Welcome

Dear Colleagues,

It is my pleasure to invite you to the 2021 virtual meeting of the Israeli Society of Gene and Cell Therapy (ISGCT 2021), set to take place online, on February 11th, 2021.

Gene and Cell therapy are finally taking center stage in global health. No longer a futuristic dream of the hopeful few but rather a true revolutionary reality, with several drugs already on the market and hundreds of advanced clinical trials. We can proudly reflect on the important role of Israeli researchers in the rising of the field, with seminal contributions to stem cell research, T cell engineering, genome editing, liver gene therapy and so much more.

Remaining challenges are nevertheless formidable. How can we improve delivery and prolong the effect? How can we increase therapeutic potency while providing safety? How can we devise new remedies for the yet incurable devastating human ailments, and do so in a scalable and affordable manner?

Meeting these challenges will require the innovation and dedication of individual researchers, but it will also depend on nourishing strong scientific communities. The Israeli Society of Gene and Cell therapy has thus set as its goals to support the sharing of data and ideas, promote collaborations, address common challenges and advance clinical translation.

In the upcoming 2021 ISGCT meeting, we are privileged to have Professor Dirk Busch give a Keynote lecture. We are also most excited by the impressive list of speakers from the Israeli academia, clinic and industry, destined to make this event a great kick-off for our rejuvenating society. Please do join us at the 2021 ISGCT meeting. 

See you online! 

Adi Barzel, PhD, Tel Aviv University,
President of Israeli Society of Gene and Cell Therapy

Organizing Committee

                      
          Adi Barzel                 Dinorah Friedmann-Morvinski                Ayal Hendel

Keep Me Updated

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Keynote Speaker

  • Prof. Dirk H. Busch
    Technical University of Munich
    Germany

    Prof. Dirk H. Busch

Prof. Dirk H. Busch

Vice Chair Executive BoardTechnical University of MunichGermany

Bio:

Education:
Study of Human Medicine, University of Freiburg and University of Mainz (1987 – 1992)
MD at University of Mainz (1993)
Habilitation in Microbiology and Immunology at Technical University of Munich (2003)


Professional Career:
since 2009 Director, Institute for Medical Microbiology, Immunology and Hygiene, Technical University of Munich
2004 - 2008 C3-Professor for Medical Microbiology and Immunology, Technical University of Munich
1999 - 2004 Senior Researcher, Technical University of Munich
1996 - 1999 Postdoctoral Researcher at the Section of Infectious Diseases and Immunobiology, Yale University, New Haven/USA
1994 - 1996 Postdoctoral Researcher at the Laboratory of Pediatric Rheumatology, University of Würzburg
1990 - 1993 Predoctoral Research Fellow (M.D.), Endocrinology, University of Mainz


Selected Honours and Awards
Elected member of the National Academy of Sciences Leopoldina (2017); Visiting Scientist of the Australasian Society for Immunology (2015); Member of the Institute of Advanced Study (IAS) at TUM (2010); Science Day Award, Helmholtz Zentrum München (2008); Wilhelm Vaillant Prize, Munich (2003); Hans Krebs Prize, Hannover (2002); Robert Koch Postdoc Award (2002); Gerhard Hess Research Award from DFG (2000); Howard Hughes Medical Institute Postdoc Fellowship (1999)


