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PROGRAMMEWEDNESDAY 4 NOVEMBER 2015

EDUCATION SESSION 1

Parallel Sessions 1a, 1b, 1c

9.00-10.30 Barandiaran

1a: Cell and viral vector manufacturing and distribution under GMP Chair: Ana Sánchez, University of Valladolid

9.00-9.20 INV001 Cell manufacturing and distribution under GMP Ana Sánchez, University of Valladolid

9.20-9.40 INV002 Cell and gene based IMPs for cancer treatment – a summary of major new strategies Farzin Farzaneh, King’s College London

9.40-10.00 INV003 Manufacturing of cell-based medicinal products under GMP environment Begoña Castro, Histocell

10.00-10.30 ROUND TABLE Current challenges in GMP manufacturing and distribution of ATMPs

9.00-10.30 Aranzadi

1b: Systems biology: computational methods applied to advanced therapies Chair: Marcos J. Araúzo-Bravo, Biodonostia

9.00-9.20 INV004 Deconstructing the mouse Embryonic Stem Cell Epigenetic Network Alfonso Valencia, CNIO

9.20-9.40 INV005 Machine learning in biosciences Borja Calvo, Facultad de Informática, UPV-EHU

9.40-10.00 INV006 Computational Biology Powers the Application of Primordial Germ Cells to Regenerative Medicine Marcos J. Araúzo-Bravo, Biodonostia

10.00-10.30 ROUND TABLE The Big Data revolution: how can computational biology help advanced therapies?

WEDNESDAY 4 NOVEMBER 2015


9.00-10.30 Elhuyar

1c: Genome modification Chair: Juan Carlos Ramirez, VIVEbioTECH

9.00-9.20 INV007 Gene Editing in induced pluripotent- and adult-stem cells for the Treatment of Hematopoietic Diseases José Carlos Segovia, CIEMAT/CIBERER/IIS-FJD

9.20-9.40 INV008 Improving delivery of gene edition tools for cell-gene therapy Francisco Martin, GENYO

9.40-10.00 INV009 Modeling diseases by genome engineering technologies Raul Torres-Ruiz, CNIC

10.00-10.30 ROUND TABLE Gene editing tools: recent evolutions and remaining challenges for clinical application

10.30-11.00 Coffee Break

EDUCATION SESSION 2



2a: Regulatory issues in advanced therapies: current situation in Spain Chair: Natividad Cuende, Andalusian Initiative for Advanced Therapies (AIAT)

11.00-11.20 INV010 Regulatory issues in advanced therapies: current issues in Spain Sol Ruiz, AEMPS

11.20-11.40 INV011 Development of “industrial” products: Alofisel case María Pascual Martinez, Tigenix

11.40-12.00 INV012 The public service perspective as complement of the market perspective in the development of advanced therapies Natividad Cuende, AIAT

12.00-12.30 ROUND TABLE ATMPs in Spain. The situation from a regulatory point of view.



11.00-12.30 Aranzadi

2b: High throughput tools for single cell analysis Chair: Ander Izeta, Biodonostia

11.00-11.20 INV013 Longitudinal survival analysis to study alpha-synuclein dependent neurodegeneration Montserrat Arrasate, CIMA

11.20-11.40 INV014 High throughput tools for single cell analysis Rosa Prieto, VHIR

11.40-12.00 INV015 Trans-generational memory and forgetting Ben Lehner, CRG

12.00-12.30 ROUND TABLE Single cell analysis: is it worthwhile?

11.00-12.30 Elhuyar

2c: Disease modelling using iPSC Chair: Angel Raya, CMRB

11.00-11.20 INV016 Possibilities and limitations of iPSC-based human disease modeling Angel Raya, CMRB

11.20-11.40 INV017 Easy- and difficult-to-reprogram cells and iPSC-based disease modeling Pablo Menendez, IJC

11.40-12.00 INV018 How immunogenic are autologous iPSCs? Jordi Barquinero, VHIR

12.00-12.30 ROUND TABLE iPS cell-based therapies. What are the major roadblocks?

