Partnering Opportunities at IMBA

SCONs - Short Conditional intrONs FOR ONE-STEP GENERATION OF CONDITIONAL ANIMAL MODELS

Gene knockouts (KO) in cells or animals have made substantial contributions to understanding molecular biology. For essential or developmentally active genes, a conditional knockout (cKO) strategy is often required to elucidate their functions. To prevent potential developmental or other unwanted physiological effects, the expression level of such targeted genes needs to be unaffected. This remains a major, unresolved challenge. “SCONs” (Short COnditional INtrons) were developed to overcome these limitations and are based on genomic integration of the SCON within an exon. SCONs do not interfere with natural gene function or expression level and do not display hypomorphic effects. Upon transcription, an additional splicing event takes place which removes the inserted SCON, reconstituting the original mRNA sequence. The conditional knock out is induced by a recombinase-based genomic event which partially removes the SCON and destroys its function.  IMBA is actively seeking licensing partners with business interests in the field of animal model generation or novel CRISPR applications, to further exploit applications for the SCON technology.

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HIGHLY IMPROVED REPROGRAMMING EFFICIENCY FOR IPS CELL GENERATION
 

Generating induced pluripotent stem cells (iPSCs) with cellular reprogramming has become a broadly used experimental tool. Beyond its use in basic and biomedical research and disease modelling, iPSC reprogramming is employed in a wide range of medical applications. Increased efforts in the development of clinical applications for iPSCs in the field of regenerative medicine and cell therapy in particular have led to a high demand for reliable and efficient iPSC methodologies. IMBA scientists have developed a new method that has the potential to increase the efficiency of iPSC reprogramming by a factor of 100+.  IMBA is looking for technology partners in the field of stem cell research or manufacturing to further develop the method to create a new generation of iPSC reprogramming kits and/or use the technology for iPSC contract manufacturing.
 

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Link to IMBA Stem Cell Facility
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CRISPR SWITCH: INDUCIBLE SGRNA EXPRESSION FOR TIGHTLY REGULATED, EFFICIENT & SAFER CRISPR/CAS9 GENOME EDITING

CRISPR/Cas9 provides an easy, efficient and affordable tool for site-specific manipulation of genomes. Despite its promising characteristics, the technology needs further improvement, e.g. with regard to efficiency, regulation and reduction of off-target effects. To address these challenges, IMBA and Vienna BioCenter Core Facilities GmbH (VBCF) scientists have developed CRISPR switch (i.e. optimised sgRNA expression cassettes) to facilitate (i) tight ON and OFF switches of sgRNA expression, (ii) OFF switches without deletion of the guiding sequence for easy sgRNA target identification, (iii) consecutive sgRNA expression for studying aspects such as synthetic lethality, temporal order of lesions in tumour progression, etc. Importantly, CRISPR switch is compatible with variations of CRISPR such as CRISPR-a, CRISPR-i and others.  IMBA is looking for licensing partners with business interests and R&D programs in the field of CRISPR/Cas9, i.e. screening, mutation analysis, safer genome editing, multiplexing and/or somatic gene therapy. 
 

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Link to Vienna Biocenter Core Facilities (VBCF)
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GENETICALLY ENGINEERED HUMAN 3D TUMOR MODELS FOR DISEASE MODELING AND COMPOUND EVALUATION
 

Human organoid tumour models have been generated by introducing oncogenic mutations during cerebral organoid formation via transposon- and CRISPR/Cas9-mediated mutagenesis. Based on clinically relevant mutations, the technology has been applied to generate central nervous system primitive neuroectodermal tumour (CNS-PNET)-like and glioblastoma (GBM)-like disease models. In contrast to patient-derived tumour models (xenografts, tumour cell lines), this novel platform technology allows defined genetic models to be created that contain tumorous and healthy tissue in the same organoid derived from the same human stem cell origin. Such disease models can be cultured over months and are suitable for various applications in the field of human disease modelling, drug discovery and therapy development. IMBA is looking for licensing partners with business interests in the field of organoid technologies, preclinical testing or brain cancer therapies to exploit the proprietary disease models and/or to expand the technology platform further.
 

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