Dr. Porteus was raised in California and was a local graduate of Gunn High School before completing A.B. degree in “History and Science” at Harvard University where he graduated Magna Cum Laude and wrote an thesis entitled “Safe or Dangerous Chimeras: The recombinant DNA controversy as a conflict between differing socially constructed interpretations of recombinant DNA technology.” He then returned to the area and completed his combined MD, PhD at Stanford Medical School with his PhD focused on understanding the molecular basis of mammalian forebrain development with his PhD thesis entitled “Isolation and Characterization of TES-1/DLX-2: A Novel Homeobox Gene Expressed During Mammalian Forebrain Development.” After completion of his dual degree program, he was an intern and resident in Pediatrics at Boston Children’s Hospital and then completed his Pediatric Hematology/Oncology fellowship in the combined Boston Chidlren’s Hospital/Dana Farber Cancer Institute program. For his fellowship and post-doctoral research he worked with Dr. David Baltimore at MIT and CalTech where he began his studies in developing homologous recombination as a strategy to correct disease causing mutations in stem cells as definitive and curative therapy for children with genetic diseases of the blood, particularly sickle cell disease. Following his training with Dr. Baltimore, he took an independent faculty position at UT Southwestern in the Departments of Pediatrics and Biochemistry before again returning to Stanford in 2010 as an Associate Professor. During this time his work has been the first to demonstrate that gene correction could be achieved in human cells at frequencies that were high enough to potentially cure patients and is considered one of the pioneers and founders of the field of genome editing—a field that now encompasses thousands of labs and several new companies throughout the world. His research program continues to focus on developing genome editing by homologous recombination as curative therapy for children with genetic diseases but also has interests in the clonal dynamics of heterogeneous populations and the use of genome editing to better understand diseases that affect children including infant leukemias and genetic diseases that affect the muscle. Clinically, Dr. Porteus attends at the Lucille Packard Children’s Hospital where he takes care of pediatric patients undergoing hematopoietic stem cell transplantation.

Prof. Cyrille Cohen heads the Laboratory of tumor immunology and immunotherapy at Bar-Ilan University, Israel. He has been active in the field of cancer immunotherapy for the past 20 years and his current research projects aim at genetically inducing anti-tumor immune responses by designing TCRs and CARs with broad tumor specificity, and by manipulating T-cell co-stimulatory and metabolic pathways to achieve superior cellular function. His group also described approaches to isolate on neo-antigen specific TILs and peripheral T-cells and is developing, together with medical centers, novel therapies for advanced cancer – some of which are now evaluated in clinical trials. He has more than 70 publications in peer-reviewed journals and patents in the field T-cell immunotherapy. He serves as president of the Israel Society for Cancer Research and as a member of regulatory committees for clinical trials based on gene and cell therapy at the Israel ministry of health.

Prof. Aviva Fattal-Valevski is Professor of Pediatrics, Incumbent of the Adler Chair in Pediatric Cardiology, Sackler Faculty of Medicine, Tel Aviv University, and director of the Pediatric Neurology Institute, Dana-Dwek Children’s Hospital, Tel Aviv Sourasky Medical Center in Israel. Her main fields of interest are rare metabolic and neurogenetic disorders, with a focus on early diagnosis and new gene therapies. She has been a member of scientific committees and advisory boards of Biogen SMA European Expert Forum, AveXis Advisory Board for Zolgensma, The Sackler Institute for Nutrition Science - New York Academy of Sciences (thiamine), and Israel Ministry of Health – Committee for New Drugs and Medical Technologies.

Full Professor of Gene and Cell Therapy at the Hebrew University of Jerusalem in Israel. I directed the Gene and Cell Therapy Institute at the Hadassah Hebrew University Hospital for 23 years, which I established in 1998. This entailed the collection of funding for the building of the Institute during the initial stage and recruiting and funding all of the research activities from grant money, collaborations with industry and fundraising. Today, we are 10 research groups, with 70 investigators. In addition to the research groups, I established a GMP-level production facility with four clean rooms engaged in the production of biological materials, including viral vectors, devices, therapeutic cells, e.g., mesenchymal stem cells and human embryonic stem cells, administered to humans in phase I/II studies.