12.30-13.30 Lunch



Session 3: Inaugural Lectures

13.30-15.00 Elhuyar

Chairs: Javier Garcia-Sancho, University of Valladolid Ramón Alemany, ICO

13.30-13.40 INV019 SETGYC in 2015: Society update Javier Garcia-Sancho, University of Valladolid

SETGyC Inaugural Lectures

13.40-14.20 INV020 Gene Therapy for Inherited Muscle Diseases Fulvio Mavilio, Généthon

14.20-15.00 INV021 AAV- APPsα brain delivery rescues synaptic failure in an Alzheimers disease mouse model Nathalie Cartier, INSERM-University Paris Descartes


15.00-16.30 Elhuyar

4a: Mesenchymal Stem Cells Chair: José Becerra, BIONAND

15.00-15.20 INV022 Mesenchymal stromal cells (MSC) in hematopoietic cell transplantation Consuelo del Cañizo, Salamanca University Hospital

15.20-15.40 INV023 Adipose Tissue Mesenchymal Stem Cells Properties and Clinical Application Damián García-Olmo, Universidad Autonoma de Madrid

15.45-16.00 OR001 Therapeutic potential of adipose stromal cells for burn wounds Isabel Sánchez Muñoz, Getafe University Hospital

16.00-16.15 OR002 Human embryonic stem cell-derived mesenchymal stromal cells ameliorate collagen-induced arthritis through induction of host-derived indoleamine 2,3 dioxygenase. Gabriel Criado, IJC

16.15-16.30 OR003 IFNγ immuno-regulates activated Vγ9Vδ2+ T cells Through a Feedback Mechanism mediated by Mesenchymal Stromal Cells César Trigueros, Inbiomed


4b: Haematopoietic Stem Cells Chair: Guillermo Güenechea, CIEMAT/CIBERER/IIS-FJD

15.00-15.20 INV024 Moving from the preclinical to the clinical gene therapy of Fanconi anemia Juan Bueren, CIEMAT /CIBERER/IIS-FJD



15.20-15.40 INV025 Notch signalling in the control of Hematopoietic Stem Cell development Anna Bigas, IMIM

15.45-16.00 OR004 Intra-bone marrow transplantation confers superior multi-lineage engraftment of murine AGM cells than intravenous transplantation Damiá Romero, IJC

16.00-16.15 OR005 Mesenchymal stromal cells reduce the incidence of graft failure in a mouse model of hematopoietic stem cell gene therapy Rosa Yañez, CIEMAT/CIBERER/IIS-FJD

16.15-16.30 OR006 Gene targeting in Hematopoietic Stem Cells: a New Tool for Future Application in Fanconi Anemia Paula del Río, CIEMAT/CIBERER/IIS-FJD


4c: Viral vector development: long term gene transfer Chair: Gloria G. Aseguinolaza, CIMA

15.00-15.20 INV026 LentiSoma™ a new generation of integrase-deficient lentiviral vectors Juan Carlos Ramirez, VIVEbioTECH

15.20-15.40 INV027 Long-Term Gene Transfer with non-integrative vectors Miguel Chillón, CBATEG

15.45-16.00 OR007 Lentiviral Vectors Pseudotyped with Measles Virus HF Envelope Proteins Efficiently Target Hematopoetic Progenitor Cells in Unfractionated Bone Marrow from Fanconi Anemia Patients Francisco J. Román, CIEMAT/CIBERER/IIS-FJD

16.00-16.15 OR008 AAV integration in the liver in a model of induced genotoxicity Nerea Zabaleta, CIMA

16.15-16.30 OR009 Enhanced production of herpesviral (HSV-1) amplicon vectors by gene modification and medium optimization of packaging cells Iván Fernández Frías, CBMSO





5a: Tissue-resident adult stem cells Chair: José López-Barneo, IBIS

17.00-17.20 INV028 Physiological plasticity of adult carotid body stem cells Ricardo Pardal, IBIS

17.20-17.40 INV029 Stem cell-niche interactions in the adult brain Isabel Fariñas, University of València

