
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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 Jerico, CIMA, Universidad de Navarra, Pamplona
P060 mRNA-based therapy in a new model of variegate porphyria in rabbits
13:25 Angie Molina, CIMA, 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
12:00-13:30
Session 3b: Oncolytic viruses & cancer immunotherapy
Chair: Alvaro Morales, Cristian Smerdou
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12:00 Marta Alonso, University of Navarra, Pamplona
INV04 Virotherapy for the Treatment of Pediatric Brain Tumors
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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 Barrios, Universidad Alfonso X el Sabio, Villanueva de la Cañada
OR09 Canine high-grade gliomas treated with the oncolytic adenovirus ICOCAV15: a preclinical model
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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
13:30-14:30
Lunch
14:30-16:00
Poster session 1
16:00-17:30
Session 4a: Gene & cell therapy for haematological disorders
Chair: Raul Torres, Susana Navarro
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16:00 Paula Rio, CIEMAT, Madrid
INV05 Therapeutic Gene Editing in Hematopoetic Stem Cells: Meeting the Challenge in Fanconi Anemia
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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 Olalla, Ciemat/CIBERER, Madrid
OR12 Gene targeting of hematopoietic stem and progenitor cells by prime editing
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17:15 Iris Ramos Hernández, GENyO-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
16:00-17:30
Session 4b: Regenerative medicine
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Chair: Manu Mazo, Ander Izeta
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16:00 Antonella Consiglio, Idibell, Barcelona
INV06 TBC
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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
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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 Barlabé, CIMA, 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
17:30-18:00
Coffee Break
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18:00-19:00
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
19:00-20:00
Welcome reception
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Tuesday 20 June
08:30-10:00
Session 6a: In vivo gene & cell therapy non viral
Chair: Maria Jesus Garrido, Kepa Berraondo
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08:30 Gemma Navarro, Curevac
INV08 Optimizing RNA therapeutics delivery
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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
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09:45 Yaiza Jimenez Martinez, Universidad de Granada
P051 ldrB gene: elucidating mechanism of action of a suicide gene.
09:50 Natalia Hernández Bellido, I3A, University of Zaragoza)
P064 Non-viral delivery of microRNA therapies for cardiac dysfunction using DNA nanostructures
09:55 Luis Sendra, Instituto 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
08:30-10:00
Session 6b: Gene editing II​
Chair: Laura Torella, Sandra Rodriguez
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08:30 Eliana Ruggiero, HSR, Milan
INV09 Engineering T lymphocytes for the treatment of hematological and solid tumors
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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 Castro, Instituto 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
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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-Sanz, CIEMAT/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 Pavlovic, GENyO-Pfizer / Universidad de Granada / Junta de Andalucia
P025 Memory Universal CD19-CAR T cells: a new strategy against B-cell malignancies
Room: Luneta
10:00-10:30
Coffee Break
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10:30-12:00
Session 7: Engineered cells for cancer immunotherapy I
Chair: Javier Garcia-Castro, Francisco Martin
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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.
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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
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11:45 Claudia Augusta Di Trani, CIMA, 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 Velázquez, GENyO-Pfizer / Universidad de Granada / Junta de Andalucia
P009 Improving exosome production and immunogenicity for more effective CAR T cell therapy
Room: Sala Ciudadela
12:00-13:00
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
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12:30 Nerea Zabaleta, Harvard Medical School / Mass Eye and Ear
INV11b Rational engineering of AAV libraries reveals capsid residues that determine tropism
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Room: Sala Ciudadela
13:00-14:00
Lunch
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14:00-15:30
Poster session 2
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15:30-17:00
Session 9: Engineered cells for cancer immunotherapy II
Chair: JuanJo Lasarte, Sonia Guedan
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15:30 Cristina Puig-Saus, UCLA
INV12 Engineering a potent T cell response against solid tumors
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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
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16:45 Angela Covo, CIMA, 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 Rodriguez, Instituto de Salud Carlos III, Madrid
P011 CAR T therapy efficacy in hypoxic osteosarcoma spheroids.
Room: Sala Ciudadela
17:00-17:30
Coffee Break
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17:30-19:00
Session 10a: Manufacturing
Chair: Maria Eugenia Fernandez, Begoña Diez
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17:30 Gemma Fernández de Miguel, Pharmaceutical Inspection and Enforcement Department
INV13 Global Overview of Advanced Therapy Medicines (ATMPs) Manufacturing
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18:00 Gloria Carmona, Junta de Andalucia
INV14 Quality Controls in Open and Closed Processes
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18:30 Maria Camanyes Novellón, IDIBAPS, Barcelona
OR28 Quality control establishment for gene therapy medicinal products
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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 González, Viralgen 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
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Room: Sala Ciudadela
17:30-19:00
Session 10b: Gene & cell therapy for neurological disorders​
Chair: Carmen Unzu, Ruben Hernandez
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17:30 Juan Jo Toledo Aral, University of Sevilla
INV016 Antiparkinsonian cell therapy: pros and cons of the coronavirus pandemic.
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18:00 Sara Marcó Costa, CBATEG - Universitat Autònoma de Barcelona
OR29 A new gene therapy approach to treat Niemann-Pick Type C2 disease
18:15 Anna Mateu Bosch, IDIBAPS, Barcelona
OR30 GCDH gene replacement therapy ameliorates glutaric aciduria type I phenotype in mice.
18:30 Sergi Verdés, Universitat Autònoma de Barcelona (UAB)
OR31 A novel gene therapy approach for ALS by overexpressing in muscles the pleiotropic chronokine α-Klotho
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18:45 Eulàlia Segur-Bailach, IDIBAPS, Barcelona
P038 Substrate reduction gene therapy rescues glutaric aciduria type I in mice.
18:50 Ruben Hernandez-Alcoceba, CIMA, 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
19:00-20:00
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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