Main domain of research:
My work centers on antigen-specific T cells and the development of new technologies to make immune cells usable for diagnostic and cell therapy applications.
5 selected publications (last 10 years)
1. Schober, K., F. Voit, S. Grassmann, T.R. Muller, J. Eggert, S. Jarosch, B. Weissbrich, P. Hoffmann, L. Borkner, E. Nio, L. Fanchi, C.R. Clouser, A. Radhakrishnan, L. Mihatsch, P. Luckemeier, J. Leube, G. Dossinger, L. Klein, M. Neuenhahn, J.D. Oduro, L. Cicin-Sain, V.R. Buchholz, and D.H. Busch (2020). Reverse TCR repertoire evolution toward dominant low-affinity clones during chronic CMV infection. Nat Immunol. 21:434-441.
2. Schober, K., T.R. Muller, F. Gokmen, S. Grassmann, M. Effenberger, M. Poltorak, C. Stemberger, K. Schumann, T.L. Roth, A. Marson, and D.H. Busch (2019). Orthotopic replacement of T-cell receptor alpha- and beta-chains with preservation of near-physiological T-cell function. Nat Biomed Eng. 3:974-984.
3. Paszkiewicz PJ, Frassle SP, Srivastava S, Sommermeyer D, Hudecek M, Drexler I, Sadelain M, Liu L, Jensen MC, Riddell SR, Busch DH. Targeted antibody-mediated depletion of murine CD19 CAR T cells permanently reverses B cell aplasia. J Clin Invest 2016;126:4262-4272.
4. Nauerth M, Weissbrich B, Knall R, Franz T, Dossinger G, Bet J, Paszkiewicz PJ, Pfeifer L, Bunse M, Uckert W, Holtappels R, Gillert-Marien D, Neuenhahn M, Krackhardt A, Reddehase MJ, Riddell SR, Busch DH. TCR-ligand koff rate correlates with the protective capacity of antigen-specific CD8+ T cells for adoptive transfer. Sci Transl Med 2013;5:192ra87.
5. Buchholz VR, Flossdorf M, Hensel I, Kretschmer L, Weissbrich B, Graf P, Verschoor A, Schiemann M, Hofer T, Busch DH. Disparate individual fates compose robust CD8+ T cell immunity. Science 2013;340:630-5.

Title:

Orthotopic T cell receptor (TCR) replacement: A new concept for engineering TCR edited T cells for adoptive immunotherapy

Abstract:

Adoptive transfer of T cells with transgenic antigen-specific T cell receptors (TCRs) has the potential to therapeutic options of infectious diseases and cancer. However, generation of defined T cell products with reliable functionality still poses a major challenge. Conventionally, genetic engineering by viral transduction is used, which leads to un-targeted transgene integration and interference through the endogenous TCR. Furthermore, careful titration of viral transduction doses is necessary to limit potential off-target effects through multiple integrations. We recently reported that CRISPR/Cas9-mediated orthotopic TCR replacement (OTR) offers the possibility of controlled transgene integration and simultaneous removal of the endogenous TCR. By genetic engineering of many different antigen-specific TCRs, we now show that knock-out of the endogenous TCR and targeted insertion through OTR independently contribute to highly defined transgenic TCR expression. In comparison to low-dose virally transduced TCR transgenic T cells, enhanced and more defined TCR expression through OTR leads to higher functionality and to a less variable T cell responses. Therefore, OTR seems indeed advantageous for the generation of more predictable TCR-transgenic T cell products.

INVITED SPEAKERS

  • Dr. Yaron Carmi
    Tel-Aviv University
    Israel

    Dr. Yaron Carmi
  • Dr. Noam Diamant
    Noga Therapeutics
    Israel

    Dr. Noam Diamant
  • Prof. Tal Dvir
    Tel Aviv University
    Israel

    Prof. Tal Dvir
  • Dr. Dinorah Friedmann-Morvinski
    Tel Aviv University
    Israel

    Dr. Dinorah Friedmann-Morvinski
  • Dr. Anat Globerson-Levin
    Sourasky/Ichilov
    Israel

    Dr. Anat Globerson-Levin
  • Prof. Erez Levanon
    Bar Ilan University
    Israel

    Prof. Erez Levanon
  • Prof. Michal Lotem
    Hadassah University Hospital
    Israel

    Prof. Michal Lotem
  • Prof. Marcelle Machluf
    Technion
    Israel

    Prof. Marcelle Machluf
  • Prof. Daniel Offen
    Tel Aviv University
    Israel

    Prof. Daniel Offen
  • Prof. Benjamin Reubinoff
    HUJI-Hadassah
    Israel

    Prof. Benjamin Reubinoff
  • Prof. Ronit Satchi-Fainaro
    Tel Aviv University
    Israel

    Prof. Ronit Satchi-Fainaro

Dr. Yaron Carmi

Tel-Aviv University
Israel

Bio:

Over the last 17 years, Dr Carmi has focused on studying how tumor-infiltrating macrophages and dendritic cells interact with T cells and with the surrounding stroma to promote tumor progression and escape immunity. He published over 35 peer-review papers and five patents and is a co-founder in Bolt Therapeutics, which raised over a 100 million US dollars and is now in a Phase I clinical trial. Since 2016 is has is own group in Tel Aviv university. His team published a number of papers including in the Journal of Clinical Investigation a paper describing a novel subset of T cells expressing FcgRI. Based on this discovery we formed a start-up company, which raised over one million US dollars and develops a universal CAR T cell for solid tumors.