17.45-18.00 OR010 AAV-delivered donor template and adenoviral vector-delivered TALENs allow efficient gene edition for the correction of a recurrent mutation in the COL7A1 gene causing recessive dystrophic epidermolysis bullosa in the Spanish population Cristina Chamorro, CIEMAT/CIBERER/IIS-FJD

18.00-18.15 OR011 Unraveling the developmental origin of neural competent cells in mouse ventral skin: role of SOX2 expression levels Araika Gutiérrez-Rivera, Biodonostia

18.15-18.30 OR012 Lack of p73 impairs ependymal Planar Cell Polarity establishment and ciliogenesis Sandra Fuertes, University of León


5b: Gene editing Chairs: Jose Carlos Segovia, CIEMAT/CIBERER/IIS-FJD Eusebio Gainza, Praxis

17.00-17.20 INV030 Targeted Gene Correction of Pyruvate Kinase Deficiency in patient-specific Stem Cells Oscar Quintana-Bustamante, CIEMAT/CIBERER/IIS-FJD

17.20-17.40 INV031 Genomic engineering of the Cystic Fibrosis gene in patient-derived iPS cells Daniel Bachiller, CSIC

17.45-18.00 OR013 Specific elimination of latent HIV infected cells through a “shock and kill” suicidal gene therapy Pedro Perdigão, University of Lisbon

18.00-18.15 OR014 CRISPR/Cas9 knock-out of the PKLR gene in human cells and hematopoietic progenitors as a cellular model of Pyruvate Kinase Deficiency Sergio Lopez, CIEMAT/CIBERER/IIS-FJD

18.15-18.30 OR015 CRISPR-Cas9 technology: new optimized protocol to model human chromosomal translocations in primary cell Raúl Torres-Ruiz, CNIC



17.00-18.30 Elhuyar

5c: Viral vector for short term gene transfer Chair: Cristian Smerdou, CIMA

17.00-17.20 INV032 Oncolytic adenoviruses for cancer virotherapy Ramon Alemany, ICO

17.20-17.40 INV033 Oncolytic viruses armed with potent immunostimulatory cytokines: strategies to improve their safey and efficacy Rubén Hernández, CIMA

17.45-18.00 OR016 Nanoparticle modification of adenoviruses for CAR-negative cells infection Rebeca González-Pastor, CIBA

18.00-18.15 OR017 Insertion of an albumin-binding domain in adenovirus hexon allows evasion of neutralizing antibodies upon systemic administration Luis A. Rojas, IDIBELL-ICO

18.15-18.30 OR018 A new propagative vector derived from alphaviruses devoid of the capsid gene Cristian Smerdou, CIMA

Session 6

18.30-19.00 Elhuyar

6. Elevator Pitch (Industry Presentations) Chair: Damian Garcia Olmo, IIS-FJD

18.30-18.35 Elevator 1 Citospin, a University of Valladolid spin off dedicated to GMP cell production for clinical use. Ana Sánchez García, Citospin SL

18.35-18.40 Elevator 2 Building an academic GMP facility for the production of advanced therapy medicinal products Raquel Martín Ibáñez, University of Barcelona

18.40-18.45 Elevator 3 Inbiomed M Paz López Mato, Inbiomed

18.45-18.50 Elevator 4 Praxis Eusebio Gainza, Praxis

18.50-18.55 Elevator 5 Gradocell, a consulting company specializing in the development of Advanced Therapy Medicinal Products and Innovative medicines Rossana García Castro, Gradocell, SL

18.55-19.00 Elevator 6 KARUNA GOOD CELLS TECHNOLOGIES: A new start-up in the field of genome editing and cell-based therapies Mónica Castresana, Karuna Good Cells Technologies

19.00-20.00 Welcome Reception: NiNeu Restaurant, Kursaal


PROGRAMMETHURSDAY 5 NOVEMBER 2015

Plenary Session 7

9.00-10.00 Elhuyar

Chair: Juan Bueren, CIEMAT

9.00-9.30 INV047 Cell therapy for muscular dystrophies: lessons learned and the road to efficacy Giulio Cossu, University of Manchester, UCL