Major achievements in my recent academic career: My achievements are concentrated in the field of translational medicine and include: 1. Development of hepatitis B and C virus monoclonal antibodies that progressed into phase II clinical studies, and the development of the Trimera HBV and HCV mouse models to assess the anti-viral effects of these antibodies. 2. Understanding the molecular and cellular factors that play a role in the biological axis of liver inflammation towards regeneration for hepatocellular cancer. 3. Development of novel gene therapy platforms, e.g., the usage of ultrafast infrared femtosecond laser for dermal, muscle and retinal naked DNA transfection.

Current research interests: Our group investigated the role of inflammatory factors contributing to liver regeneration upon infection. While investigating these factors along the years, we learned that the IL6-gp130 pathway is pivotal for the regeneration of liver parenchyma. This understanding further engaged us in studies aimed at translating the knowledge generated to determine the role of IL6-gp130 in the regeneration and anti-apoptosis in other tissues. Our current data show that this pathway is involved in the protection and regeneration of the kidney, heart, bone marrow stem cells and the salivary gland. In the recent years we have also been investigating the significance of microRNAs in the regulation of physiological and pathological human conditions by studying mouse models. These have revealed some astonishing results. One is the role of miR 122 in controlling lipid metabolism, influencing anemia development and encountering anti-tumor properties. 

Translational research: Our researchers at the Institute and I, in particular, are believers in translational investigation. During recent years, I have been involved in a number of programs and these include: 1. Development of a novel therapy for pancreatic cancer based on siRNA targeting K-Ras (I established a company around this technology called Silenseed); 2. Development of FIV as a novel lentiviral vector for gene therapy. 3. Identification of new therapeutic targets for liver cancer. 4. Development of novel imaging technologies for the identification of liver pathology, tools that were further used in our program on liver inflammation for regeneration of hepatocellular cancer.

Director of the Hadassah Biologicals Production Site (Good Manufacturing Production, GMP): I established the Hadassah GMP facility for biologicals in 1997. Since its creation, we have produced 50 different 1^st in man products. These were either our products of the Gene and Cell Therapy Institute, or from the hospital or collaborations with outside investigators and companies. This year, we reached an unprecedented achievement: We are the only worldwide academic facility that received a phase 3 license from the Israeli government’s Ministry of Health to produce a therapy. This product is also approved by the FDA for a phase 3 trial.

Roi Gazit, PhD. is a Principal Investigator of Hematopoietic Stem Cell (HSC) and Immunology at the Ben-Gurion University of the Negev. He is an active member of NIBN, ISCS, IIS, and ISEH.

Roi earned a B.Sc. and M.Sc. in life science and developmental biology from the Hebrew University of Jerusalem. He obtained PhD in immunology, under supervision of Ofer Mandelboim, studying NK-cell in vivo, including the generation of the first NK specific mouse model and elucidation of novel NK-cell abnormalities in human patients. He conducted postdoctoral studies at the Harvard Medical School, at Derrick Rossi laboratory, focusing on the identification and utilization of HSCs’ genes, including reprogramming of committed blood cells into HSCs using defined transcription-factors and generation of a novel HSC-reporter mouse. His laboratory at the Ben-Gurion University is studying HSCs’ transcriptome along normal development and during immune-challenges.

Current research interests of Gazit lab includes:

1. Transcriptional regulation of HSCs, and reprogramming into HSC-state.
2. Immune-activation impact on HSCs.
3. Generate novel leukemia models, and identify Leukemia-Initiating-Cells in immune competent mice.

Yona Geffen, Ph.D., M.Sc.

Vice President of Research and Development Gamida-Cell.

