ONLINE EVENT: Translation Hub Organoids and Cell Engineering - Project Update

The online event ”Hub Organoids and Cell Engineering - Project Update” will feature projects funded by the Translation Hub and is intended to give the community an overview about past and ongoing projects and research activities.

The Organoids and Cell Engineering Hub aims to harness new approaches in genetic engineering (e.g. CRISPR/Cas) or cell breeding, stem cell technology and bioprinting (e.g. organoids, human-on-a-chip models) to facilitate precision medicine through the development of new human models for preclinical research and the methodical development of cell-based therapies. This includes the use and improvement of new 2D and 3D cell culture models such as organoid, human-on-a-chip or mini-organ models. Human cells and their genetically edited forms can be examined under almost physiological conditions for a variety of enquiries, for example, regarding functional characteristics or processes of vascularization.

To promote this mission, we are delighted to present three talks featuring projects funded by the Translation Hub in 2019:

1:10 - 1:25 pm
Prof. Dr. Johannes Schulte
Charité | Department of Pediatrics, Division of Oncology and Hematology

"Assessing Therapeutic Options for High-Risk Neuroblastomas in Patient-derived Models"

Despite intensive multimodal therapy, more than 50% of high-risk neuroblastoma relapse, which mostly results in a fatal outcome. We demonstrated in the past that RAS/p53 mutations delineate an ultra-high risk group of neuroblastoma with an especially adverse prognosis. Both, the de novo acquisition of mutations resulting in activation of components of the RAS pathway, and tumor evolution of initial subclones harboring those underline their mechanistic importance in the progressive high-risk disease and suggest targeted treatment options along ALK/RAS/MEK pathway signaling. We hypothesized that patient-derived xenograft (PDX) mouse models and patient-derived primary 3D (PD3D) culture models closely recapitulate the conditions in the patients and present bona fide model systems to investigate therapy evasion and the mechanistic relevance of underlying, secondary mutations. Monitoring the response of alternative, targeted treatment approaches in PDX/PD3D should allow for an early evaluation of potential success or risk of therapy-evasion and may inform about required treatment adaptation.

1:25 - 1:40 pm
PD Dr. Annette Künkele
Charité | Department of Pediatrics, Division of Oncology and Hematology

Report on Workshop: “How to Translate ATMPs Into the Clinic”

Advanced Therapy Medicinal Products (AMTPs) are medicines for human use that are based on genes, tissues or cells. They offer groundbreaking new opportunities for the treatment of disease and injury but to bring AMTPs into the clinic is a huge challenge. Complex manufacturing processes, implementation of Good Manufacturing Practices (GMP) specifically for cell and gene products, complex trial designs and regulatory requirements are just a few of the difficulties AMTP developers have to deal with.
In order to learn more about those difficulties and how to overcome them, three speakers from different areas were invited (regulatory authorities, quality risk management in ATMP development, IMPD preparation for ATMPs, Crispr/Cas technology): 1) Gabriele Dallmann “IMPDs and ODDs for ATMPs” (Biopharma Excellence GmbH), 2) Markus Hoffmann “Quality Risk Management” and 3) Shaun Stapleton “Preparing for Clinical Trials with Cell and Gene Therapies – A Journey Through the FDA” (Rapport – global strategies services). The aim was to offer an interactive workshop requiring active contribution of selected participants.

1:40 - 1:55 pm
Prof. Dr. Simone Spuler
MDC, Charité | ECRC - Experimental and Clinical Research Center and Charité | Outpatient Clinic for Muscle Disorders

"Humanized Immuno-Compromised Animal Models"

Advanced therapeutic medicinal products (ATMP) were defined as an own entity in 2008 (EG Nr. 1394/2007) and comprise of biological and cellular therapies. Cell therapies are, in the case of engineered T cells (with either CARs or TCRs), dramatically changing therapeutic and prognostic approaches to hematopoietic and also to solid cancers. Primary, organ specific, stem cell therapies, as for muscular dystrophies, still have to prove their efficacy. Although research on organoids is suitable to replace animal experiments to some extend, research in whole organisms is essential to translate new therapeutic concepts into clinical practice. We have created several new humanized mouse models and demonstrate their usefulness for gene- and cell therapy development.
Applicants for this project were (alphabetical): Annette Künkele, Ralf Kühn, Klaus Rajewsky, Chiara Romangnani, Simone Spuler, Gerald Willimsky

 

Registration

Due to the current situation regarding the pandemic, the Translational Hub Project Update will be held online via GoToMeeting. To participate in the online event, please register in advance here. The registration will be open until maximal participant capacity is reached.

Login via GoToMeeting

Please note that the login link and access code are not sent out immediately after your registration! You will receive both the evening before the event. You have the following possibilities to participate:

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Discussion via Chat Room

The discussion will be held via the Chat Room function of GoToMeeting. If you would like to join the discussion, please type in your question into the chat area and moderators will pose the question to the speaker or arrange a dialogue if applicable.

Thank you for your cooperation!