9.30-10.00 INV035 Converting viral vectors into genome editing devices Manuel Gonçalves, Leiden University


10.00-11.30 Elhuyar

8a: Pluripotent stem cells and cell reprogramming Chair: Daniel Bachiller, Fundación Caubet-Cimera

10.00-10.20 INV036 Human oocyte as source of information to study cell pluripotency and reprogramming Elena Gonzalez-Muñoz, LARCEL

10.20-10.40 INV037 NF-κB activation impairs somatic cell reprogramming in ageing Clara Soria, University of Oviedo

10.45-11.00 OR019 p73 deficiency impairs mesenchymal to epithelial transition (MET) during somatic cell reprogramming Marta Martín, University of León

11.00-11.15 OR020 Reprogramming human B-cells into induced pluripotent stem cells and its enhancement by C/EBPα Alvaro Muñoz, IJC

11.15-11.30 OR021 Hepatocytes derived from human VAL9 embryonic stem cells protect mice from liver injury after transplantation Laia Tolosa, IIS La Fe


8b: Non-viral strategies: immunomodulation, exosomes and RNA-mediated therapy Chair: Virginia Arechavala-Gomeza, Biocruces

10.00-10.20 INV038 Non-viral gene therapy for inherited retinal disorders Gustavo Puras, UPV/EHU

10.20-10.40 INV039 Antisense-mediated splicing modulation in neuromuscular disorders Virginia Arechavala-Gomeza, Biocruces

10.45-11.00 OR022 Generation of human myeloid-derived suppressor cells from monocytes and hematopoietic progenitors for their potential clinical application Silvia Casacuberta, VHIR

THURSDAY 5 NOVEMBER 2015


11.00-11.15 OR023 Preliminary assessment of dimercaptosuccinic acid-coated superparamagnetic nanoparticles loading on NK92MI cell line to improve NK cell delivery to tumour cells Laura Sanz Ortega, CNB-CSIC


8c: Nanotechnology, biomaterials and tissue/organ engineering Chair: Manuel Arruebo, University of Zaragoza

10.00-10.20 INV040 Non-viral vectors for the treatment of ocular diseases: application to X-linked juvenile retinoschisis (XLRS) Alicia Rodríguez Gascón, UPV-EHU

10.20-10.40 INV041 Application of nanostructured materials as gene delivery vectors Manuel Arruebo, University of Zaragoza

10.45-11.00 OR025 Identification and characterization of murine dermal precursor cells with myogenic potential Neia Naldaiz-Gastesi, Biodonostia Institute

11.00-11.15 OR026 Stem cell therapy with limbal cells for corneal epithelium regeneration Ana Sánchez, University of Valladolid

11.15-11.30 OR027 Preclinical and clinical studies for development of a tissue engineering treatment for bone loses of substance Luis M. Redondo, Río Hortega University Hospital


Parallel Sessions 9a, 9b

12.00-13.30 Elhuyar

9a: Targeting stem cells and cell-based approaches in cancer therapy Chair: Joan Seoane, VHIO

12.00-12.20 INV042 Cancer-initiating cells in brain tumours Joan Seoane, VHIO

12.20-12.40 INV043 Signaling and metabolic requirements of breast cancer initiating cells Arkaitz Carracedo, CIC Biogune



12.45-13.00 OR028 A therapeutic NKT cell adjuvant-based vaccine induces an effective antitumoral immune response in B-cell lymphoma Laura Escribá, Sant Pau Hospital

13.00-13.15 OR029 Mesenchymal Stem Cells as Hollow Gold Nanoparticles vectors for tumor treatments Marimar Encabo, University of Zaragoza

13.15-13.30 OR030 Human menstrual blood-derived mesenchymal stem cells as cell carriers for in vivo oncolytic adenovirus tumor delivery Rafael Moreno, IDIBELL-ICO


9b: Rare Diseases: the front runner of gene and cell therapy Chair: Fatima Bosch, CBATEG