Title:

A novel CAR-like therapy for solid cancer

Abstract:

We have recently discovered a novel subset of CD4+ T cell that express the high affinity receptor for IgG (FcgRI) in both mouse and human and efficiently kill tumor cells coated with antibodies. While the factors that induce these cells are currently unknown, we recapitulate their killing capacity by transducing conventional CD8+ T cells with altered FcgRI along with its signaling chain. The unique design of this genetic construct enables integration of complex signals, which are impossible to transmit using conventional CAR designs, thus inducing more potent cytotoxic activity. Moreover, since targeting tumor cells is mediated by antibodies, this technology can be applied to treat a wide range of cancers as well as refractory cancers that lost their expressed antigen, by changing the tumor-binding antibody.

Dr. Noam Diamant

Co-Founder and CEO Noga TherapeuticsIsrael

Bio:

Dr. Noam Diamant is the co-founder and CEO of Noga Therapeutics, a gene therapy company focusing on lentiviral vectors. Noam is a biopharma professional with a strong scientific background and extensive knowledge and experience in intellectual property and in cell and gene therapy. Prior to Noga Therapeutics, he co-founded Emendo biotherapeutics, a company that develops next generation gene editing tools for genetic disorders and Ingenium IP a business IP consultancy firm that provides IP strategy and management services to biopharma companies. Dr. Diamant holds a PhD in the field of DNA damage repair from the Weizmann Institute.

Title:

Development of a Codon Optimized BTK Lentiviral Vector for Gene Therapy of X-linked Agammaglobulinemia

Abstract:

Prof. Tal Dvir

Tel Aviv UniversityIsrael

Bio:

Tal Dvir is a Professor at Tel Aviv University, Israel. He obtained his B.Sc. (2003) and Ph.D (2008) degrees from the faculty of Engineering at Ben-Gurion University of the Negev in Israel. His Ph.D research focused on cardiac tissue engineering and regeneration.  Tal continued his postdoctoral studies in the laboratory of Prof. Robert Langer in the Department of Chemical Engineering at MIT. His postdoc research focused on advanced materials for tissue engineering and regeneration. On October 2011 Tal was recruited by the Department of Biotechnology and the Center for Nanotechnology at Tel Aviv University to establish the Laboratory for Tissue Engineering and Regenerative Medicine. In 2013, Tal joined the newly established Department of Materials Science and Engineering at Tel Aviv.

Tal’s lab designs and develops smart biomaterials and nanotechnologies for engineering complex tissues and organs. This includes the heart, brain, spinal cord, intestine, eyes and more.  Tal has published numerous high impact papers and received numerous awards and prizes. He is also an inventor of numerous patents.

Tal is currently the director of the Tel Aviv University Center for Nanoscience and Nanotechnology and the founding director of the Sagol Center for Regenerative Biotechnology.

Title:

Advanced technologies for engineering heart tissues

Abstract:

In this talk I will describe cutting-edge technologies for engineering functional cardiac tissues and whole hearts. I will focus on the design of new biomaterials mimicking the natural microenvironment, or releasing biofactors to promote stem cell recruitment and tissue protection. In addition, I will discuss the development of patient-specific materials and 3D-printing of personalized vascularized tissues and organs. Finally, I will show a new direction in tissue engineering, where micro and nanoelectronics are integrated within engineered tissues to form cyborg tissues.

Dr. Dinorah Friedmann-Morvinski

Tel Aviv UniversityIsrael

Bio:

Title:

Adoptive T-cell Immunotherapy targeting both glioma cells and tumor derived endothelial

Abstract:

Dr. Anat Globerson-Levin

Sourasky/IchilovIsrael

Bio:

Dr. Globerson Levin earned an MSc (2006) in Cell Biology, at Tel Aviv University and PhD (2011) at the Weizmann Institute, where she studied under Prof. Zelig Eshhar supervision. Her PhD focused on developing the CAR T cell therapy towards solid tumor. From 2013-Today, she is a senior scientist and leading the Immunology Research lab. at Tel Aviv Sourasky Medical Center.