Yona Geffen joined Gamida Cell’s leadership team as Vice President of Research and Development in December 2020, bringing over two decades of experience in leading clinical and drug development in the biotechnology and pharmaceutical industry. Prior to joining Gamida Cell, Yona served in different positions including Vice President, Research and Development at Stem Cell Medicine, Vice President, Research and Validation at Compugen and several roles including Senior Vice President, Clinical Development, Chief Operations Officer and Chief Executive Officer at Avraham Pharmaceuticals.

Yona holds a Ph.D. in immunology and microbiology and an M.Sc. in business management from the Ben-Gurion University, Israel.

Dr. Ayal Hendel is a Principal investigator in the Mina and Everard Goodman Faculty of Life Sciences at Bar-Ilan University (https://www.hendelab.com/). Dr. Hendel’s research focuses on developing genome editing as a curative therapy for childhood disorders and for cancer immunotherapy applications. Dr. Hendel received his BA with High Honors from the Hebrew University of Jerusalem in Biology and his MSc and PhD from the Weizmann Institute of Science with Prof. Zvi Livneh. Dr. Hendel carried out post-doctoral research with Dr. Matthew Porteus at Stanford University School of Medicine where he was part of a team that achieved therapeutically relevant genome editing frequencies in human stem cells confirming the potential of this technological breakthrough (PCT/US2016/026028 & PCT/US2016/053344). He is the Chair of the CRISPR-IL scientific committee (https://crispril.com/) and a member of the 8400 Health Network, which aims to gather leaders at the intersection of health and technology to help Israel become a life science powerhouse. He also serves as an Associate Editor in the journal Frontiers in Genome Editing. Dr. Hendel won the ERC Starting Grant of the H2020 program and He is a working group leader in the EDITSCD consortium funded by Horizon Europe which involves eight academic and industrial partners, aims at assessing the efficacy and safety of genome editing approaches for sickle cell disease.

Elad Jacoby graduated from the Faculty of Medicine, Tel Aviv University, and completed his residency at Sheba Medical Center and post-doctororal fellowship at the Johns Hopkins Hospital and the National Institutes of Health. He heads the cell therapy center in Pediatrics at the Sheba Medical Center, and is a Senior Lecturer in Pediatrics at the Faculty of Medicine, Tel Aviv University. He is a board certified pediatric hematologist and oncologist, and an expert in childhood leukemia and in CAR-T cells.

Dr Jacoby leads clinical trials in advanced cellular therapies including CAR-T cells, first-in-human mitochondrial augmentation and anti-viral T-cells.  His translational research interests are in the field of CAR-T cell biology, leukemia resistance mechanisms, and mitochondrial metabolism of hematopoietic cells.

Shlomit Kfir-Erenfeld, Ph.D., is the manager of the Advanced Cellular Therapies Laboratory affiliated to Bone Marrow Transplantation and Cancer Immunotherapy Department at Hadassah Hebrew University. Dr. Shlomit Kfir-Erenfeld conducted her Ph.D and post-Ph.D studies at the Lautenberg Center of Immunology and Cancer Research, at the Hebrew University of Jerusalem. Her research focused on the mechanisms underlying the response of various hematological malignancies to chemotherapy. Five years ago, she established a new laboratory for adoptive cell transfer therapies and together with her colleagues and outstanding team, she developed a novel BCMA-targeted CART cell therapy for relapsed and refractory multiple myeloma. Since then, more than seventy Israeli myeloma patients received these engineered T cell therapy, displaying an overall response rate of 87%. Last year, three papers describing her studies were published in leading scientific journals with high impact factors. Dr. Shlomit Kfir-Erenfeld is currently working on developing new strategies to overcome myeloma resistance and/or relapse after BCMA-targeted CART therapies. Today, she aims at developing novel modalities and new lines of treatment to prolong life expectancy and improve quality of life of myeloma patients.