12.00-12.20 INV044 Gene Therapy for acute intermittent porphyria Gloria González Aseguinolaza, CIMA

12.20-12.40 INV045 Intracerebrospinal Fluid AAV-mediated Gene Therapy for Mucopolysaccharidosis Fátima Bosch, CBATEG

12.45-13.00 OR031 Systemic AAV8-MTM1 gene therapy for X-linked myotubular myopathy Ana Buj Bello, Genethon

13.00-13.15 OR032 Intrathecal AAVrh10 corrects biochemical and histological hallmarks of Mucopolysaccharidosis VII mice and improves bone pathology, behavior and survival Assumpcio Bosch, UAB

13.15-13.30 OR033 Improving gene edition tools for Wiskott-Aldrich Syndrome gene therapy Karim Benabdellah, Genyo

13.30-14.30 Lunch and Poster Session 1

Plenary Session 10

14.30-15.30 Elhuyar

Chair: Jose Carlos Segovia, CIEMAT

14.30-15.00 INV046 The bone marrow stem cell niche as a potential therapeutic target in myeloproliferative neoplasias Simón Méndez-Ferrer, CNIC

15.00-15.30 INV034 Molecular Mechanisms for Assembling and Resolving Distinct Pluripotent States Jacob Hanna, Weizmann Institute of Science





11a: Preclinical proof of concept studies based in animal models Chair: Jose Luis Labandeira, Univ. of Santiago de Compostela

15.30-15.50 INV048 Preclinical validation of a new generation of anti-Myc cell-penetrating peptides Laura Soucek, VHIO

15.50-16.10 INV049 Geriatric muscle regeneration and growth is regulated by interplay between quiescence and senescence Pura Muñoz-Canoves, UPF

16.15-16.30 OR034 Long-term efficacy and safety in mice and dogs following delivery of AAV9-sulfamidase vectors to the CSF for the treatment of MPSIIIA Sara Marcó, CBATEG

16.30-16.45 OR035 In vivo AAV-mediated IGF1 expression to the pancreas as a new gene therapy for type 1 diabetes Cristina Mallol, CBATEG

16.45-17.00 OR036 Pre-clinical development of lentiviral gene therapy for SCID-X1 Valentina Poletti, Genethon


11b: Immunotherapy Chair: Ramon Alemany, ICO

15.30-15.50 INV050 CD137-Based treatments in combinatorial immunotherapy of cancer Ignacio Melero, CIMA

15.50-16.10 INV051 Selection of circulating CD8+PD-1+ cells leads to direct enrichment of cancer mutation-specific lymphocytes and T-cell receptors Alena Gros, NCI-NIH

16.15-16.30 OR037 T-cell engager-armed oncolytic adenoviruses: an immunotherapy approach to improve virotherapy Carlos A. Fajardo, IDIBELL-ICO

16.30-16.45 OR038 Complementary effects of interleukin-15 and interferon-α restore anti-HBV immunity in vitro and in vivo Marianna Di Scala, CIMA

16.45-17.00 OR039 Efficient expression of bioactive murine IL12 as a self-processing P2A polypeptide driven by inflammation-regulated promoters in tumor cell lines Guillermo Garaulet, UAM



15.30-17.00 Elhuyar

11c: Cell therapy in the nervous system Chair: Juan J. Toledo, IBIS

15.30-15.50 INV052 From midbrain dopaminergic neuron development to novel cell replacement strategies for Parkinson’s disease Ernest Arenas, Karolinska Institutet

15.50-16.10 INV053 Autologous bone marrow intra-spinal grafts develop a neurotrophic activity in CNS motor areas of ALS patients Salvador Martínez, University of Murcia

16.15-16.30 OR040 Quantitative high-throughput gene expression profiling of human development to routinely screen stem cells derived products Marco Straccia, University of Barcelona

16.30-16.45 OR041 Neuronal differentiation of Human Neural Stem Cells is regulated by APP expression Isabel Liste, ISCIII

16.45-17.00 OR042 Role of Rho-Kinase inhibition on ventral mesencephalic grafts in a rat model of Parkinson’s disease Juan A. Parga, University of Santiago de Compostela