Dr. Globerson Levin research has been focused on developing immunotherapy treatment using the chimeric antigen receptor (CAR) concept. Her effort today is overcoming the immunosuppressive environment, overcoming toxicity related to the treatment and developing new methods to redirect CAR T toward the tumor site. She developed a dual CAR T cell treatment towards multiple myeloma, which is now moving ahead to a clinical trial. She was involved in the establishment of the immunotherapy center at TASMC (I-ACT), which will be treating patients using cell therapy.

Dr. Globerson received with Eshhar several grants including the ROI/NIH, CRBC, ISF and ISF, Kamin, and Persol foundation grant that were selectively given to her. She also possess several patents related to CAR T inventions.

Title:

New strategies for targeting ovarian tumors using CAR T cells

Abstract:

High-grade serous carcinoma (HGSC) is a type of epithelial ovarian cancer (EOC). EOC is the fourth most common cause of cancer-related death in women in the developed world and the leading cause of death from gynecological malignancies.  EOC is often diagnosed at advanced-stage disease with an overall 5-year survival rate of <40%. While a significant progress has been made in surgical and chemotherapeutic treatments for EOC, the survival rates for this disease have only modestly improved. We constructed a ‘dual-Chimeric Antigen Receptor’ (dCAR) for ovarian cancer. The dCAR represent a new approach that addresses the major challenge of 'off-tumor on-target' toxicity in CAR therapy, namely, the risk of damage to healthy tissues of the patient which express the target antigen of the selected CAR. The underlying idea is that restricting the activity of CAR T cells against a combination of two antigens which are co-expressed by the tumor, but not by normal tissues, can prevent off target toxicity. We analyzed the potential efficacy and safety of the dual CAR in vitro. Additionally, we compared the administration of CAR T cells locally to intra venues injection and compared it to i.v injections. We show that intra- tumoral injection have significant superior effect in vivo

Prof. Erez Levanon

Bar Ilan UniversityIsrael

Bio:

Erez Levanon is a full professor at the faculty of Life sciences of Bar-Ilan University where he is the head of the genomic research lab. He received his PhD from Tel-Aviv University after graduating the Adi Lautman Interdisciplinary Program and was a postdoctoral research follow at the genetics department of Harvard Medical School. Prior to that, he was a senior scientist in Compugen LTD. He has published over 80 research manuscripts and won several awards and fellowships. His main contribution is in the study of RNA and DNA editing.

Title:

RNA editing by the endogenous ADAR protein

Abstract:

Modifications of RNA affect its function and stability. RNA editing is unique among these modifications because it not only alters the cellular fate of RNA molecules but also alters their sequence relative to the genome. The most common type of RNA editing is A-to-I editing by double-stranded RNA-specific adenosine deaminase (ADAR) enzymes. Recent transcriptomic studies have identified a number of ‘recoding’ sites at which A-to-I editing results in non-synonymous substitutions in protein-coding sequences. However, systematic mapping of the editome across the animal kingdom has revealed that most A-to-I editing sites are located within mobile elements in non-coding parts of the genome. Editing of these non-coding sites is thought to have a critical role in protecting against activation of innate immunity by self-transcripts. Both recoding and non-coding events have implications for genome evolution and, when deregulated, may lead to disease. Finally, ADARs are now being adapted for RNA engineering purposes.

Prof. Michal Lotem

Hadassah University HospitalIsrael

Bio:

Title:

Improving anti-cancer T cells via alternative splicing modifications

Abstract:

Alternative splicing (AS) involves the selective inclusion and exclusion of exons from a nascent pre-mRNA that results in various protein sequences. Activated T cells display dynamics in isoform ratios of immune modulatory receptors and of splicing factors. Splicing variants of immune receptors may act in opposite directions but splicing factors are the major category of proteins that undergoes splicing shifts. By the use of splice-switching antisense oligonucleotides or splice-disrupting gene edits, a new avenue of transcriptional manipulations opens up to produce T cells with improved tumoricidal capacity for successful implementation of adoptive cell therapy against cancer.