Dr. Arthur Machlenkin joined Pluri in May 2022 and serves as Chief Scientific Officer. He is a biotech industry professional with nearly 20 years of academic and industrial experience at the intersection of drug discovery and development. He was most recently CEO at Pantheon Biosciences, an Israeli early-stage biotechnology company translating the bacterial immunity into antiviral platforms and therapeutics. As CEO, he led Pantheon’s vision and strategy, built an international team of top-notch researchers and academic collaborators. Prior to Pantheon, Arthur spent four years at Compugen where he was a Head of Immuno-Oncology (I-O) Research with responsibility for building the I-O infrastructure of the company and for target validation process. Before Compugen, Arthur spent 10 years in Hadassah Medical Center as Principal Investigator. He co-led (with Prof. Lotem) basic and translational research, discovery and development of innovative anticancer cell therapies including adoptive cell transfer and genetically modified tumor vaccines. Arthur has a PhD from Weizmann Institute of Science.

Prof. Rachela Popovtzer is the Vice Dean of the Faculty of Engineering at Bar Ilan University, and a member of the Bar-Ilan Institute of Nanotechnology and Advanced Materials (BINA). She has previously served as head of the Bioengineering Track at Bar Ilan University, and was a visiting Professor at Memorial Sloan Kettering Cancer Center, NY. Rachela has a multidisciplinary background in physics, biomedical and electrical engineering. Currently, her lab focuses on theranostic nanoparticles as targeted drug carriers and real-time imaging contrast agents, for cancer, brain disease, highlighting the value of nanoparticles as multifunctional elements. Prof. Popovtzer is a winner of numerous national and international grants, from the NIH, ERC, Horizon2020, ISF, Israel Innovation Authority, the Ministry of Science and Technology, and more. She is also the co-founder (2021) and CTO of NanoCarry LTD., an Israeli biotechnology company that develops a nanoplatform for the delivery of biologics across the BBB.

Dr. Liat Rockah is the Vice President of Nuclease Technologies. Since joining Emendo in 2016, Liat has been responsible for leading the discovery, characterization and engineering of CRISPR-based novel nucleases. Liat directs the optimization of Emendo’s nucleases to achieve gene-editing based composition solutions that successfully target a desired indication, by using enzyme engineering and directed evolution and computational-driven design, such as machine-learning. Liat brings more than 15 years of experience and knowledge in the evolution of various mechanisms of the arms race of bacterial immune systems: the Toxin-Antitoxin, Restriction-modification, and CRISPR-Cas systems. Liat holds a Ph.D. in Biochemistry and Protein Engineering from the Weizmann Institute with Prof. Dan Tawfik and further has been awarded post-doctoral fellowships with Prof. Ron Milo in synthetic metabolic pathway engineering.

Dr. Marianna Truman has over 17 years of R&D experience in molecular and cell biology, biotechnology engineering, toxicology and applied research. She currently serves as a Research Associate Director at Pluri Biotech, where she leads the nonclinical cell therapy programs, focusing on the mechanism of action, pharmacokinetics and pharmacodynamics of the PLacental eXpanded (PLX) allogeneic cell product, and its pipeline development for regenerative medicine. Marianna did her postdoc at the US National Toxicology Program Laboratory (NIH/NIEHS, NC, USA) and developed 3D architecture systems suitable for high-throughput screening for kidney and liver toxicity. As a toxicologist at the Cancer Biology and Cannabinoid Research Laboratory (Biology Faculty, Technion – IIT), she explored the effects of cannabis extracts on the kidneys by developing experimental models for human renal barriers. During her PhD studies (Biotechnology and Food Engineering, Technion – IIT), Marianna investigated the mechanism of action and regulation of ferritin trafficking and secretion and discovered two molecular secretion pathways for ferritin. During her military service, Marianna served as a Research Navy Officer at the Israel Naval Medical Institute and investigated the recovery from CNS oxygen toxicity. Dr. Truman also serves as a Lecturer in Toxicology at the Technion - IIT, and has presented at conferences and congresses worldwide.