17.30-19.00 Poster Session 2

19.00-19.30 SETGyC General Assembly

20.00 Society Gala Dinner at Palacio de Miramar


PROGRAMMEFRIDAY 6 NOVEMBER 2015

Plenary Session 12

9.00-10.00 Elhuyar

Chairs: Juan Bueren, CIEMAT; Javier Garcia-Sancho, University of Valladolid

9.00-9.30 INV054 Fighting Tumor Escape by Gene Modified Cells Massimo Dominici, University of Modena

9.30-10.00 INV055 Hematopoietic stem cell gene therapy for genetic disorders Alessandro Aiuti, San Raffaele Hospital


10.00-11.30 Elhuyar

13a: Imaging and in vivo cell tracking Chair: Pilar Martin Duque, UFV

10.00-10.20 INV056 Three-dimensional quantitative imaging of the bone marrow microenvironment in health and disease César Nombela-Arrieta, University of Zürich

10.20-10.40 INV057 Assessing tumoral microenvironment: hypoxia with F18-MISO PET-CT in Cancer Research Francisca Mulero, CNIO

10.45-11.00 OR043 Genetic tracking of psoriatic epidermal cell lineages in mutant mice Nuria Gago-López, CNIO

11.00-11.15 OR044 Poor cartilage index (PCI), a procedure for image analysis of MRI T2 mapping that improves quantitative follow-up of cartilage repair in osteoarthritis Javier García-Sancho, University of Valladolid

11.15-11.30 OR045 Simultaneous tracking of Bone Marrow-MSCs migration towards different pathologies. Promise for cancer cell therapies Alejandra Crespo, UFV


13b: Chimeric antigen receptor T-cell therapy for cancer Chair: Pablo Menendez, IJC

10.00-10.20 INV058 The use of chimeric antigenic receptors targeting CD19 as a therapeutic approach for B-cell malignancies Julio Delgado, ICMHO

10.20-10.40 INV059 New Approaches using CAR T cells for Cancer Sonia Guedan, University of Pennsylvania

10.45-11.00 OR046 NKG2D CAR expressing T cells target osteosarcoma cells Lucía Fernández Casanova, IdiPAZ

FRIDAY 6 NOVEMBER 2015

PROGRAMMEFRIDAY 6 NOVEMBER 2015

11.00-11.15 OR047 Novel Approaches for the generation of memory stem T cells for adoptive immunotherapy Carmen Alvarez-Fernandez, Sant Pau Hospital


13c: Tissue resident adult stem cells (II): muscle and cardiovascular system Chair: Antonio Bernad, CNB-CSIC

10.00-10.20 INV060 Adult cardiac stem cells in mammalian heart homeostasis and repair are defined by Bmi1 expression Antonio Bernad, CNB-CSIC

10.20-10.40 INV061 Myocardial infarction scar formation: endogenous cell dynamics & the development of cell therapies to treat the diseased heart José Maria Pérez-Pomares, University of Malaga

10.45-11.00 OR048 Characterisation of induced pluripotential stem cells from a patient with the transposition of the great arteries cardiac disease Imelda Ontoria, La Fe Hospital

11.00-11.15 OR049 Glucose starvation in cardiomyocytes modify exosome cargo molecules to promote angiogenesis in endothelial cells Nahuel A. García, La Fe Hospital

11.15-11.30 OR050 Can a protease modulate the aging process of muscle? Miriam Bobadilla, CIMA


Session 14

12.00-13.30 Elhuyar

14: Closing Lecture and Award Ceremony Chair: Marcos J Araúzo-Bravo, Biodonostia

12.00-13.00 INV062 POU Factors dictating Mammalian Multi- and Pluripotency Hans Schöler, Max Planck Institute for Molecular Biomedicine, Münster, Germany

13.00-13.30 Award Ceremony Chairs: Javier Garcia-Sancho, University of Valladolid; Ramón Alemany, ICO

FRIDAY 6 NOVEMBER 2015

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