Prof. Marcelle Machluf

TechnionIsrael

Bio:

Title:

NanoGohost not just a drug/gene delivery system

Abstract:

Prof. Daniel Offen

Tel Aviv UniversityIsrael

Bio:

For more than 30 years, Prof. Offen work has focused on the biology of neurodegenerative diseases. His lab, in Tel Aviv University, was the first to describe the programmed death of dopaminergic cells in Parkinson's disease. This discovery was followed by a series of studies on neuroprotection by anti-apoptotic anti-oxidants and treatments with stem cells. Prof. Offen’s work has appeared in over 200 publications, with a high H-Index (67) and more than 13,000 citations.  Prof. Offen is a co-inventor of more than three dozen patents, on stem cells, exosomes, CRISPR and gene therapy, some of which served as the basis for biotech companies (Brainstorm Cell Therapeutics, eggXYt) and enterprise startups.

Title:

Exosomes derived from mesenchymal stem cells improved core symptoms of mouse models of Autism

Abstract:

Reut Horev1,2, Nisim Perets1,2, Yona Gefen3, Daniel Offen1,2

1Sackler School of Medicine, 2Sagol School of Neuroscience, Tel Aviv University, 3Stem Cell Medicine Ltd, Israel

Autism Spectrum disorder (ASD) is a neurodevelopmental disorder, mainly characterized by impairment of social interaction, communication deficits, and restricted interests. Currently, there is no pharmacologic medication that can reverse ASD symptoms. Using Shank3 InsG3680 mouse model of autism, mutation that present in 1% of ASD, we show that intranasal administration of exosomes derived from adipose mesenchymal stem cells has a remarkable ability to improve autistic-like behaviors. The treatment shows significant improvement in social interaction, communication, and risk assessment that preserved six months after single intranasal administration. Thus, our results suggest that non-invasive exosomes treatment might be used for ameliorating autistic-like phenotypes in patients.

Prof. Benjamin Reubinoff

HUJI-HadassahIsrael

Bio:

Prof. Reubinoff is a full Professor of Obstetrics and Gynecology and serves as Chairman of the Department of Obstetrics and Gynecology at Hadassah University Medical Center, Jerusalem, Israel. He is also the director of the Sidney and Judy Swartz Embryonic Stem Cell Research Center of The Goldyne Savad Institute of Gene Therapy at Hadassah. He has founded and serves as the Chief Scientific Officer (CSO) of Cell Cure Neurosciences Ltd. 

The major focus of Prof. Reubinoff’s research has been human embryonic stem cells (hESCs).  He has been among the world pioneers in deriving ES cell lines from human embryos. 

Prof. Reubinoff is mainly interested in the development of the technology that may eventually allow the exploitation of hESCs for regenerative medicine. Towards this goal, he developed hESC lines that are suitable for clinical transplantation use, and further derived progeny from these hESC lines for Cell Therapy in neural and retinal degenerative disorders.

Title:

Phase I/IIa Clinical Trial of Human Embryonic Stem Cell (hESC)-Derived Retinal Pigmented Epithelium (RPE) Transplantation in Advanced Dry Form Age-Related Macular Degeneration (AMD)

Abstract:

Authors:
Banin, Eyal1, Reimann, Christopher2, Barak, Adiel3; Boyer, David4; Ehrlich, Rita5; Jaouni, Tareq2; McDonald, Richard6; Telander, David G.7; Keane, Michael8; Ackert, Jessica8; Ferguson, Mark D.8; Shabat, Avi Ben-9; Monés, Jordi M.10; Velez, Joyce11; Hogge, Gary S.11; Reubinoff, Benjamin12

Institutions:

1. Center for Retinal and Macular Degenerations, Department of Ophthalmology, Hadassah-Hebrew University Medical Center, Jerusalem, Israel. 2. Cincinnati Eye Institute & University of Cincinnati School of Medicine, Cincinnati, Ohio, USA. 3. Sourasky Medical Center, Tel Aviv, Israel. 4. Retina Vitreous Associates Medical Group, Los Angeles, CA, USA. 5. Rabin Medical Center, Petah Tikva, Israel. 6. West Coast Retina Group, San Francisco, CA, USA. 7. Retinal Consultants Medical Group, Sacramento, CA, USA. 8. Gyroscope Therapeutics, Ambler, PA, USA. 9. Lineage Cell Therapeutics, Inc. (Cell Cure Neurosciences, Ltd.,) Jerusalem, Israel. 10. Institut de la Màcula, Barcelona, Spain. 11. Lineage Cell Therapeutics, Inc., Carlsbad, CA, USA. 12. Center for Embryonic Stem Cells Goldyn Savad Institute of Gene Therapy and the Department of Gynecology and Obstetrics, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.