Dan Peer is a Professor and the Director of the Laboratory of Precision NanoMedicine at Tel Aviv University (TAU). He is also the Vice President for Research and Development at Tel Aviv University. From 2016 - 2020, he was the Chair of Tel Aviv University Cancer Biology Research Center that includes 17 affiliated hospitals and from 2017 - Present, he is the Founding and Managing Director of the SPARK program of Translational Medicine at TAU. Prof. Peer is also the Chairman of Ramot, TAU Tech Transfer Company and Chairman of TAU Ventures, the Venture arm of TAUץ

Prof. Peer’s work was among the first to demonstrate systemic delivery of RNA molecules using targeted nanocarriers to the immune system and he pioneered the use of RNA interference (RNAi) in immune cells. In addition, his lab was the first to show systemic, cell specific delivery of modified mRNA to cells to induce therapeutic gene expression of desired proteins within the immune system that has enormous implications in cancer, inflammation and infection diseases (e.g. COVID 19 mRNA vaccines). In addition, his lab was the first to show high efficiency, systemic, cell specific therapeutic genome editing in cancer.

Prof. Peer has more than 130 pending and granted patents. Some of them have been licensed to several pharmaceutical companies and one is currently under registration (as a new biological drug in Inflammatory Bowel Disease). In addition, based on his work, five spin-off companies were generated aiming to bring innovative personalized medicine into clinical practice. 

Prof. Peer received more than 30 awards and honors and he serves on the scientific advisory board of more than 15 companies, and on the editorial board of more than 20 journals. He is also an Associate Editor of the Journal of Controlled Release. Prof. Peer is a past President of the Israeli Chapter of the Controlled Release Society, and a Past Member of the Board of the Israel Young Academy.

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.

Laura Sepp-Lorenzino, Ph.D. oversees Research and Early Development at  Intellia Therapeutics in her role as Chief Scientific Officer. Intellia’s leading CRISPR/Cas9-based platform supports a full-spectrum strategy, which deploys differentiated, modular solutions across in vivo and ex vivo therapeutic applications. Before joining Intellia, Laura was VP, Head of Nucleic Acid Therapies and member of the External Innovation team at Vertex Pharmaceuticals, with previous positions as VP and entrepreneur-in-residence at Alnylam Pharmaceuticals, Inc., and Executive Director RNA Therapeutics at Merck & Co., Inc. In addition to her extensive experience in nucleic acid therapies, Laura has expertise in oncology drug discovery and development acquired earlier in her career by leading the Cancer Research Department at Merck West Point and working as an assistant attending molecular biologist at Memorial Sloan-Kettering Cancer Center. She received her professional degree in Biochemistry from the University of Buenos Aires, Argentina and both her M.S. and Ph.D. in Biochemistry from New York University. Laura is a member of the board of directors of Taysha Gene Therapies, the Alliance for Regenerative Medicine and the Oligonucleotide Therapeutics Society, and sits on the scientific advisory board of Thermo Fisher Scientific, Zymergen Inc., and the U.K. Nucleic Acid Therapy Accelerator.

Affiliation:

Dotan center for Advanced Therapies, Tel Aviv Sourasky Medical Center

Faculty of Medicine, Department of Pathology, Tel Aviv University

Faculty of Life Sciences, School of Neurobiology, Biochemistry and Biophysics, Tel Aviv University

Eric Shifrut is a senior lecturer at Tel Aviv University and a principal investigator at Tel Aviv Sourasky Medical Center. Eric completed his graduate studies under the mentorship of Prof. Nir Friedman at the Weizmann Institute of Science, mapping the TCR repertoire in health and disease. For his postdoctoral training, he joined Alex Marson at UCSF and Gladstone Institutes to pioneer CRISPR discovery platforms to study primary human T cells. Today, Eric leads an independent team with the mission to leverage these achievements in CRISPR engineering to design best-in-class T cell therapies against cancer.