Transplantation of RPE cells may be of therapeutic benefit in AMD. We developed RPE cells from hESCs using cGMP directed differentiation. Safety and tolerability of these cells is being evaluated in a Phase I/IIa clinical study in patients with dry AMD and geographic atrophy (GA) (NCT02286089). We report accumulated safety and imaging data from 17 subjects. 
RPE cells in suspension (OpRegen; 50-200k) were subretinally transplanted under local anesthesia to the worse vision eye using either pars plana vitrectomy (PPV) and retinotomy or via an alternative surgical approach utilizing Gyroscope's Orbit Subretinal Delivery System. RPE cells ready for onsite thawing and immediate transplantation have also been evaluated. Short course perioperatively systemic immunosuppression is used. Systemic and ocular safety is closely observed, and retinal function and structure are monitored using various imaging modalities.
Treatment has been well tolerated and there have been no unexpected adverse events (AEs) or treatment-related systemic serious AEs.  Visual improvement has been noted in all patients of cohort 4. In several subjects, within the area of RPE cell transplant, improvements of the ellipsoid zone and RPE layers at the border of GA, as well as directional growth changes in the area of GA, has been seen. Persistent changes observed following treatment include, alterations in drusen appearance, subretinal pigmentation and hyper-reflective areas, suggestive of the presence of transplanted RPE cells. In conclusion, subretinal transplantation of hESC-derived RPE cells in patients with dry AMD and GA appears well tolerated. Imaging findings suggest presence of transplanted cells in the subretinal space. Encouraging structural and clinical changes observed in some patients will require additional follow-up

Prof. Ronit Satchi-Fainaro

Tel Aviv UniversityIsrael

Bio:

Prof. Ronit Satchi-Fainaro (Ph.D.) is a Full Professor at Tel Aviv University, Head of the Cancer Research & Nanomedicine Laboratory at the Department of Physiology & Pharmacology at the Sackler Faculty of Medicine, The Lion Chair in Nanosciences and Nanotechnologies and the Director of the Cancer Biology Research Center. She received her B.Pharm. from the Hebrew University in Jerusalem, her Ph.D. from the University of London and a Postdoctoral Research Fellowship at Harvard University and Children’s Hospital Boston. She joined Tel Aviv University in 2006. The overarching goal of Prof. Satchi-Fainaro’s multidisciplinary research is to establish preclinical models of cancer in order to rationally design novel clinically-translatable nanomedicines targeting tumor cells and their stroma. Prof. Satchi-Fainaro serves on several Boards of Directors of pharmaceutical companies and hospitals, VCs and Editorial Boards, and is a founder of a start-up biotech company. She published more than 150 manuscripts and is named inventor on 60 patents.

Title:

Fighting melanoma brain metastases in 3 dimensions

Abstract:

Despite the remarkable efficiency of immune checkpoint modulators against metastatic melanoma, there is a low percentage of responders and clinical trials report severe immune-mediated side effects and disease relapse. Recent evidences show that non-tumor cells within the tumor microenvironment (TME), including tumor vasculature and immune stromal cells, dictate the overall therapeutic efficacy. We synthesized off-the-shelf and cost-effective nano-sized polymeric platform that combines a cancer vaccine with the targeted inhibition of molecular and/or cellular immune suppressive players. These precision nano-sized medicines aim to re-educate and harness patient T-cell response against tumors, leading to an immunological memory able to control tumor relapse without any follow-up treatment. The design of these advanced immunotherapies is guided by the identification of lead immune suppressor factors and tumor specific antigens using novel 3D bio-printed tumor-immune spheroids developed in our lab. Our first nano-immunotherapy candidates sensitized melanoma mouse models to immune-checkpoint modulators, dramatically increasing disease-free survival rates.

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